Sanford Health is continuing to explore the potential of orthobiologics and regenerative medicine with a study that may advance treatment for those living with osteoarthritis.

This trial known as ENDURE, which is run in full compliance with the FDA, will examine the safety and effectiveness of adult, adipose (fat)-derived stem cells.

Im really excited to get the trial started, said Donella Herman, M.D., a specialist in sports medicine at Sanford Health. Im even more excited about a year from now because I feel like were just going to learn so much about how effective this treatment is and what it is effective for.

Learn more: Orthopedics regenerative medicine at Sanford Health

Essentially, orthobiologics and regenerative medicine tap into the bodys natural healing ability.

Orthobiologics, a part of regenerative medicine, refers to the use of biological substances found in the body to help treat muscle and bone issues.

This may include injuries to muscles, tendons, and ligaments and as the focus of this trial more chronic conditions such as osteoarthritis.

When used properly by qualified providers, cell therapies are proving to be effective treatment options.

Our goals are to collect data for the different branches of orthobiologics, Dr. Herman said. Do people with knee osteoarthritis respond better with PRP (platelet rich plasma) or bone-marrow derived cells or fat-derived stem cells? Which works best? Which works for the longest time? Thats the kind of information were looking for. Its that kind of ratio that we just dont have right now.

One of the obstacles in regenerative medicine at this point, Dr. Herman said, is that its often being marketed by people with dubious clinical qualifications. They use underdeveloped research and make unfounded claims.

Its become a little bit of a wild, wild west with stem cells and PRP, Dr. Herman said. There are pop-up shops all over the place. Its one thing to have access to these things and allow access to people, but if youre not doing that in a responsible way and using that opportunity to build on a knowledge base, its kind of smoke and mirrors.

In sharp contrast to that scenario, this trial aims to add to the bank of knowledge in regenerative medicine.

We know we can tell people what were putting in their knee or their hip or their shoulder because were testing, Dr. Herman said. A lot of other places theyre just injecting it. They dont know if the stem cells are viable or not.

A typical candidate for the study, as described by Dr. Herman, might have osteoarthritis in their wrist and would not want to sacrifice a loss of motion that could come with surgery. They also would likely have exhausted the steroid injections treatment options that offer temporary relief.

We think of regenerative medicine as a possible alternative therapy that may help people put off surgery, Dr. Herman said. I dont ever tell people it means you will never need surgery but what it does is hopefully buy a little time until youre ready for that. There are lots of times where well do steroid injections until those fail and then they have to get a new joint. Were hoping for this to give us a bridge.

Once a patient is approved to be part of the ENDURE study, the procedure includes:

Follow-ups are then completed in person and over the phone.

Qualified candidates must be 18 or older and:

The cost associated with the study is not currently covered by insurance. By enrolling in the ENDURE study, however, participants may be playing a role in advancing regenerative cell research that could have the potential to improve care for future generations.

Those interested in hearing more about possible eligibility in the study can call (605) 328-3700.

Posted In Innovations, Orthopedics, Research, Specialty Care, Sports Medicine

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What happened

Shares of Longeveron (NASDAQ:LGVN), a clinical-stage biotechnology company headquartered in Miami, are soaring in response to good news from the U.S. Food and Drug Administration (FDA). Investors excited about an orphan drug designation for the company's stem cell treatment pushed the stock 36.7% higher as of 10:37 a.m. ET on Friday.

Longeveron is developing bone marrow-derived mesenchymal stromal cells for the treatment of a variety of age-related disorders including Alzheimer's disease. Today, the FDA granted the company's lead candidate, Lomecel-B, an orphan designation for the treatment of a rare condition called hypoplastic left heart syndrome.

Image source: Getty Images.

Generally, orphan drug designations aren't something to get excited about. The FDA hands them out like candy to just about anyone that intends to develop a drug for underserved patients with a rare condition.

If approved to treat hypoplastic left heart syndrome, this orphan drug designation can begin assisting Longeveron with a handful of useful benefits. The most important one is seven years of market exclusivity.

There isn't much of a difference between the bone marrow-derived stem cells in Lomecel-B and the stem cells biomedical scientists have been researching for decades. They've been renamed medical signaling cells in recent years, but evidence of a significant benefit for specific indications remains elusive.

