Stem Cell Therapy Market Research Report by Cell Source (Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs), by Type (Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy), by Therapeutic Application, by End-User, by Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2026 - Cumulative Impact of COVID-19

New York, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region - Global Forecast to 2026 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06175517/?utm_source=GNW

The Global Stem Cell Therapy Market size was estimated at USD 202.87 million in 2020 and expected to reach USD 240.88 million in 2021, at a CAGR 19.07% to reach USD 578.27 million by 2026.

Market Statistics:The report provides market sizing and forecast across five major currencies - USD, EUR GBP, JPY, and AUD. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2019 are considered historical years, 2020 as the base year, 2021 as the estimated year, and years from 2022 to 2026 are considered the forecast period.

Market Segmentation & Coverage:This research report categorizes the Stem Cell Therapy to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Cell Source, the market was studied across Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs.

Based on Type, the market was studied across Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy.

Based on Therapeutic Application, the market was studied across Cardiovascular Diseases Surgeries, Inflammatory & Autoimmune Diseases, Musculoskeletal Disorders, Neurological Disorders, Other Therapeutic Applications, and Wounds & Injuries.

Based on End-User, the market was studied across Academic and Research Centers, Ambulatory Surgical Centers (ASCs), and Hospitals & Clinics.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, and Thailand. The Europe, Middle East & Africa is further studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Stem Cell Therapy Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Stem Cell Therapy Market, including Advanced Cell Technology, Inc., AlloSource, Inc., Anterogen Co., Ltd., Bioheart Inc., BioTime, Inc., BrainStorm Cell Therapeutics Inc., Celgene Corporation, Cellartis AB, CellGenix GmbH, Cellular Engineering Technologies Inc., Gamida Cell Ltd, Gilead Sciences, Inc., Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Lonza Group AG, Medipost Co., Ltd., Nuvasive, Inc., Osiris Therapeutics, Inc., Pharmicell Co., Ltd., Pluristem Therapeutics Inc., PromoCell GmbH, RTI Surgical, Inc., STEMCELL Technologies, Inc., Takeda Pharmaceutical Company Limited, Vericel Corporation, and VistaGen Therapeutics, Inc..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Stem Cell Therapy Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Stem Cell Therapy Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Stem Cell Therapy Market?4. What is the competitive strategic window for opportunities in the Global Stem Cell Therapy Market?5. What are the technology trends and regulatory frameworks in the Global Stem Cell Therapy Market?6. What is the market share of the leading vendors in the Global Stem Cell Therapy Market?7. What modes and strategic moves are considered suitable for entering the Global Stem Cell Therapy Market?Read the full report: https://www.reportlinker.com/p06175517/?utm_source=GNW

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Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region - Global Forecast to 2026 -...

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region – Global Forecast to 2026 -… | October 16th, 2021

By Sam Boughedda

Investing.com Shares of biotechnology firm Jasper Therapeutics (NASDAQ:JSPR) soared 112% after Oppenheimer analyst Jay Olson initiated coverage of the company with a buy-equivalent outperform rating and $21 price target.

In a research note released after the close on Tuesday, Olson told investors that he sees Jasper as an "emerging leader" in developing novel targeted conditioning agents for hematopoietic stem cell transplantation.

The company's lead candidate, JSP191, is used to remove hematopoietic stem cells from bone marrow before a transplant.

Olsen believes Jasper Therapeutics is well-positioned to conceivably change what he describes as the "decades-old standard" hematopoietic stem cell transplantation for various diseases "either as a standalone conditioning agent or as the backbone of the conditioning regimen."

At the beginning of October, biopharmaceutical firm Amgen (NASDAQ:AMGN) disclosed a 7.4% stake in Jasper.

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Jasper Therapeutics Stock Soars after Oppenheimer Calls it a Buy - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Stock Soars after Oppenheimer Calls it a Buy – Yahoo Finance | October 16th, 2021

By Alycea Wood and Kamran Zamanian, Ph.D., iData Research Inc.

When choosing a treatment option for orthopedic procedures, biomaterials have become widely popular. Biomaterials are biomedical materials that can be safely implanted or injected into the body and are, more often than not, a form of biologically active tissue themselves.1 Their prevalence in orthopedic procedures is largely attributed to their ability to mimic the structure or properties of osseous tissue. Many products can offer a number of beneficial properties, such as promoting bone growth within the body (osteoinduction), promoting bone growth on the biomaterials scaffold (osteoconduction), or inducing the differentiation of stem cells into osseous tissue (osteogenesis).2,3 The orthopedic biomaterials market includes bone graft substitutes, growth factors, cellular allografts, cell therapy, hyaluronic acid viscosupplementation, and even cartilage repair devices. The U.S. orthopedic biomaterials market saw a dramatic dip and subsequent rebound in market value in 2020 and 2021 as a result of the COVID-19 pandemic. After recovery, the market is projected to see a consistently steady growth in value within the next few years. This growth is expected to be seen across all market segments apart from cellular allograft devices (Figure 1).4

Figure 1: Orthopedic biomaterials market growth trends by market segment, U.S., 20192028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

Cellular allografts may consist of either allograft bone (donated bone tissue) in conjunction with adipose-derived adult stem cells or viable cells within a cortical cancellous bone matrix.4,5 In both scenarios, the devices provide osteoconduction, osteoinduction, and osteogenesis to the site of implantation. Historically, this market had seen promising growth because of the optimal environment for bone growth they can provide.6 The cellular allograft market is projected to see a much slower rate of growth in market value in the next few years despite its market potential due to increased constraints on the market itself. These include, but are not limited to, direct federal restriction on product research, cost of product development, and product recalls.4

There is a strong interest in the scientific community in embryonic stem cell (ESC) research, which is largely due to ESCs high differentiability when compared to adult stem cell (ASC) lines.7 The development of new cellular allograft products, and the resulting growth in the market, is dependent on continued research into realizing the full medical potential of stem cell use. In 2019, the Trump administration eliminated federal funding of research relying on ESC tissue and instituted the National Institutes of Health (NIH) Human Fetal Tissue Research Ethics Advisory Board. This negatively impacted a large number of studies in progress while restricting the ability of new projects to commence.8,9,10 While the board was in effect, it rejected all but one application for funding.11 In April 2021, the Biden administration removed both the board and the restrictions on current projects, allowing federally funded research using ESC to continue.12 This was not the first instance where restrictions were placed and then removed on ESC research. In March 2009, President Obama signed an executive order to overturn the Bush administrations restriction on ESC research.13

The repeated restrictions on ESC research have a number of long-term ramifications in the development and implementation of new, effective cellular allograft treatments. Scientists may need to divert their research efforts away from stem cells and into less turbulent fields, and the progress of product development slows down as studies have funding pulled; this may contribute to increased hesitancy by end users to use stem cell products. Reduced research efforts, funding, and faith in stem cell products will continue to limit the growth of the cellular allograft market.

Cellular allografts tend to be notably more expensive than others within the broader cell-based biomaterials market. When compared to the cell therapy market, which uses either concentrated platelet-rich plasma (PRP) or bone marrow aspirate concentrate (BMAC) in its treatment, the cellular allograft average selling price (ASP) sits over three times higher (Figure 2).3

Figure 2: The average selling price (ASP) of the cellular allograft & cell therapy markets, U.S., 20182028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

The ASP of the cellular allograft market is so high because of the prohibitively expensive cost of developing new products. During the development process, reliable efficacy of a new product is uncertain, and using protein markers to help distinguish stem cell types can be very challenging.4,14 The increased cost of product development acts as a significant barrier to parties looking to enter the U.S. cellular allograft market. The result is fewer products entering and rejuvenating the market, and existing products sit at prohibitively high prices as they have low direct competition.4 The high cost of cellular allograft products hinders new entrants from introducing products and prevents end users from being able to afford existing ones. A broader consequence of this is end users turning to more affordable orthopedic biomaterial types to reduce procedural costs.

Any product recalls within the U.S. orthopedic biomaterials market, especially within cell-based therapies, will negatively impact the use of cellular allografts. This impact is amplified when a recall occurs within the market segment itself, which was seen in the cellular allograft market as recently as June 2021. On June 2, 2021, Aziyo Biologics recalled its product FiberCel following a number of patients contracting tuberculosis.15 Recalls deter the use of cell-based products through increased distrust in the safety of the products themselves, potential public backlash against the specific product itself or, in the market more broadly, reduced reimbursement from health insurance providers as well as the introduction of more restrictive FDA protocols. This is another reason why effective, safe, cell-based products are necessary for the cellular allograft market to move forward.

Conclusion

Federal research restrictions, high development costs, and product recalls all negatively impact the growth of the cellular allograft market in the United States. These constraints contribute to the projected low growth rate in market value in the coming years despite the potential uses for stem cell therapies. To shift the tide back toward growth, the cellular allograft space will need consistent research progress through large-scale studies, more affordable product development, and strict enforcement of sanitization protocols for existing products to prevent future product recalls. The large therapeutic potential of stem cell therapy has been discussed extensively in scientific and popular literature, but it may take a while to realize it.

References

About The Authors:

Alycea Wood is a research analyst at iData Research. She develops and composes syndicated research projects regarding the medical device industry, and published the U.S. Orthopedic Biomaterials report series.

Kamran Zamanian, Ph.D., is CEO and founding partner of iData Research. He has spent over 20 years working in the market research industry with a dedication to the study of medical devices used in the health of patients all over the globe.

About iData Research

For 16 years, iData Research has been a strong advocate for data-driven decision-making within the global medical device, dental, and pharmaceutical industries. By providing custom research and consulting solutions, iData empowers its clients to trust the source of data and make important strategic decisions with confidence.

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The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online

Bone Marrow Stem Cells Comments Off on The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market – Med Device Online | October 16th, 2021

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

T-Cell Immune Response to COVID-19 Vaccines and Natural Infections

Much of the discussions and news reports about immune responses to vaccines and COVID-19 revolve around antibody levels. Much less has been said about T-cells, which provide longer-term protection. Researchers atGladstone Institutesconducteda detailed T-cell survey before and after COVID-19 immunization, which they published ineLife. They concluded that the Pfizer-BioNTech and Moderna mRNA vaccines create long-term populations of T-cells that recognize multiple SARS-COV-2 virus variants. They also found key differences in the T-cell responses in people who had COVID-19 infections before vaccination compared to people who had never been infected.

Overall, our data support the idea that vaccines are eliciting a very robust T-cell response in healthy individuals, said Nadia Roan, senior author of the study and Gladstone Associate Investigator. But they also suggest there may be some ways to improve them further, by getting more of the vaccine-elicited T-cells to park themselves in the respiratory tract.