Longeveron is now a good stock to buy, and betting against it could prove disastrous, too. In the first quarter of 2022, Longeveron intends to begin clinical trials with Lomecel-B as a treatment for Alzheimer's disease.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Here's Why Longeveron Is Surging Today - Motley Fool

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Significance

The recently introduced RNA velocity methods, by leveraging the intrinsic RNA splicing process, have shown their unique capability of identifying the directionality of the cell differentiation trajectory. However, due to the minimal amount of unspliced RNA contents, the estimation of RNA velocity suffers from high noise and may result in less reliable trajectories. Here, we present Velocity Autoencoder (VeloAE), a tailored autoencoder to denoise RNA velocity for more accurate quantification of cell transitions. Through various biological systems, we demonstrate its effectiveness for correcting the inferred trajectory and its interpretability for linking the learned dimensions to underlying biological processes.

RNA velocity is a promising technique for quantifying cellular transitions from single-cell transcriptome experiments and revealing transient cellular dynamics among a heterogeneous cell population. However, the cell transitions estimated from high-dimensional RNA velocity are often unstable or inaccurate, partly due to the high technical noise and less informative projection. Here, we present Velocity Autoencoder (VeloAE), a tailored representation learning method, to learn a low-dimensional representation of RNA velocity on which cellular transitions can be robustly estimated. On various experimental datasets, we show that VeloAE can both accurately identify stimulation dynamics in time-series designs and effectively capture expected cellular differentiation in different biological systems. VeloAE, therefore, enhances the usefulness of RNA velocity for studying a wide range of biological processes.

Author contributions: C.Q. and Y.H. designed research; C.Q. performed research; C.Q. contributed new reagents/analytic tools; C.Q. analyzed data; and C.Q. and Y.H. wrote the paper.

The authors declare no competing interest.

This article is a PNAS Direct Submission.

This article contains supporting information online at https://www.pnas.org/lookup/suppl/doi:10.1073/pnas.2105859118/-/DCSupplemental.

VeloAE is an open-source Python package available at GitHub, https://github.com/qiaochen/VeloAE. All the analysis notebooks for reproducing the results are also available in this repository. Previously published data were used for this work (8, 10, 1925).

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Representation learning of RNA velocity reveals robust cell transitions - pnas.org

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In the summer of 2015, a 7-year-old named Hassan was admitted to the burn unit of the Ruhr University Childrens Hospital in Bochum, Germany, with red, oozing wounds from head to toe.

It wasnt a fire that took his skin. It was a bacterial infection, resulting from an incurable genetic disorder. Called junctional epidermolysis bullosa, the condition deprives the skin of a protein needed to hold its layers together and leads to large, painful lesions. For kids, its often fatal. And indeed, Hassans doctors told his parents, Syrian refugees who had fled to Germany, the young boy was dying.

The doctors tried one last thing to save him. They cut out a tiny, unblistered patch of skin from the childs groin and sent it to the laboratory of Michele de Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia. De Lucas team used a viral vector to ferry into Hassans skin cells a functional version of the gene LAMB3, which codes for laminin, the protein that anchors the surface of the skin to the layers below.

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Then the scientists grew the modified cells into sheets big enough for Ruhr University plastic surgeons Tobias Hirsch and Maximilian Kueckelhaus to graft onto Hassans raw, bedridden body, which they did over the course of that October, November, and the following January.

It worked better than the boys doctors could have imagined. In 2017, de Luca, Hirsch, Kueckelhaus, and their colleagues reported that Hassan was doing well, living like a normal boy in his lab-grown skin. At the time though, there was still a big question on all their minds: How long would it last? Would the transgenic stem cells keep replenishing the skin or would they sputter out? Or worse could they trigger a cascade of cancer-causing reactions?

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Today, the same team is out with an update. Five years and five months after the initial intervention, Hassan is still, for the most part, thriving in fully functional skin that has grown with the now-teenager. He is attending school, and playing sports with his friends and siblings, though he avoids swimming due to blistering in the areas that werent replaced by the lab-grown skin. One of his favorite activities is a pedal-powered go kart. There are no signs his modified stem cells have lost their steam, and no traces of tumors to be found.

The encouraging follow-up data has been instrumental in moving forward a larger clinical trial of the approach, offering hope to the 500,000 epidermolysis bullosa patients worldwide currently living without treatment options.