Antibodies produced by B-cells quickly recognize viruses, target them, and prevent infection by destroying the viruses. T-cells, however, identify and destroy cells that are already infected. Antibodies are better at stopping initial infection, but T-cells typically last longer after an initial infection or vaccine. At that point they are better at fighting off disease in its early stages, which prevents severe symptoms. But T-cells are very diverse and difficult to analyze. Some subsets respond differently to infected cells and behave differently, while others have different functions within the overall T-cell immune response.

One key finding was that in people who had not been previously infected, the T-cell responses become stronger in quantity and quality after the second dose of the vaccine. But in people who had previously had COVID-19, there was not much of a change between the first and second vaccine dose.

Blood Biomarkers Provide Warning Signs of Dementia

Investigators at theGerman Center for Neurodegenerative Diseases (DZNE)identifiedmolecules in the blood that potentially warn of impending dementia. The research study included several university hospitals across Germany. The biomarkers were based on measuring levels of microRNAs. They say that the technique isnt ready yet for practical use, but they hope to develop a simple blood test. MicroRNAs have regulatory properties, influencing protein production and metabolism. In tests in humans, mice and cell cultures, they found three microRNAs whose levels were linked to mental performance. The three microRNAs also influence neuro-inflammation and neuroplasticity, including the ability of neurons to establish connections with each other.

Stem Cell Population Essential for Bone Regeneration

Researchers at theUniversity of Tsukuba, Japan,identifieda subpopulation of mesenchymal stem cells that play a major role in bone healing. The stem cells are found in the bone marrow and express the marker CD73. When a bone fracture heals, it moves through a series of stages, including clotted blood forming at the fracture. This clot is replaced by fibrous tissue and cartilage, then by a hard bony callus. The bone is then remodeled, with regular bone replacing the hard callus. They found that the generation of the callus is critically dependent on recruiting MSCs from the surrounding tissue and bone marrow. They observed the CD73-positive MSCs migrating toward the fracture site and forming new cartilage and bone cells. When they grafted CD73-positive MSCs into the fracture, they noted enhanced healing processes.

Antiviral Molecule Prevents SARS-CoV-2 from Entering Cells

Scientists atWashington University School of Medicinein St. Louisdevelopeda compound that prevents the SARS-CoV-2 virus, which causes COVID-19, from entering cells. The compound is called MM3122 and has been studied in cell cultures and in mice. MM3122 targets a key human protein called transmembrane serine protease 2 (TMPRSS2), which coronaviruses use to enter and infect human cells. Once the virus attached onto a cell in the epithelia of the airway, the TMPRSS2 protein cuts the viral spike protein, which activates the spike protein to mediate fusion of the viral and cellular membranesstarting the infection process. In cell cultures, MM3122 protected cells from viral damage better than remdesivir,Gilead Sciences antiviral against COVID-19; and an acute safety assay in mice demonstrated that large doses of MM3122 given for seven days did not cause noticeable issues. The compound also was effective against SARS-CoV, the virus behind SARS, and MERS-CoV, the coronavirus that causes MERS. The researchers are now working with researchers at the NIH to test it in animal models of COVID-19. They are also working on an oral version of the injectable compound.

Specific Personality Traits Might Signal Pending Alzheimers

Researchers atFlorida State Universityfoundthat specific changes in the brain linked with Alzheimers disease are often visible earlier in people with personality traits associated with the disease. The research focused on two traits: neuroticism, or a predisposition for negative emotions, and conscientiousness, linked to a tendency to be careful, organized, goal-directed and responsible. They found that people with amyloid and tau deposits, proteins linked to Alzheimers disease in the brain, were identified in participants who scored higher in neuroticism levels and lower in conscientiousness. The study suggests that personality traits might help protect against Alzheimers and other brain diseases by delaying or preventing the neuropathology for people strong in conscientiousness and low in neuroticism.

Why We Overeat

Astudyfrom theUniversity of Washington School of Medicine/UW Medicinereported on the function of glutamatergic neurons in mice. These neurons communicate to the lateral habenula, a brain region associated with the pathophysiology of depression, and the ventral tegmental area, which is involved in motivation, reward and addiction. They found that when mice are eating, the neurons in the lateral habenula are more responsive than the neurons in the ventral tegmental area. They suggest that these neurons might play a bigger role in guiding feeding. In addition, they studied the influence of the leptin and ghrelin hormones, which are believed to regulate behavior via the mesolimbic dopamine system, part of the reward pathway. The research adds additional insight into satiety and why people or at least mice overeat.

We found these cells are not a monolithic group, and that different flavors of these cells do different things, said Garret Stuber, a joint UW professor of anesthesiology and pain medicine and pharmacology, the papers senior author.

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Research Roundup: T-Cell Immune Response to COVID-19 Vaccines and More - BioSpace

Bone Marrow Stem Cells Comments Off on Research Roundup: T-Cell Immune Response to COVID-19 Vaccines and More – BioSpace | October 16th, 2021

Nine accepted abstracts demonstrate broad potential of the companys HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

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Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress – Yahoo Finance | October 16th, 2021

TEL AVIV, Israel, Oct. 13, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company's view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin's lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient's own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient's stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, as well as reporting positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -...

Bone Marrow Stem Cells Comments Off on BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -… | October 16th, 2021

GRAND RAPIDS, Mich. As day turned to darkness, a crowd gathered at Monroe North on Saturday in Grand Rapids for the annual Light the Night celebration.

Its an inspirational community event, said Anne Bradley, Light the Night campaign development manager.

People eventually made their way to the street and showed support in each stride for blood cancer patients, their families and the countless others impacted by the disease.

Among the participants was Colette Smiley. Her white light gave a hint as to why she showed up.

I am a leukemia survivor, said Smiley. It completely alters your life.

In 2014, doctors diagnosed Smiley with Acute Myeloid Leukemia, or AML. Its an often fatal cancer that forms in a persons blood and bone marrow.

Smiley credits her treatment plan and a strangers stem cells for helping her enter remission within weeks of her diagnosis.

I come here to celebrate, to celebrate each re-birthday that I have, but to also thank LLS for [what] they do each and every day and each and every year for blood cancer patients, said Smiley.

According to the Leukemia and Lymphoma Society, blood cancers impact an estimated 1.5 million people in the United States, with a person diagnosed once every three minutes.

Leukemia is the most common cancer diagnosed in children and adolescents younger than 20 years old. It accounts for 25.1 percent of all cancer cases in the age group.

Symptoms of blood cancers often only appear in advanced stages, but can include ones similar to a severe cold or flu. Survival rates at one point hovered around 3 percent, but now, its up to 95 percent.

Its our goal and our mission to end blood cancer and to improve the quality of life for patients and their families, said Bradley.

All proceeds from Light the Night benefit LLS, which says 80 cents of every dollars goes directly to their mission.

Attendees, though, found a deeper value in the night.

We all live different stories, said Smiley. We all work in different places and our families are different and our backgrounds are different, but something brought us together that we certainly didnt wish for, blood cancer, but its brought us together and we can support each other now.

Light the Night hopes to collect $1 million in Michigan.

To donate, click here.

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Bone Marrow Stem Cells Comments Off on ‘Light the Night’ returns to Grand Rapids, raising awareness, donations for blood cancers – Fox17 | October 16th, 2021

NEW YORK, Oct. 14, 2021 /PRNewswire/ --BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, will present findings from a multicenter, open label clinical trial of NurOwn in progressive multiple sclerosis. The study, "Phase 2 Safety and Efficacy Study of Intrathecal MSC-NTF cells in Progressive Multiple Sclerosis," will be delivered in an oral presentation today at the fully digital37thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

The Phase 2 clinical trial was designed to evaluate intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS. The study achieved the primary endpoint of safety and tolerability. It demonstrated a reduction of neuroinflammatory biomarkers and an increase in neuroprotective biomarkers in the cerebrospinal fluid (CSF) and consistent improvement across MS functional outcome measures, including measures of walking, upper extremity function, vision and cognition.

"We were pleased that this study demonstrated safety, preliminary evidence of efficacy and relevant biomarker outcomes in patients with progressive multiple sclerosis, in an area of high unmet need," said Jeffrey Cohen, M.D., Director of Experimental Therapeutics at the Cleveland Clinic Mellen Center for MS and principal investigator for the trial. "These results should be confirmed in a randomized placebo-controlled trial."

The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses from the National Multiple Sclerosis Society Fast-Forward Program. It was conducted at four U.S. MS centers of excellence:

"We very much appreciate the tremendous collaboration among many premier organizations, for their generous sharing of expertise, support and data, which enabled the important balance between scientific rigor and ethical treatment of progressive MS participants in the trial," said Ralph Kern, M.D., MHSc., President and Chief Medical Officer, Brainstorm Cell Therapeutics. "We are holding discussions with key MS experts, and seeking guidance from the FDA to determine next steps for the development of NurOwn in progressive MS."

"The National MS Society is pleased to support the biomarker portion of this study through our commercial funding program Fast Forward," said Mark Allegretta, Ph.D., Vice President, Research. "We're encouraged to see evidence that the biomarker analysis showed proof of concept for detecting neuroprotection and reduced inflammation."

About the trial

The Phase 2 open-label studyevaluated the safety and efficacy of intrathecal administration of autologous MSC-NTF cells in patients with primary or secondary progressive MS. The primary study endpoint was safety and tolerability. Secondary efficacy endpoints included: timed 25-foot walk (T25FW); 9-Hole Peg Test (9-HPT); Low Contrast Letter Acuity (LCLA); Symbol Digit Modalities Test (SDMT); 12 item MS Walking Scale (MSWS-12); as well as cerebrospinal fluid (CSF) and blood biomarkers. Clinical efficacy outcomes were compared with matched (n=48) participants in the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry, Tanuja Chitnis, MD Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases, and 255 patient randomized double blind placebo controlled NN-102 SPRINT-MS Study, courtesy NIH/NINDS, PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic, CTR: NCT01982942. Baseline characteristics from these two cohorts were similar allowing for comparison of efficacy results, comparisons with SPRINT-MS were with the placebo arm of this study.

Mean age of participants was 47 years, 56% were female, and mean baseline EDSS score was 5.4. 18 participants were treated, 16 (80%) received all 3 treatments and completed the entire study; 2 study discontinuations were due to procedure-related adverse events. No deaths or treatment-related adverse events due to worsening of MS were observed.

In responder analyses, 14% and 13% of MSC-NTF treated participants showed at least a 25% improvement in T25FW and 9-HPT (combined hands) respectively, compared to 5% and 0% in matched CLIMB patients and 9% and 3% in SPRINT. Twenty-seven percent (27%) showed at least an 8-letter improvement in LCLA (binocular, 2.5% threshold) and 67% showed at least a 3-point improvement in SDMT, compared to 6% and 18% in CLIMB and 13% and 35% in SPRINT, respectively.

Mean improvements of +0.10 ft/sec in T25FW and -0.23 sec in 9-HPT (combined hands), were observed in MSC-NTF treated participants, compared to a mean worsening of -0.07 ft/sec and +0.49 sec in CLIMB and -0.06 ft/sec and +0.28 sec in SPRINT, respectively. MSC-NTF treated participants showed a mean improvement of +3.3 letters in LCLA (binocular, 2.5% threshold) and 3.8 points in SDMT, compared to a mean worsening of -1.07 letters in LCLA (binocular, 2.5% threshold) and mean improvement of +0.10 in SDMT, in CLIMB and -0.6 and -0.1 in SPRINT. In addition the MSFC-4 Composite Z-score of T25W, 9-HPT, SDMT and LCLA showed a 0.18 point improvement in MSC-NTF treated participants, while CLIMB and SPRINT showed decreases of -0.02 and -0.05.

Furthermore, 38% of treated patients showed at least a 10-point improvement in the MSWS-12 a patient reported outcome that evaluates the impact of MS on walking function, whereas this outcome was not evaluated in CLIMB or SPRINT.

CSF biomarkers obtained at 3 consecutive time points, showed increases in neuroprotective molecules (VEGF, HGF, NCAM-1,Follistatin, Fetuin-A) and decreases in neuroinflammatory biomarkers (MCP-1, SDF-1, sCD27 and Osteopontin).

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Investor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 (646) 791-9729egoldstein@lifesciadvisors.com

Media:Mariesa Kemble kemblem@mac.com

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Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in...

Bone Marrow Stem Cells Comments Off on Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in… | October 16th, 2021

The first step in any flawless makeup application is nailing your moisturizing routine (who wants makeup clinging to dry skin patches, #AmIRite?). And makeup artist Charlotte Tilburythe pro best known for making her celeb and model clients glow like they just stepped off the beaches in her hometown Ibizahas created a glowing skincare trifecta to prime and perfect skin 24/7, all but guaranteeing a *chefs kiss* makeup application.

The trio includes her iconic Magic Creamadored by celebrities and supermodels alike, thanks to star ingredients like peptides, hyaluronic acid plus antioxidants like vitamins C & E. With the Magic Night Cream, Tilbury turns up the glow while you sleep. Its a nourishing balm texture that boasts time-released retinol (famed to smooth lines), plant stem cells, plus CoEnzyme Q10 and vitamin E to ensure dewy, bouncy looking skin by morning. The final secret weapon of the trio is the Magic Eye Rescue: a smoothing, brightening eye cream packed with time-released retinol and plant stem cell extracts to reduce the appearance of wrinkles, dark circles and puffiness on tired-looking eyes. (Bonus: each is now available in refillable, recyclable glass jars).

To learn more on how to keep the glow going, we asked board-certified dermatologist Dr. Angela Lamb, who has teamed up with Charlotte Tilbury, how to nail your best day-and-night skincare routine for dewy results (including her pro advice on eye cream).

For the winter you want a product with humectants. These are ingredients that trap moisture and can fall into many categories. Some examples are glycerin, shea butter, and hyaluronic acid. They create a seal on the skin and attract water, which helps keep water close to the skin surface and prevents it from evaporating too quickly. The Charlotte Tilbury Magic Cream contains two of these key humectants, hyaluronic acid as well as aloe vera and shea butter, helping to hydrate skin for up to 24 hours and making it a perfect product for the winter.

The key ingredients for long-lasting improvement would be retinol. It builds collagen and decreases fine lines. To get that immediate boost, you want ingredients that provide hydration and reflect light, and give a refreshed look, like in the Charlotte Tilbury Magic Eye Rescue.

A retinol! It is the main ingredient that will provide what most people want: younger-looking eyes.

The time that you sleep allows the body to regenerate, repair cells and refresh. A great night cream will help your body accomplish these goals. The CoEnzyme Q10 in the Charlotte Tilbury Magic Night Cream does this well. As an antioxidant, it is able to actually help your skin repair some of the daily damage and regenerate for smooth, healthy-looking skin.

For product layering, I like moisturizer or serum first, then eye creams on top. I recommend layering a retinol on top of a moisturizer. You get the same benefits without any potential dryness.

The Kit created this content; Charlotte Tilbury funded and approved it.

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Skin Stem Cells Comments Off on Get Around-The-Clock Glowing Skin With Charlotte Tilbury Magic Cream – The Kit | October 16th, 2021

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, Inc., a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Tecentriq (atezolizumab) as adjuvant treatment following surgery and platinum-based chemotherapy for adults with Stage II-IIIA non-small cell lung cancer (NSCLC) whose tumors express PD-L11%, as determined by an FDA-approved test.

Tecentriq is now the first and only cancer immunotherapy available for adjuvant treatment of NSCLC, introducing a new era where people diagnosed with early lung cancer may have the opportunity to receive immunotherapy to increase their chances for cure, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Todays landmark approval gives physicians and patients a new way to treat early lung cancer that has the potential to significantly reduce risk of cancer recurrence, after more than a decade with limited treatment advances in this setting.

Too many patients with early-stage lung cancer experience disease recurrence following surgery. Now, the availability of immunotherapy following surgery and chemotherapy offers many patients new hope and a powerful new tool to reduce their risk of cancer relapse, said Bonnie Addario, Co-founder and Chair, GO2 Foundation for Lung Cancer. With this approval, it is more important than ever to screen for lung cancer early and test for PD-L1 at diagnosis to help bring this advance to the people who can benefit.

The approval is based on results from an interim analysis of the Phase III IMpower010 study that showed treatment with Tecentriq following surgery and platinum-based chemotherapy reduced the risk of disease recurrence or death by 34% (hazard ratio [HR]=0.66, 95% CI: 0.50-0.88) in people with Stage II-IIIA (UICC/AJCC 7th edition) NSCLC whose tumors express PD-L11%, compared with best supportive care (BSC). Safety data for Tecentriq were consistent with its known safety profile and no new safety signals were identified. Fatal and serious adverse reactions occurred in 1.8% and 18%, respectively, of patients receiving Tecentriq. The most frequent serious adverse reactions (>1%) were pneumonia (1.8%), pneumonitis (1.6%), and pyrexia (1.2%).

The review of this application was conducted under the FDAs Project Orbis initiative, which provides a framework for concurrent submission and review of oncology medicines among international partners. According to the FDA, collaboration among international regulators may allow patients with cancer to receive earlier access to products in other countries where there may be significant delays in regulatory submissions. Simultaneous applications were submitted to regulators in the United States, Switzerland, the United Kingdom, Canada, Brazil and Australia under Project Orbis. Additionally, the FDA reviewed and approved the supplemental application under its Real-Time Oncology Review pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

Tecentriq has previously shown clinically meaningful benefit in various types of lung cancer, with six currently approved indications in the U.S. In addition to becoming the first approved cancer immunotherapy for adjuvant NSCLC, Tecentriq was also the first approved cancer immunotherapy for front-line treatment of adults with extensive-stage small cell lung cancer (SCLC) in combination with carboplatin and etoposide (chemotherapy). Tecentriq also has four approved indications in advanced NSCLC as either a single agent or in combination with targeted therapies and/or chemotherapies. Tecentriq is available in three dosing options, providing the flexibility to choose administration every two, three or four weeks.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies across different lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines, as well as studies in metastatic, adjuvant and neoadjuvant settings across various tumor types.

About the IMpower010 study

IMpower010 is a Phase III, global, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq compared with BSC, in participants with Stage IB-IIIA NSCLC (UICC/AJCC 7th edition), following surgical resection and up to 4 cycles of adjuvant cisplatin-based chemotherapy. The study randomized 1,005 people with a ratio of 1:1 to receive either Tecentriq for 1 year (16 cycles), unless disease recurrence or unacceptable toxicity occurred, or BSC. The primary endpoint is investigator-determined DFS in the PD-L1-positive Stage II-IIIA, all randomized Stage II-IIIA and intent-to-treat (ITT) Stage IB-IIIA populations. Key secondary endpoints include overall survival (OS) in the overall study population, ITT Stage IB-IIIA NSCLC.

About lung cancer

According to the American Cancer Society, it is estimated that more than 235,000 Americans will be diagnosed with lung cancer in 2021. NSCLC accounts for 80-85% of all lung cancers and approximately 50% of patients diagnosed with NSCLC are diagnosed with early-stage (Stages I and II) or locally advanced (Stage III) disease. Today, about half of all people with early lung cancer still experience a cancer recurrence following surgery. Treating lung cancer early, before it has spread, may help prevent the disease from returning and provide people with the best opportunity for a cure.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues in any area of the body and can affect the way they work. These problems can sometimes become severe or life threatening and can lead to death. Patients can have more than one of these problems at the same time. These problems may happen anytime during their treatment or even after their treatment has ended.

Patients should call or see their healthcare provider right away if they develop any new or worse signs or symptoms, including:

Lung problems

Intestinal problems

Liver problems

Hormone gland problems

Kidney problems

Skin problems

Problems can also happen in other organs.

These are not all of the signs and symptoms of immune system problems that can happen with Tecentriq. Patients should call or see their healthcare provider right away for any new or worse signs or symptoms, including:

Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include:

Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if patients undergo transplantation either before or after being treated with Tecentriq. A healthcare provider will monitor for these complications.

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider will check patients for these problems during their treatment with Tecentriq. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may also need to delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information about the benefits and side effects of Tecentriq.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

Please see http://www.Tecentriq.com for full Prescribing Information and additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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FDA Approves Genentech's Tecentriq as Adjuvant Treatment for Certain People With Early Non-Small Cell Lung Cancer - BioSpace

Skin Stem Cells Comments Off on FDA Approves Genentech’s Tecentriq as Adjuvant Treatment for Certain People With Early Non-Small Cell Lung Cancer – BioSpace | October 16th, 2021

Skeletal muscle enables the body to move and maintain posture. Direct injury for instance, from trauma can impair a persons movement and quality of life.

People have been using massage and other mechanotherapies for thousands of years to soothe aching and injured muscles. However, the science behind the effects of massage has not been examined in detail.

Lots of people have been trying to study the beneficial effects of massage and other mechanotherapies on the body, but up to this point, it hadnt been done in a systematic, reproducible way, explains lead author of the current study Dr. Bo Ri Seo, Ph.D.