We were astonished by the speedy recovery, Kueckelhaus, now at University Hospital Muenster, told STAT via email. But experience from skin transplantation in other settings made him and his colleagues wary of the grafts failing as the months and years wore on. Thankfully, wrote Kueckelhaus, those fears never materialized. We are very happy to be able to prove that none of these complications appeared and the genetically modified skin remains 100% stable. The chances are good that he will be able to live a relatively normal life.

Over the last five years, Hassans team of doctors and researchers has put his new skin through a battery of tests checking it for sensitivity to hot and cold, water retention, pigmentation and hemoglobin levels, and if it had developed all the structures youd expect healthy skin to have, including sweat glands and hair follicles. Across the board, the engineered skin appeared normal, without the need for moisturizers or medical ointments. The only flaw they found was that Hassans skin wasnt as sensitive to fine touch, especially in his lower right leg. This mild neuropathy they attributed not to the graft itself, but to how that limb was prepared doctors used a more aggressive technique that might have damaged the nerves there.

The team also used molecular techniques to trace the cells theyd grown in the lab as they divided and expanded over Hassans body. They found that all the different kinds of cells composing the boys new skin were being generated by a small pool of self-renewing stem cells called holoclone-forming cells, carrying the Italian teams genetic correction.

This was quite an insight into the biology of the epidermis, said de Luca. Its an insight he expects will have huge consequences for any efforts to advance similar gene therapies for treating other diseases affecting the skin. You have to have the holoclone-forming cells in your culture if you want to have long-lasting epidermis, he said.

The approach pioneered by de Lucas team will soon be headed for its biggest clinical test yet, after nearly a decade of fits and starts. They expect to begin recruiting for a multi-center Phase 2/3 trial sometime next year.

De Luca first successfully treated a junctional EB patient in 2005. But then a change to European Union laws governing cell and gene therapies forced his team to stop work while they found ways to comply with the new rules. It took years of paperwork, building a manufacturing facility, and spinning out a small biotech company called Holostem to be ready to begin clinical research again. Hassan came along right as they were gearing up for a Phase 1 trial, but data from the boys case, which was granted approval under a compassionate use provision, convinced regulators that the cell grafts could move to larger, more pivotal trials, according to de Luca.

We didnt cure the disease, he told STAT. But the skin has been restored, basically permanently. We did not observe a single blister in five years. The wound healing is normal, the skin is robust. From this point of view, the quality of life is not even comparable to what it was before.

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Syrian refugee is thriving five years after last-gasp gene therapy - STAT - STAT

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Stem cell therapy, sometimes called regenerative medicine, is one of the most exciting areas of the life sciences sector right now.

Since the pandemic, the sector has emerged into the publics spotlight with new developments in mRNA-based vaccines and therapies.

Nasdaq is the obvious breeding ground for world-class stem cell companies with the likes of Moderna and BioNTech, and lesser known names like Anavex and Enochian.

In Australia, Mesoblast (ASX:MSB) has long been the local poster child for the regenerative medicine industry.

Mesoblast has developed a platform of innovative cellular medicines, but the company has struggled since the FDA rejected its drug in October last year.

Now, other ASX companies like Cynata Therapeutics (ASX:CYP)are making rapid progress to take over the mantle from MSB in this hot field.

Cynata is developing a mesenchymal stem cells (or MSC) technology, which it says has huge therapeutic potential for numerous unmet medical needs.

This includes asthma, heart attack, sepsis, and acute respiratory distress syndrome (ARDS), which all add up to a market opportunity worth $46bn, says the company.

According to CEO Dr Ross Macdonald, who spoke to Stockhead today, MSC is the hottest segment of stem cell therapy at the moment, and has gained a lot of attention recently.

There is a huge interest, and theres been more than 1000 clinical trials conducted around the world using MSC, Dr Macdonald told Stockhead.

He explains that the humans immune system controls many of the bodys functions responsible for repairing tissue after injury or disease, and defending against invading germs like viruses or bacteria.

And just like an orchestral conductor, MSC seems to be playing a central role in that coordination within our immune system.

We now have a firm understanding of how those cells coordinate the bodys responses, and can use that knowledge to enhance those processes that they control, Dr Macdonald explained.

In short, MSC therapies work by expressing a variety of chemokines and cytokines that aid in repair of degraded tissue, restoration of normal tissue metabolism and, most importantly, counteracting inflammation.

And because MSCs play that co-ordination role within the immune system, they can be used to treat different diseases.

However theres one big problem with cell-based therapies, and its not to do with the safety and efficacy.

Its how to manufacture these products on a mass scale, that is the greatest challenge right now, says Dr Macdonald.

Unlike aspirin where it can be synthesised in a chemical lab and produced in bulk, manufacturing a living drug like a cell is a whole lot more complicated.

But that big challenge is the exact area of strength and competitive advantage that Cynata has, Dr Macdonald told Stockhead.

He says Cynata has a technology platform which allows it to manufacture essentially limitless quantities of MSCs, consistently and economically.

Dr Macdonald explains there are two approaches to using cell therapy, the autologous and the allogeneic approach.

The autologous approach is where the patient themselves serves as their own donor.

This is obviously bespoke and inefficient, because the drug can only be manufactured for that one patient, and is obviously not an industrialised process, he said.

But by taking an allogeneic approach, Cynata has the ability to start with a one time donation of cells from one single donor.

Well never have to go back to that human donor ever again, so our process of producing cells has become a very much more typical industrialised process.

The company has a patent for this, with two clinical trials underway and two more under preparation.

A Phase 3 clinical trial for osteoarthritis which is funded by a NHMRC grant has progressed the furthest, while a Phase 2 trial in COVID-19 is ongoing.

Meanwhile a Phase 1 study in GvHD, which was published in prestigious journal Nature Medicine, is probably the closest to commercialisation according to Dr Macdonald.

GvHD is a challenging disease which occurs in patients who have had a bone marrow transplant as part of their chemotherapy treatment for cancer.

Chemo is still very much a sledgehammer therapy where you use very toxic drugs that do kill the cancer cells, but they also kill the surrounding healthy cells that grow hair and bone marrow.

Unfortunately for many patients, the bone marrow transplant reacts against their body and starts to attack all of the tissues in the body, and its ultimately fatal.

Its a horrible death, destroying the lungs, liver, intestines and the skin, Macdonald explains.

Cynatas MSC therapy has been shown to reset that reaction, so the patient can recover from the GvHD, and also recover from their underlying cancer.

With all these clinical trials concurrently under way, Macdonald believes there is a clear significant upside potential for Cynata, particularly given its small market cap of $70m compared to other similar plays like Mesoblast ($1 billion market cap).

Osteopore (ASX:OSX) focuses in bones and specialises in the production of 3D printed bioresorbable implants that are used in surgical procedures to assist with the natural stages of bone healing.

The 3D bio-printer makes a scaffold that mimics bone, with a patented micro-architecture which traps the patients own stem cells.

Orthocell (ASX:OCC) develops collagen medical devices and cellular therapies for the repair and regeneration of human tendons, bone, nerve and cartilage defects.

Its flagship product, the CelGro, is a naturally derived collagen medical device for tissue repair.

Aroa Biosurgery (ASX:ARX) develops FDA-approved medical devices for wounds and tissue repair using its extracellular matrix (ECM) technology, mainly in the United States.

Recent study shows 100% success rates from the use of its Myriad product when patients underwent surgical reconstruction of exposed vital structures such as bone and tendon.

Regeneus (ASX:RGS) Progenza is a cellular therapy targeting pain and inflammation which uses Secretome to improve not only the resident tissue, but the MSCs themselves.

It fills a gap in the current treatment market for osteoarthritis, by providing disease modification and pain relief to address patient symptoms.

Anteris Technologies (ASX:AVR) claims that its Adapt Technology is the first and only bio-scaffold technology that completely re-engineers xenograft tissue into a pure collagen scaffold.

A recent study indicated that Adapt-treated tissue has superior anti-calcification attributes compared with tissues used in competitor valves.