Our work shows a very clear connection between mechanical stimulation and immune function, he continues.

Like much of the human body, skeletal muscle can repair itself. The process involves three stages:

When muscle injury occurs, muscle fibers rupture and die. White blood cells invade the injury site, removing the dead muscle cells and activating cells that help mount an immune response. This includes the release of growth factors, cytokines, and chemokines.

During the repair phase, satellite cells, or muscle precursor cells, grow and differentiate into muscle cells. These then replace injured cells in the muscle fiber, and scar tissue forms.

In the remodeling phase, muscle fibers mature, and scar tissue contracts. However, extensive injuries may cause dense scar tissue formation, impeding muscle repair and resulting in incomplete recovery of muscle function.

Despite its complications, surgical treatment remains the current standard of care for severe muscle injury.

Recognizing the need for an effective noninvasive treatment for severe skeletal muscle injury, researchers from Harvard University in Cambridge, MA, conducted a study investigating the use of mechanotherapy as a potential treatment.

Their findings appear in the journal Science Translational Medicine.

Dr. Seo, who is a postdoctoral fellow at the Wyss Institute at Harvard, explained to Medical News Today:

Our previous study has shown the beneficial impacts of compressive loading for skeletal muscle regeneration. Based on the finding, we wanted to develop a [scientifically] validated, optimal protocol for mechanotherapy and to understand the mechanisms associated with the therapeutic impacts.

The researchers developed an external robotic device to deliver a precise, controlled, and measurable pressure to the leg muscle in mice. The scientists also used ultrasound to measure tissue response to the stress applied.

One to 14 days after injury, the scientists gave the mice in the treatment group mechanotherapy with pressure corresponding to muscle strains of 10, 20, or 40% for 5 minutes every 1012 hours. The scientists did not treat mice in the control group.

Compared with the control mice, the mice in the treatment group demonstrated a significant reduction in muscle fiber damage and scarring. The authors also note increased muscle fiber diameter, which is an indicator of repair and strength recovery.

Since muscle repair improvements were similar across the 10, 20, and 40% groups, the study continued using the 20% muscle strain pressure setting for the remaining experiments.

To uncover how mechanotherapy promoted muscle repair, the researchers also measured levels of inflammatory factors cytokines and chemokines over time.

The study identified that mechanotherapy reduced levels of a cytokine responsible for the movement of neutrophils by more than half by day 3. Neutrophils help clear damaged cells and communicate with other cells to promote repair and immune response. Neutrophils also play a role in inflammation.

To understand why neutrophils and cytokines were moving out of the muscle, the researchers injected a fluorescent compound into the muscle. They observed that mechanotherapy was directly causing this exodus from the muscle.

Next, the scientists cultured satellite cells which are essentially muscle stem cells with factors that neutrophils secrete. They wanted to assess their effects on muscle repair.

The study authors found that neutrophil-secreted factors initially promoted repair, but when they were present for a longer time, they impaired muscle fiber production.

After analyzing the muscle fibers produced in the two groups after 14 days, the researchers found that the leg muscle cells treated with mechanotherapy contained more type IIX fibers.

Type IIX fibers have a larger diameter and can produce increased force, consistent with the results the researchers saw in the mice that received mechanotherapy.

In the final experiment, the scientists used an antibody treatment to remove neutrophils in the mice during the first 3 days after injury. They found that the muscles of the treated mice recovered more quickly.

They discovered that both mechanotherapy and antibody treatment led to significantly reduced muscle damage and the development of larger muscle fibers.

Dr. Bert Mandelbaum, who was not involved in the study, also spoke with MNT. Dr. Mandelbaum is an orthopedic surgeon at Cedars-Sinai Kerlan-Jobe Institute and co-director of the Cedars-Sinai Regenerative Orthobiologics Center in Los Angeles.

He was intrigued by the experimental design particularly the use of robotics to prescribe specific muscle loads and then assessing the biological factors that the loads produce.

Speaking about the results, lead author Dr. Seo told MNT:

It was super exciting to see that the severely injured muscle treated with biologic-free/noninvasive compressive loading shows comparable functional outcomes to the ones treated with biologics-based therapies found from other studies.

Also, we were surprised by the fact that neutrophils were significantly involved in this process by directly influencing muscle progenitor cell activities, he continued.

In addition, we found that compressive loading rapidly reduces neutrophils and their associated factors by day 3 after injury with this change [] confined to the injured site. This makes mechanotherapy a great therapeutic candidate for patients who are already using other medical interventions or existing health complications for example, inflammatory diseases.

He went on to say: Our findings are based on [studies in] mice, so further studies are needed to confirm its impacts for larger animals and humans. Furthermore, since the kinetics and amplitudes of immune response can differ depending on types of injuries, how and what to be delivered should be optimized accordingly.

In conclusion, Dr. Mandelbaum told MNT, I think its a great hypothesis, something needing to be proven over time.

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Are 'robot massages' the future of muscle repair? - Medical News Today

Skin Stem Cells Comments Off on Are ‘robot massages’ the future of muscle repair? – Medical News Today | October 16th, 2021

KENILWORTH, N.J. & TOKYO--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted positive opinions recommending approval of the combination of KEYTRUDA, Mercks anti-PD-1 therapy, plus LENVIMA (marketed as KISPLYX in the European Union [EU] for the treatment of advanced renal cell carcinoma [RCC]), the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for two different indications. One positive opinion is for the first-line treatment of adult patients with advanced RCC, and the other is for the treatment of adult patients with advanced or recurrent endometrial carcinoma (EC) who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and are not candidates for curative surgery or radiation. Decisions on the CHMPs recommendations will be given by the European Commission for marketing authorization in the EU, and are expected in the fourth quarter of 2021. If approved, this would be the first combination of an anti-PD-1 therapy with a tyrosine kinase inhibitor approved for the treatment of two different types of cancer in the EU.

The positive CHMP opinions are based on data from two pivotal Phase 3 trials: CLEAR (Study 307)/KEYNOTE-581 evaluating the combination in adult patients with advanced RCC and KEYNOTE-775/Study 309 evaluating the combination in certain patients with advanced EC.

In CLEAR/KEYNOTE-581, KEYTRUDA plus LENVIMA demonstrated statistically significant improvements versus sunitinib in the efficacy outcome measures of overall survival (OS), reducing the risk of death by 34% (HR=0.66 [95% CI, 0.49-0.88]; p=0.0049) versus sunitinib, and progression-free survival (PFS), reducing the risk of disease progression or death by 61% (HR=0.39 [95% CI, 0.32-0.49]; p<0.0001) with a median PFS of 23.9 months versus 9.2 months for sunitinib. Additionally, the confirmed objective response rate was 71% (95% CI: 66-76) (n=252) for patients who received KEYTRUDA plus LENVIMA versus 36% with sunitinib (95% CI: 31-41) (n=129).

In KEYNOTE-775/Study 309, KEYTRUDA plus LENVIMA demonstrated statistically significant improvements in the studys dual efficacy outcome measures of OS, reducing the risk of death by 38% (HR=0.62 [95% CI, 0.51-0.75]; p<0.0001) with a median OS of 18.3 months versus 11.4 months for chemotherapy (investigators choice of doxorubicin or paclitaxel), and PFS, reducing the risk of disease progression or death by 44% (HR=0.56 [95% CI, 0.47-0.66]; p<0.0001), with a median PFS of 7.2 months versus 3.8 months for chemotherapy (investigators choice of doxorubicin or paclitaxel).

KEYTRUDA plus LENVIMA demonstrated a survival benefit for advanced renal cell carcinoma in the first-line setting and represents an important potential new treatment option for these patients. Additionally, KEYTRUDA plus LENVIMA is the first anti-PD-1 and tyrosine kinase inhibitor combination to demonstrate a survival benefit in advanced endometrial carcinoma patients, and the benefit was shown regardless of mismatch repair status, said Dr. Gregory Lubiniecki, Vice President, Clinical Research, Merck Research Laboratories. We are pleased that the CHMP has recognized the important role of the combination therapy in these difficult-to-treat cancers.

We appreciate the positive opinions rendered by the EU CHMP recommending approval of KEYTRUDA plus LENVIMA in advanced renal cell carcinoma and advanced endometrial carcinoma, underscoring the potential significance of the outcomes observed in the CLEAR/KEYNOTE-581 and KEYNOTE-775/Study 309 trials, said Dr. Takashi Owa, President, Oncology Business Group at Eisai. We are grateful to the patients who participated in these studies, their families and clinicians. Their commitment made these meaningful milestones possible.

The safety of KEYTRUDA in combination with axitinib or LENVIMA in advanced RCC, and in combination with LENVIMA in advanced EC has been evaluated in a total of 1,456 patients with advanced RCC or advanced EC. In these patient populations, the most frequent adverse reactions were diarrhea (58%), hypertension (54%), hypothyroidism (46%), fatigue (41%), decreased appetite and nausea (40% each), arthralgia (30%), vomiting, weight decreased, dysphonia and abdominal pain (28% each), proteinuria (27%), palmar plantar erythrodysesthesia syndrome and rash (26% each), stomatitis and constipation (25% each), musculoskeletal pain and headache (23% each) and cough (21%).

About Renal Cell Carcinoma (RCC)

Worldwide, it is estimated there were more than 431,000 new cases of kidney cancer diagnosed and more than 179,000 deaths from the disease in 2020. In Europe, it is estimated there were more than 138,000 new cases of kidney cancer diagnosed and more than 54,000 deaths from the disease in 2020. Renal cell carcinoma is by far the most common type of kidney cancer; about nine out of 10 kidney cancer diagnoses are RCC. Renal cell carcinoma is about twice as common in men as in women. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. Approximately 30% of patients with RCC will have metastatic disease at diagnosis. Survival is highly dependent on the stage at diagnosis, and the five-year survival rate is 13% for patients diagnosed with metastatic disease.

About Endometrial Cancer

Endometrial cancer begins in the inner lining of the uterus, which is known as the endometrium and is the most common type of cancer in the uterus. Worldwide, it was estimated there were more than 417,000 new cases and more than 97,000 deaths from uterine body cancers in 2020 (these estimates include both endometrial cancers and uterine sarcomas; more than 90% of uterine body cancers occur in the endometrium, so the actual numbers for endometrial cancer cases and deaths are slightly lower than these estimates). In Europe, it is estimated there were more than 130,000 new cases and more than 29,000 deaths in 2020. The five-year relative survival rate for metastatic endometrial cancer (stage IV) is estimated to be approximately 17%.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS 1)] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):

Non-muscle Invasive Bladder Cancer

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after 2 or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:

Cervical Cancer

KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).

KEYTRUDA, in combination with LENVIMA, is indicated for the first-line treatment of adult patients with advanced RCC.

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any settings and are not candidates for curative surgery or radiation.

Tumor Mutational Burden-High Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.

Triple-Negative Breast Cancer

KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the PD-1 or the PD-L1, blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. For patients with TNBC treated with KEYTRUDA in the neoadjuvant setting, monitor blood cortisol at baseline, prior to surgery, and as clinically indicated. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (20%) and increased aspartate aminotransferase (13%) were seen at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis, rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

See the rest here:
Merck and Eisai Receive Positive EU CHMP Opinions for KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) in Two Different Types of Cancer - Business...

Cardiac Stem Cells Comments Off on Merck and Eisai Receive Positive EU CHMP Opinions for KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) in Two Different Types of Cancer – Business… | October 16th, 2021

The Autologous Stem Cell Based Therapies market report delivers a complete analysis of critical aspects such as the predominant trends and growth opportunities that will assure considerable returns in the ensuing years. Also, it offers various solutions to tackle the present and upcoming challenges in the industry vertical. Besides, the document expounds the size and share of market segments including the product landscape, application spectrum, and geographical ambit. Furthermore, it discusses the after-effects of COVID-19 pandemic on this domain, uncovering the top revenue generating strategies for the approaching years.

Key pointers from case studies on COVID-19 impact:

Overview of the regional assessment:

Request Sample Copy of this Report @ https://www.nwdiamondnotes.com/request-sample/37424

Other vital inclusions in the Autologous Stem Cell Based Therapies market report:

Research Objective:

Why to Select This Report:

Key questions answered in the report:

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Autologous Stem Cell Based Therapies Market Consumption Status and Prospects Professional Market Research Report 2025 - Northwest Diamond Notes

Cardiac Stem Cells Comments Off on Autologous Stem Cell Based Therapies Market Consumption Status and Prospects Professional Market Research Report 2025 – Northwest Diamond Notes | October 16th, 2021

DUBLIN--(BUSINESS WIRE)--Horizon Therapeutics plc (Nasdaq: HZNP) today announced the publication of a post-hoc analysis from the N-MOmentum phase 2/3 pivotal trial of UPLIZNA, which highlights a sustained effect on attack risk with no new safety signals in people with NMOSD who received the treatment for four or more years. These data are published in the Multiple Sclerosis Journal.

NMOSD is a rare, severe autoimmune disease that attacks the optic nerve, spinal cord and brain stem. The attacks are often recurrent and can cause irreversible damage to the nerves, leading to cumulative visual and motor disabilities over time. UPLIZNA is the first and only FDA-approved anti-CD19 B-cell-depleting humanized monoclonal antibody for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive NMOSD.

This long-term study is important because NMOSD is a chronic disease that requires lifelong management. Physicians need to understand the implications of prolonged treatment, said Bruce Cree, M.D., Ph.D., MAS, professor of clinical neurology at the University of California San Francisco Weill Institute for Neurosciences and primary study investigator. It is highly encouraging to see that most patients in this study were attack-free after the first year of UPLIZNA treatment and that new safety concerns were not observed. The data demonstrate that long-term UPLIZNA use is associated with a reduced risk of NMOSD attacks possibly due to the depth and extent of B-cell depletion with repeated doses.

The post-hoc analysis represents the experience of 75 people with AQP4 antibody positive NMOSD who were treated with UPLIZNA for four or more years during the open-label extension period of the N-MOmentum trial.

Key study findings include the following:

NMOSD is a complex and often unpredictable B-cell-mediated disease that presents significant challenges to both patients and physicians, said Kristina Patterson, M.D., Ph.D., medical director, neuroimmunology, Horizon. With recent treatment advancements, the NMOSD community now has more options than ever before including UPLIZNA, which is engineered for broad, deep and durable B-cell depletion. We are fully committed to increasing our understanding of this disease so we can continue to improve patient care.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.1,2 Approximately 80 percent of all patients with NMOSD test positive for anti-AQP4 antibodies.3 AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.4

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.5 Depletion of these CD19+ B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.4,6 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.7 Each NMOSD attack can lead to further cumulative damage and disability.8,9 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.10,11

About UPLIZNA

INDICATION

UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%) and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of hepatitis B virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation. Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see Prescribing Information at http://www.UPLIZNA.com.

About Horizon

Horizon is focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit http://www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the potential benefits of UPLIZNA and Horizons research and development plans. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include, but are not limited to, risks regarding whether future results of clinical trials will be consistent with preliminary results or results of prior trials or other data or Horizons expectations, the risks associated with clinical development and adoption of novel medicines and risks related to competition or other factors that may change physician treatment strategies. For a further description of these and other risks facing Horizon, please see the risk factors described in Horizons filings with the United States Securities and Exchange Commission, including those factors discussed under the caption Risk Factors in those filings. Forward-looking statements speak only as of the date of this press release and Horizon undertakes no obligation to update or revise these statements, except as may be required by law.

References

Go here to read the rest:
New Analysis Published in Multiple Sclerosis Journal Assesses Long-Term Use of UPLIZNA (inebilizumab-cdon) for the Treatment of Neuromyelitis Optica...

Spinal Cord Stem Cells Comments Off on New Analysis Published in Multiple Sclerosis Journal Assesses Long-Term Use of UPLIZNA (inebilizumab-cdon) for the Treatment of Neuromyelitis Optica… | October 5th, 2021

DUBLIN - Horizon Therapeutics plc (Nasdaq: HZNP) announced the publication of a post-hoc analysis from the N-MOmentum phase 2/3 pivotal trial of UPLIZNA, which highlights a sustained effect on attack risk with no new safety signals in people with NMOSD who received the treatment for four or more years. These data are published in the Multiple Sclerosis Journal.

NMOSD is a rare, severe autoimmune disease that attacks the optic nerve, spinal cord and brain stem. The attacks are often recurrent and can cause irreversible damage to the nerves, leading to cumulative visual and motor disabilities over time. UPLIZNA is the first and only FDA-approved anti-CD19 B-cell-depleting humanized monoclonal antibody for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive NMOSD.

'This long-term study is important because NMOSD is a chronic disease that requires lifelong management. Physicians need to understand the implications of prolonged treatment,' said Bruce Cree, M.D., Ph.D., MAS, professor of clinical neurology at the University of California San Francisco Weill Institute for Neurosciences and primary study investigator. 'It is highly encouraging to see that most patients in this study were attack-free after the first year of UPLIZNA treatment and that new safety concerns were not observed. The data demonstrate that long-term UPLIZNA use is associated with a reduced risk of NMOSD attacks - possibly due to the depth and extent of B-cell depletion with repeated doses.'

The post-hoc analysis represents the experience of 75 people with AQP4 antibody positive NMOSD who were treated with UPLIZNA for four or more years during the open-label extension period of the N-MOmentum trial.

Key study findings include the following:

A total of 18 attacks occurred in 13 people, with an annualized attack rate of 0.052 attacks per person year.

The small number of total attacks decreased significantly after the first year of treatment with UPLIZNA.

67% of attacks occurred within the first year (12 attacks).

92% of patients were attack-free in subsequent years (two attacks each during years two to four).

The infection rate did not increase over time on treatment with UPLIZNA.

UPLIZNA was generally well tolerated, with few treatment-related dose interruptions and no treatment discontinuations.

'NMOSD is a complex and often unpredictable B-cell-mediated disease that presents significant challenges to both patients and physicians,' said Kristina Patterson, M.D., Ph.D., medical director, neuroimmunology, Horizon. 'With recent treatment advancements, the NMOSD community now has more options than ever before - including UPLIZNA, which is engineered for broad, deep and durable B-cell depletion. We are fully committed to increasing our understanding of this disease so we can continue to improve patient care.'

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.1,2 Approximately 80 percent of all patients with NMOSD test positive for anti-AQP4 antibodies.3 AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.4

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.5 Depletion of these CD19+ B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.4,6 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.7 Each NMOSD attack can lead to further cumulative damage and disability.8,9 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.10,11

About UPLIZNA

INDICATION

UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

A history of life-threatening infusion reaction to UPLIZNA

Active hepatitis B infection

Active or untreated latent tuberculosis

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%) and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of hepatitis B virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation. Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see Prescribing Information at http://www.UPLIZNA.com.

About Horizon

Horizon is focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit http://www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the potential benefits of UPLIZNA and Horizon's research and development plans. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include, but are not limited to, risks regarding whether future results of clinical trials will be consistent with preliminary results or results of prior trials or other data or Horizon's expectations, the risks associated with clinical development and adoption of novel medicines and risks related to competition or other factors that may change physician treatment strategies. For a further description of these and other risks facing Horizon, please see the risk factors described in Horizon's filings with the United States Securities and Exchange Commission, including those factors discussed under the caption 'Risk Factors' in those filings. Forward-looking statements speak only as of the date of this press release and Horizon undertakes no obligation to update or revise these statements, except as may be required by law.

Contact:

Rachel Vann

Director

Product Communications

E: media@horizontherapeutics.com

Original post:
Horizon Therapeutics Public : plc - New Analysis Published in Multiple Sclerosis Journal Assesses Long-Term Use of UPLIZNA (inebilizumab-cdon) for the...

Spinal Cord Stem Cells Comments Off on Horizon Therapeutics Public : plc – New Analysis Published in Multiple Sclerosis Journal Assesses Long-Term Use of UPLIZNA (inebilizumab-cdon) for the… | October 5th, 2021

Researchers have been looking for something that can help the body heal itself. Although studies are ongoing, stem cell research brings this notion of regenerative medicine a step closer. However, many of its ideas and concepts remain controversial. So, what are stem cells, and why are they so important?

Stem cells are cells that can develop into other types of cells. For example, they can become muscle or brain cells. They can also renew themselves by dividing, even after they have been inactive for a long time.

Stem cell research is helping scientists understand how an organism develops from a single cell and how healthy cells could be useful in replacing cells that are not working correctly in people and animals.

Researchers are now studying stem cells to see if they could help treat a variety of conditions that impact different body systems and parts.

This article looks at types of stem cells, their potential uses, and some ethical concerns about their use.

The human body requires many different types of cells to function, but it does not produce every cell type fully formed and ready to use.

Scientists call a stem cell an undifferentiated cell because it can become any cell. In contrast, a blood cell, for example, is a differentiated cell because it has already formed into a specific kind of cell.

The sections below look at some types of stem cells in more detail.

Scientists extract embryonic stem cells from unused embryos left over from in vitro fertilization procedures. They do this by taking the cells from the embryos at the blastocyst stage, which is the phase in development before the embryo implants in the uterus.

These cells are undifferentiated cells that divide and replicate. However, they are also able to differentiate into specific types of cells.

There are two main types of adult stem cells: those in developed bodily tissues and induced pluripotent stem (iPS) cells.

Developed bodily tissues such as organs, muscles, skin, and bone include some stem cells. These cells can typically become differentiated cells based on where they exist. For example, a brain stem cell can only become a brain cell.

On the other hand, scientists manipulate iPS cells to make them behave more like embryonic stem cells for use in regenerative medicine. After collecting the stem cells, scientists usually store them in liquid nitrogen for future use. However, researchers have not yet been able to turn these cells into any kind of bodily cell.

Scientists are researching how to use stem cells to regenerate or treat the human body.

The list of conditions that stem cell therapy could help treat may be endless. Among other things, it could include conditions such as Alzheimers disease, heart disease, diabetes, and rheumatoid arthritis. Doctors may also be able to use stem cells to treat injuries in the spinal cord or other parts of the body.

They may do this in several ways, including the following.

In some tissues, stem cells play an essential role in regeneration, as they can divide easily to replace dead cells. Scientists believe that knowing how stem cells work can help treat damaged tissue.

For instance, if someones heart contains damaged tissue, doctors might be able to stimulate healthy tissue to grow by transplanting laboratory-grown stem cells into the persons heart. This could cause the heart tissue to renew itself.

One study suggested that people with heart failure showed some improvement 2 years after a single-dose administration of stem cell therapy. However, the effect of stem cell therapy on the heart is still not fully clear, and research is still ongoing.

Another investigation suggested that stem cell therapies could be the basis of personalized diabetes treatment. In mice and laboratory-grown cultures, researchers successfully produced insulin-secreting cells from stem cells derived from the skin of people with type 1 diabetes.

Study author Jeffrey R. Millman an assistant professor of medicine and biomedical engineering at the Washington University School of Medicine in St. Louis, MO said, What were envisioning is an outpatient procedure in which some sort of device filled with the cells would be placed just beneath the skin.

Millman hopes that these stem cell-derived beta cells could be ready for research in humans within 35 years.

Stem cells could also have vast potential in developing other new therapies.

Another way that scientists could use stem cells is in developing and testing new drugs.

The type of stem cell that scientists commonly use for this purpose is the iPS cell. These are cells that have already undergone differentiation but which scientists have genetically reprogrammed using genetic manipulation, sometimes using viruses.

In theory, this allows iPS cells to divide and become any cell. In this way, they could act like undifferentiated stem cells.

For example, scientists want to grow differentiated cells from iPS cells to resemble cancer cells and use them to test anticancer drugs. This could be possible because conditions such as cancer, as well as some congenital disabilities, happen because cells divide abnormally.

However, more research is taking place to determine whether or not scientists really can turn iPS cells into any kind of differentiated cell and how they can use this process to help treat these conditions.

In recent years, clinics have opened that offer different types of stem cell treatments. One 2016 study counted 570 of these clinics in the United States alone. They appear to offer stem cell-based therapies for conditions ranging from sports injuries to cancer.

However, most stem cell therapies are still theoretical rather than evidence-based. For example, researchers are studying how to use stem cells from amniotic fluid which experts can save after an amniocentesis test to treat various conditions.

The Food and Drug Administration (FDA) does allow clinics to inject people with their own stem cells as long as the cells are intended to perform only their normal function.

Aside from that, however, the FDA has only approved the use of blood-forming stem cells known as hematopoietic progenitor cells. Doctors derive these from umbilical cord blood and use them to treat conditions that affect the production of blood. Currently, for example, a doctor can preserve blood from an umbilical cord after a babys birth to save for this purpose in the future.

The FDA lists specific approved stem cell products, such as cord blood, and the medical facilities that use them on its website. It also warns people to be wary of undergoing any unproven treatments because very few stem cell treatments have actually reached the earliest phase of a clinical trial.

Historically, the use of stem cells in medical research has been controversial. This is because when the therapeutic use of stem cells first came to the publics attention in the late 1990s, scientists were only deriving human stem cells from embryos.

Many people disagree with using human embryonic cells for medical research because extracting them means destroying the embryo. This creates complex issues, as people have different beliefs about what constitutes the start of human life.

For some people, life starts when a baby is born, while for others, it starts when an embryo develops into a fetus. Meanwhile, other people believe that human life begins at conception, so an embryo has the same moral status and rights as a human child.

Former U.S. president George W. Bush had strong antiabortion views. He believed that an embryo should be considered a life and not be used for scientific experiments. Bush banned government funding for human stem cell research in 2001, but former U.S. president Barack Obama then revoked this order. Former U.S. president Donald Trump and current U.S. president Joe Biden have also gone back and forth with legislation on this.

However, by 2006, researchers had already started using iPS cells. Scientists do not derive these stem cells from embryonic stem cells. As a result, this technique does not have the same ethical concerns. With this and other recent advances in stem cell technology, attitudes toward stem cell research are slowly beginning to change.

However, other concerns related to using iPS cells still exist. This includes ensuring that donors of biological material give proper consent to have iPS cells extracted and carefully designing any clinical studies.

Researchers also have some concerns that manipulating these cells as part of stem cell therapy could lead to the growth of cancerous tumors.

Although scientists need to do much more research before stem cell therapies can become part of regular medical practice, the science around stem cells is developing all the time.

Scientists still conduct embryonic stem cell research, but research into iPS cells could help reduce some of the ethical concerns around regenerative medicine. This could lead to much more personalized treatment for many conditions and the ability to regenerate parts of the human body.

Learn more about stem cells, where they come from, and their possible uses here.

Read the original:
Stem cells: Therapy, controversy, and research

IPS Cell Therapy Comments Off on Stem cells: Therapy, controversy, and research | October 5th, 2021

One of the most common questions Ive gotten over the last decade is, how much does stem cell therapy cost? They actually seem most often to want to know more specifically how much itshould cost.

To try to authoritatively answer this now in 2021 we need data from the present and past along with expert perspectives.

These kinds of questions on what are common and reasonable prices have continued in 2021. However, the types of queries have also evolved as things have gotten more complicated. There are many layers to the question of cost, which I cover here in todays article. In the big picture, the most worrisome potential cost is to your health if you proceed with unproven stem cell injections.

Stem cell cost questions | Stem cells cost $2,500 to $20,000| Why do stem cells cost so much? | How have stem cell prices changed? | Stem cell supplement cost | FTC actions and patients as consumers | Does insurance or Medicare cover stem cell therapy? | Patient fundraising | Looking ahead will stem cell costs go down?| References

This post is the most comprehensive look at stem cell treatment cost and costs of related therapies that Ive seen on the web, especially factoring in our inclusion of historical polling data from past years here on The Niche. The above bullet point list is what is covered in todays post and you can jump to sections that interest you most by clicking on those table of contents bullet points.

You can also watch the video I made summarizing the key points of this post below.

Furthermore, it encompasses other important issues related to insurance, fundraising, and approaches to being a smart consumer. Keep in mind that almost all stem cell therapies outside the bone marrow/hematopoietic sphere are not FDA-approved. They mostly lack rigorous data to back them up too. So this post is definitely not recommending you get them. I advise against it, but many people still want info on cost.

Lets get started.

After more than a decade of blogging about stem cells from just about every angle, its interesting to consider trends in the types of questions I get asked. Beyond cost, I also often get asked How much of a stem cell treatment price does insurance cover?

Of course, insurance (or lack thereof) directly bears on cost too. Ill get more into insurance later in the post.

In a way its not so surprising that cost is so much on peoples minds now for a few reasons.

First, as compared to many years back, people now view stem cell injections as a more everyday thing. Stem cell therapy is often available just down the street at a local strip mall.

Back in 2010 and in the 5 or so years after that, people instead more often viewed stem cells as some amazing thing out of reach to them at that time. Now people view stem cell offerings through the lens of consumers.

Sadly, another major part of the reason for the change in perceptions of stem cell treatments is the tidal wave of stem cell clinics from coast to coast in the US selling unproven and sometimes dangerous offerings.

At the same time, some universities and large medical centers also sell stem cell or similar offerings that arent proven. Im worried that that number may be increasing too and patients who may be paying there for unproven stem cells way at the very high end of the cost spectrum, sometimes above $100K.

Other stem cell suppliers and clinics market stem cell-related stuff that isnt real stem cells such as platelet rich plasma or PRP (see my comprehensive guide to PRP including a helpful infographic here) or injections of often dead perinatal stem cell products.

For all these reasons about once every year or two, I do polling asking the readers of The Niche here about their experiences.

Ive done the polling again now in 2020 in a more comprehensive form.

To have a sense of cost, we need to ask patients certain questions. How much did you pay per injection? How many injections did you get? Where did you get them?

Keep in mind that the total cost of stem cell therapy is the product of the cost per injection times the # of injections. For instance, if a stem cell injection costs $8,000 and you get 10 injections, your total cost is $80,000.

Unfortunately, the unproven stem cell clinics generally do not volunteer data on how much they charge. They also often encourage patients to get many injections.

Our 2020 polling data (you can still participate and I will update this) for stem cell treatments are in the graphic above. Here are some highlights.

The self-reported responses on cost for stem cell treatments, as indicated by respondents to our 2020 polling, suggest the price has gone up.

While the most common answer in 2019 was $2,501-$5,000, in 2020 the most common response was $10,001-$20,000, while $2,501-$5,000 was close behind.

The percentage of people paying the most, more than $100,000, was only slightly (probably non-significantly) higher in 2020, but both in 2019 and 2020 the percentage of people paying over $100K was much higher than in 2018 polling.

Keep in mind this is the cost per injection so how many injections do patients typically get? While the number of injections reported most commonly was 1 in both 2019 and 2020, in 2020, the second most common answer was 6-10 injections, a big boost from 2019. Again, more injections end up multiplying things up to boost the total cost. Only a few people in the polling had many injections, but in my view it is still striking to see anyone say theyve received more than 20 stem cell injections.

For comparison, the 2019 polling can be found here, but some of the key results are captured in a combo screenshot Ive included here. I got a lot more responses to the polling in 2019 so that makes me more confident in the data than in the 2020 polling so far, but I hope well get more responses moving forward in 2020 and if we do, again Ill update the info in this post.

What you can see from 2019 is that a plurality of respondents reported getting one stem cell injection, but 60% of people nonetheless got more than one stem cell injection.

Remarkably about 1 in 20-25 people received more than 20 stem cell injections.

About another 1 in 20 people got 6-20 injections. I find this amount of repeat injections to be surprising and concerning as it amplifies health and financial risks.

In terms of cost per injection, the results are pretty similar to 2018 (see at right below) on the whole.

This kind of polling isnt super scientific, but can gauge trends. Unfortunately, I havent really seen much other published data on stem cell clinic costs in actual journals.

I dont know if its noise or not, but the percentage of people paying over $100K is about 2-fold higher in 2019 versus 2018.

There are more people may be paying $10K-$20K as well now in 2020 vs. 2019 or 2018.

There is growing interest from the public in stem cell supplements. I did a post on this earlier in 2020 so take a look here, which was essentially a review of stem cell supplements like Regenokine. In terms of cost, while supplements are far less expensive than getting stem cell, PRP, or exosome injections, supplements are still pricey for what you get. Its not unusual to pay $100 for a small bottle of stem cell supplements, the other factor to consider is that these supplements generally have no solid, published data behind them so you might as well be paying $100 for water. Its unclear what risks taking these supplements might bring as well.

On the economic side, you might think that the feds like the FTC would be actively pursuing false or even fraudulent marketing of stem cells via the web and other kinds of advertising, but in total so far the FTC to my knowledge has only taken relatively few actions such as this one. and then some letters for COVID-related marketing of stem cells and other biologics earlier this year in 2020.

Oddly, there were just that a couple blips of FTC activity, especially considering the sea of questionable stem cell clinic-related ads out there. This ranges from major newspapers to inflight magazines to mobile ads on a stem-cell-mobile to television. Then of course there are the infomercial seminars.

Patients should also view themselves as consumers. Savvy customers considering paying money to stem cell clinics should do their homework. I often tell patients to use at a minimum the kinds of tough standards they bring to the car-buying process. Over the last few years Consumer Reports has been interested in the stem cell treatment world and done some reporting that is worth reading.

A common question I hear is the following: is stem cell therapy covered by insurance? Unfortunately for patients desperate to try stem cells, insurance generally does not provide any coverage, which often leads them to take extreme financial measures. These steps can include fundraising (more below).

In my view, the Regenexx brand has made a big deal out of how some employers contribute towards costs of their clinics offerings. Im not so clear on where that stands today in 2020.

Does Medicare cover stem cell therapies? Medicare will generally cover the cost of established bone marrow transplantation type therapies. However it does not cover unproven stem cell therapies.

Patients are often reaching out to me so I know that many of them have gone to extraordinary measures to raise the money to pay to unproven stem cell clinics. Its painful to think about what little they get in return. Since we are by definition talking about unproven medical procedures here, in my view this money is largely down the drain.

If you have other data on stem cell economic issues such as what patients pay please let me know. Then theres the issue of what it actually costs the clinics per injection and in turn: whats their profit margin?

What ends up happening is that patients take out second mortgages on their houses, try to collect funds from friends and relatives, or turn to online fundraising. The internet fundraising efforts most often end up on GoFundMe. This is a trend Ive been noticing for years. Some colleagues even published a paper on this trend, a very interesting and an important read. The paper is Crowdfunding for Unproven Stem CellBased Interventions in JAMA by Jeremy Snyder,Leigh Turner , and Valorie A. Crooks. Heres a key passage:

As of December 3, 2017, our search identified 408 campaigns (GoFundMe=358; YouCaring=50) seeking donations for stem cell interventions advertised by 50 individual businesses. These campaigns requested $7439308 and received pledges for $1450011 from 13050 donors. The campaigns were shared 111044 times on social media. Two campaigns were duplicated across platforms but shared separately on social media. Of the 408 campaigns, 178 (43.6%) made statements that were definitive or certain about the interventions efficacy, 124 (30.4%) made statements optimistic or hopeful about efficacy, 63 (15.4%) made statements of both kinds, and 43 (10.5%) did not make efficacy claims. All mentions of risks (n=36) claimed the intervention had low/no risks compared with alternative treatments.

Supposedly GoFundMe has taken some steps to lower the often ethically thorny stem cell fundraising on its site, but Im not sure how much it has changed.

There is pressure on stem cell clinics now in 2021 in large part due to two factors. These could drive costs down or up depending on how things play out. First, the FDA is much more active against unproven stem cell clinics. This may mean more money from the clinics going toward paying attorneys or FDA compliance experts. Youd think this might drive costs up. However, the still large number of clinics may keep pressure to stay with keeping price tags lower.

The second factor is the COVID-19 pandemic, which has forced many clinics to stop injections temporarily. While a surprising number of clinicsI did by phone were still open in a small informal survey, others were in a holding pattern. This may lower supply which could raise prices. But I think demand is likely way down as many patients stay home to avoid COVID risks. This could be temporary though. As things start re-opening, as they are now, the clinics may be able to capitalize on pent-up demand.

To sum up, the answer to the question, How do stem cells cost? is largely driven by clinic firms aiming to profit. Overall, clinics will charge what they think patients will pay them, which will always be a moving target. I urge patients to be cautious both medically, talking to their doctors, and financially.

Link:
How much does stem cell therapy cost in 2021? - The Niche

IPS Cell Therapy Comments Off on How much does stem cell therapy cost in 2021? – The Niche | October 5th, 2021

In conversation with Dr Koji Tanabe, Founder and CEO, I Peace, Inc., The United States/Japan

To make the trial investments more meaningful and to avoid ambivalence in animal models, medical science is adopting novel in vitro models of specialised human pluripotent cell lines. Pluripotent stem cells(PSCs) have the agility to expand indefinitely and differentiate into almost any organ-specific cell type. iPSC-derived organs andorganoidsare currently being evaluated in multiple medical research arena like drug development, toxicity testing, drug screening, drug repurposing, regenerative therapies, transgenic studies, disease modeling and more across clinical developments. Innovative pharmacovigilance methodologies are preferring induced pluripotent stem cells (iPSCs) for pre-clinical and clinical investigational studies. Global Induced Pluripotent Stem Cell (iPSC) market is expected to reach $2.3 B by 2026. The iPSC market inAsia-Pacificis estimated to witness fast growth due to increasing R&D projects across countries likeAustralia,JapanandSingapore.

I Peace, Inc. a Palo Alto-based global biotech company with its manufacturing base in Japan, has succeeded in developing and mass-producing clinical grade iPS cells through its proprietary iPS cell manufacturing services. The human iPSC (hiPSC) lines at I Peace leverage differentiated cells across clinical research and medical applications. Biopsectrum Asia discovered more about Japan's stem cell manufacturing ecosystem with Dr Koji Tanabe, Founder and CEO, I Peace, Inc., (The United States/Japan). Tanabe earned his doctorate under Dr Shinya Yamanaka, a Kyoto University researcher who received the 2012 Nobel Prize in Physiology or Medicine for discovery of reprogramming adult somatic cells to pluripotent cells. I Peace is focusing on this Nobel Prize-winning iPSCs technology where Tanabe had played a key role in generating the worlds first successful human iPSCs as one of the team members and is currently industrialising it in the US and Japan.

How do you define Japans Stem cell manufacturing dynamics aligning with regional and APAC market potential?

We believe that human cells play a pivotal role in next-generation drug therapy. Clinical trials of iPSC applications are in full swing not only in Japan, but worldwide as well. In the US, the momentum of clinical trial research is astounding. Yet, mass production of GMP compliant cell products remains a challenge. Entry into this venture is no easy task. As a contract development and manufacturing organisation (CDMO), I Peace is geared to tackle that challenge and become the pioneer of mass production technology of clinical grade cell products.

Can you elaborate I Peaces cost-effective proprietary stem cell synthesis solution and its manufacturing scale?

The key advantage of iPSCs is the ability to create pluripotent cells from an individuals own cells. Furthermore, iPSCs can multiply indefinitely and evolve into any type of cell, making iPSCs an ideal tool for transplant and regenerative medicine and drug research. However, clinical applications of iPSCs to date, utilise heterogenic transplantation. It is because manufacturing of just one line of iPSCs requires a cost intensive clean room to be occupied for several months. Manufacturing process complexities also pose a barrier to cost reduction and mass production.

In contrast, I Peace has developed a proprietary, fully automated closed system for iPS manufacturing, enabling cost-effective production of multiple lines of iPSCs from multiple donors in a single room. Within a few years, we expect to manufacture several thousand lines of iPSCs simultaneously in a single room. With this technology, I Peace can efficiently generate an ample supply of various iPSCs for heterogenic transplant, while also fostering a society where everyone can bank their own iPSCs for potential medical use.

How does I-Peace better position its businesses objectives and go-to-market strategies?

I Peaces manufacturing facility and its processes have undergone rigorous audits and are certified to be in compliance with GMP guidelines of the US, Japan, and Europe. We have the capacity to manufacture clinical-grade iPSCs and iPSC-derived cells for clinical use in the global market. Our manufacturing staff have unparalleled expertise in the manufacturing of iPSCs, and their knowledge and experience make it possible to mass produce high quality clinical-grade iPSCs in the shortest possible time. Additionally, we streamlined the iPSC use licensing scheme to expedite collaborative ventures with downstream partners. We believe these strategies position I Peace as a global leader in iPSC technology.

How do you outline the concept of democratising access to iPSC manufacturing?

At I Peace, we envision a world in which everyone would possess their own iPSCs and if needed, receive autologous transplant medication using their own iPSC. We believe in the importance of raising awareness of Nobel Prize winning iPSC technology and we think much more needs to be done. We need to enlighten the public about iPSCs - what they are, how they are created, and how they play a role in next-generation medical therapies. We also need to underscore the benefits of early banking ones own iPSCs, such as autologous transplant and the fact that cells taken in the early stages of life are preferable over cells collected later in life.

To democratise iPSC access, it is also important to expedite application research. We work closely with downstream partners, and support their iPSC-derived drug therapy development efforts by providing iPSCs to meet their needs. We also collaborate with downstream partners in the development of promising therapies including the use of T-cells for cancer therapy, cardiomyocytes for the treatment of heart disease, and neurocytes for neurological disease.

What is your outlook around boosting public-private stakeholders initiatives to encourage awareness on stem-cell-based therapeutics?

iPSC research has advanced tremendously over the past 16 years, and even more so since Dr Shinya Yamanakas Nobel Prize award in 2012. The acceleration of applied research is paving the way for stem cell-based therapeutics to become a common treatment modality in the near future. As human cell manufacturing requires specialised professional skills and knowledge, it is important to promote functional specialisation. These specialisations include donor recruiting, cell manufacturing (where I Peace is the key player), and implementing cell transplant as a medical practice. We believe that creating a systematic industry structure will build awareness and further drive the growth of stem cell-based therapy.

Can you brief Japans licensing key notes to manufacture and process clinical-grade cells in the region?

Japan enacted three laws to promote the use of regenerative medicine as a national policy:

1) The Regenerative Medicine Promotion Act -- representing the country's determination to promote regenerative medicine;

2) The Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act (PMD Act); and

3) The Act on the Safety of Regenerative Medicine (RM Act). The U.S. also has various tracks such as the Regenerative Medicine Advanced Therapy (RMAT) Designation, Breakthrough Therapy designation, and Fast Track designation.

Of significance, the PMD Act enables a fast-track for regulatory approval of regenerative medicalproducts in Japan. In compliance with the RM Act, I Peace was audited by the PMDA and licensed by the Ministry of Health, Labour, and Welfare to manufacture specific cell products.

Because cell product manufacturing regulations are not standardised globally, cell therapy developers are forced to source GMP iPSCs for each market. I Peace however, has overcome this hurdle. We have built in compliance with global GMP regulations, including FDA's cGMP regulations per 21 CFR 210/211 in our operation. As a result, we can provide cells for global use in multiple markets, accelerating both product development and regulatory approval.

Hithaishi C Bhaskar

hithaishi.cb@mmactiv.com

See original here:
"Stem cell-based therapeutics poised to become mainstream option - BSA bureau

IPS Cell Therapy Comments Off on "Stem cell-based therapeutics poised to become mainstream option – BSA bureau | October 5th, 2021

Every person, every mouse, every dog, has one unmistakable sign of aging: hair loss. But why does that happen?

Rui Yi, a professor of pathology at Northwestern University, set out to answer the question.

A generally accepted hypothesis about stem cells says they replenish tissues and organs, including hair, but they will eventually be exhausted and then die in place. This process is seen as an integral part of aging.

Instead Dr. Yi and his colleagues made a surprising discovery that, at least in the hair of aging animals, stem cells escape from the structures that house them.

Its a new way of thinking about aging, said Dr. Cheng-Ming Chuong, a skin cell researcher and professor of pathology at the University of Southern California, who was not involved in Dr. Yis study, which was published on Monday in the journal Nature Aging.

The study also identifies two genes involved in the aging of hair, opening up new possibilities for stopping the process by preventing stem cells from escaping.

Charles K.F. Chan, a stem cell researcher at Stanford University, called the paper very important, noting that in science, everything about aging seems so complicated we dont know where to start. By showing a pathway and a mechanism for explaining aging hair, Dr. Yi and colleagues may have provided a toehold.

Stem cells play a crucial role in the growth of hair in mice and in humans. Hair follicles, the tunnel-shaped miniature organs from which hairs grow, go through cyclical periods of growth in which a population of stem cells living in a specialized region called the bulge divide and become rapidly growing hair cells.

Sarah Millar, director of the Black Family Stem Cell Institute at the Icahn School of Medicine at Mount Sinai, who was not involved in Dr. Yis paper, explained that those cells give rise to the hair shaft and its sheath. Then, after a period of time, which is short for human body hair and much longer for hair on a persons head, the follicle becomes inactive and its lower part degenerates. The hair shaft stops growing and is shed, only to be replaced by a new strand of hair as the cycle repeats.

But while the rest of the follicle dies, a collection of stem cells remains in the bulge, ready to start turning into hair cells to grow a new strand of hair.

Dr. Yi, like most scientists, had assumed that with age the stem cells died in a process known as stem cell exhaustion. He expected that the death of a hair follicles stem cells meant that the hair would turn white and, when enough stem cells were lost, the strand of hair would die. But this hypothesis had not been fully tested.

Together with a graduate student, Chi Zhang, Dr. Yi decided that to understand the aging process in hair, he needed to watch individual strands of hair as they grew and aged.

Ordinarily, researchers who study aging take chunks of tissue from animals of different ages and examine the changes. There are two drawbacks to this approach, Dr. Yi said. First, the tissue is already dead. And it is not clear what led to the changes that are observed or what will come after them.

He decided his team would use a different method. They watched the growth of individual hair follicles in the ears of mice using a long wavelength laser that can penetrate deep into tissue. They labeled hair follicles with a green fluorescent protein, anesthetized the animals so they did not move, put their ear under the microscope and went back again and again to watch what was happening to the same hair follicle.

What they saw was a surprise: When the animals started to grow old and gray and lose their hair, their stem cells started to escape their little homes in the bulge. The cells changed their shapes from round to amoeba-like and squeezed out of tiny holes in the follicle. Then they recovered their normal shapes and darted away.

Sometimes, the escaping stem cells leapt long distances, in cellular terms, from the niche where they lived.

If I did not see it for myself I would not have believed it, Dr. Yi said. Its almost crazy in my mind.

The stem cells then vanished, perhaps consumed by the immune system.

Dr. Chan compared an animal's body to a car. If you run it long enough and dont replace parts, things wear out, he said. In the body, stem cells are like a mechanic, providing replacement parts, and in some organs like hair, blood and bone, the replacement is continual.

But with hair, it now looks as if the mechanic the stem cells simply walks off the job one day.

But why? Dr. Yi and his colleagues next step was to ask if genes are controlling the process. They discovered two FOXC1 and NFATC1 that were less active in older hair follicle cells. Their role was to imprison stem cells in the bulge. So the researchers bred mice that lacked those genes to see if they were the master controllers.

By the time the mice were 4 to 5 months old, they started losing hair. By age 16 months, when the animals were middle-aged, they looked ancient: They had lost a lot of hair and the sparse strands remaining were gray.

Now the researchers want to save the hair stem cells in aging mice.

This story of the discovery of a completely unexpected natural process makes Dr. Chuong wonder what remains to be learned about living creatures.

Nature has endless surprises waiting for us, he said. You can see fantastic things.

Excerpt from:
Losing Your Hair? You Might Blame the Great Stem Cell Escape. - The New York Times

Skin Stem Cells Comments Off on Losing Your Hair? You Might Blame the Great Stem Cell Escape. – The New York Times | October 5th, 2021

As a beauty writer and longtime skincare fanatic, Ive subscribed to various multi-step routines. Fast forward to having a baby and the general day-to-day life upheaval that comes along with it, and my definition of a worthwhile personal beauty regimen has changed a bit. While I still enjoy trying new products and learning about innovations in the beauty space, I find myself with less time (and patience) for the more laborious treatments and layering routines, and more interest in noticeable efficacy and multitasking capabilities.

The most effective skincare products and tools are great for paring down your routineand also ... [+] saving you some precious minutes in your day.

And whether youre a busy mom or simply short on me time these days (who isnt?), there are a myriad of tried-and-true products that get the job done without requiring a ton of effort on your part. From Herauxs next-level anti-inflammaging serum to NuFaces skin-smoothing device, these standout skincare products and tools are ideal for a results-driven regimen thats effective enough to knock a few steps off your routineand save you some precious minutes in your day.

Since its launch seven years ago, this antioxidant-rich hydrating facial oil has remained a cult favorite for anyone who wants an instant glow, with the added benefit of skin soothing, balancing and repair. Infused with 22 active botanical and essential oils, the lightweight yet potent treatment can help with everything from acne to sun damage. And when applied using founder April Gargiulos signature push-press technique, it feels like a simple yet luxurious way to start and end the day.

Like the brands Trinity device, the NuFace Fix uses microcurrent technology at a gentler level to help reduce the look of fine lines and wrinkles on the more delicate areas of the face: on the forehead, between brows and around the eyes and lips (filler-free plumping, anyone?). Its sleek, compact shape combined with ease of use makes it a no-brainer for skin smoothing results in a matter of minutes. Plus, it holds an ample 120-minute charge, which amounts to a couple months worth of use before needing to plug it ini.e. no fumbling with cords or a hefty device on a regular basis.

The latest product in Kate McLeods sustainably packaged, handcrafted self care collection and the first to focus on skincare, the Face Stone is essentially a waterless moisturizer that melts on contact with skin. A rich blend of nourishing and antioxidant ingredients like blue tansy, kokum butter and plum kernal borage helps even and soothe stressed skinsomething we could all use these days. An added bonus? Its solid form and shape makes it a natural massaging tool, making it ideal for a morning pick-me-up or the start of an evening wind-down ritual.

While it covers your mineral-based broad spectrum sunscreen needs, this multitasker does much more than that. The unique product uses the U Beautys proprietary Sun-Siren Capsule Technology to help reduce hyperpigmentation, discoloration and dark spots (whether from pregnancy melasma or suntanning sins of the past) while also shielding against UVA, UVB, infrared and blue light exposure. A little goes a long way with this rich balm-cream formula, and its hydrating enough to double as a moisturizer or primer by day and also great as an overnight spot treatment.

An excellent (and cleaner) dupe for Biologique Recherches oft-elusive Lotion P50, this Moon Juice exfoliator is a skin savior in its own right. The liquid formula includes glycolic, lactic and salicylic acids for gentle, pore-minimizing exfoliation paired with niacinamide and adaptogenic reishi to help boost the skins natural barrier. And besides looking pretty, the packaging is completely recyclable, from the sculptural cap and glass bottle to the outer carton.

January Labs is a clean beauty favorite for its science-backed, results-driven products that have long been favored by top aestheticians. Even those with more sensitive (or dry) skin can use this retinol serum without dealing with typical downsides (like redness, drying or peeling) thanks to its use of Retistar, a .5% retinol thats super effective yet non-irritating.

This at-home peel is as easy as can be, requiring a single once-over with the pre-soaked pad to revive dull skin. The duo of glycolic and salicylic acids provide skin-smoothing exfoliation while ingredients like chamomile and bilberry extract calm, soothe and help even skin tone. Pro tip: Use up excess product on any dry, flakey patches on arms and legs.

Created by stem cell biologists at the University of Southern California, this innovative serum features an anti-inflammaging HX-1 molecule thats combined with tried-and-tested ingredients like vitamin C, hyaluronic acid, peptides and red maple bark. The result? A silky, lightweight formula thats both rejuvenating (designed to help reduce the effects of stress and aging factors on skin) and preventative (strengthening stem cells on a molecular level).

The rest is here:
The Best Skincare Treatments For Time-Crunched Moms (Or Anyone Else Who Only Has Five Minutes To Spare) - Forbes

Skin Stem Cells Comments Off on The Best Skincare Treatments For Time-Crunched Moms (Or Anyone Else Who Only Has Five Minutes To Spare) – Forbes | October 5th, 2021