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Mesoblast has long been the one poster child for stem cell therapy. Now Cynata and other ASX stocks have e ... - Stockhead

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Inbreeding and wild tigers at risk of extinction

As habitat fragmentation increases worldwide, wild animal populations are shrinking and becoming more isolated, thus facing a heightened risk of inbreeding and extinction. The extent to which the viability of small, isolated populations could be improved by purging deleterious alleles through natural selection is unclear. Anubhab Khan et al. analyzed whole-genome sequences from 57 wild Bengal tigers from either a small, isolated population or large, connected populations in India. The results revealed evidence of partial purging of highly detrimental variants across populations. However, the small, isolated population showed genomic signs of greater inbreeding and a higher overall frequency of deleterious alleles, compared with two large populations. On average, pairs of individuals from the small, isolated population shared approximately 40% of their genomes in tracts at least 1 megabase long, whereas pairs from the large, connected populations shared approximately 1525% of their genomes. Together, the findings suggest that purging may not eliminate all detrimental alleles and inbreeding-associated fitness costs in small, isolated populations. According to the authors, the findings highlight the need for genetic rescue strategies that enhance the fitness of inbred populations by decreasing the frequency of harmful mutations and increasing genetic variation. J.W.

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In This Issue - pnas.org

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All articles published by MDPI are made immediately available worldwide under an open access license. No specialpermission is required to reuse all or part of the article published by MDPI, including figures and tables. Forarticles published under an open access Creative Common CC BY license, any part of the article may be reused withoutpermission provided that the original article is clearly cited.

Feature Papers represent the most advanced research with significant potential for high impact in the field. FeaturePapers are submitted upon individual invitation or recommendation by the scientific editors and undergo peer reviewprior to publication.

The Feature Paper can be either an original research article, a substantial novel research study that often involvesseveral techniques or approaches, or a comprehensive review paper with concise and precise updates on the latestprogress in the field that systematically reviews the most exciting advances in scientific literature. This type ofpaper provides an outlook on future directions of research or possible applications.

Editors Choice articles are based on recommendations by the scientific editors of MDPI journals from around the world.Editors select a small number of articles recently published in the journal that they believe will be particularlyinteresting to authors, or important in this field. The aim is to provide a snapshot of some of the most exciting workpublished in the various research areas of the journal.

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Cells | Free Full-Text | Improving Cardiac Reprogramming ...

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Event summary produced by The Globe and Mail Events team. The Globes editorial department was not involved.

Canada was a pioneer of stem cell research and today, innovators are developing clinical trials to test regenerative treatments for a range of illnesses including cardiac disease and Parkinsons. At the same time, theyre navigating risks and considerations that often surround medical innovations.

The Globe and Mail hosted a webcast on November 30 to explore the promise and potential of stem cells. Speakers also discussed ethical issues, misinformation and the importance of rigorous evaluation in bringing new treatments to market.

Missed the live event or would like to view it again? Scroll down to the video player, below.

Andr Picard, health reporter and columnist with The Globe and Mail moderated the event and hosted the following speakers:

Read a summary of the event here

View the full webcast, below:

The Globe and Mail presented the webcast with sponsor support from Bayer.

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LAWRENCE The human body contains trillions of cells at any given moment, each doing highly specialized work to help us function but they dont operate in isolation. Imagine a sophisticated FedEx or UPS delivery network empowering communication between our cells. The nano-sized delivery vehicles in this scenario are called exosomes, and a company born from technology developed at the University of Kansas is harnessing the power of these tiny vessels to enable tomorrows medical breakthroughs.

Clara Biotech, founded by KU engineering alumnus Jim West and former KU professor of chemical & petroleum engineering and chemistry Mei He, has spent the last three years refining a novel technology to isolate and purify exosomes, which can be used for early disease diagnosis, targeted drug delivery, cancer immunotherapy and other forms of regenerative medicine.

Now, the company is poised to commercialize its first product after recently finalizing $1.5 million in seed funding and being recognized in a national competition. Clara Biotech was the only Midwest company singled out in MedTech Innovators Biotools Innovator program, which recognizes the 10 best life science tools startups. The company received $10,000 for securing a spot in the 2021 cohort and a $5,000 best-video award for a one-minute spot introducing the company and detailing what sets it apart.

Clara Biotech was founded to help move exosomes from the bench to the bedside, said West, who serves as Claras CEO. Our company is about building a platform that everybody can leverage to bring their products to market and help solve challenges around isolation and purification, which today is one of the number one issues in the field.

Exosomes deliver genetic information to cells throughout the body. Exosomes from regenerative cells, such as stem cells, can help the body heal and repair itself. Exosomes released from diseased cells might be used for early detection and diagnosis of cancer and other conditions.

But at 100 nanometers in diameter less than the wavelength of visible light exosomes are difficult to handle.

Clara Biotechs patented ExoRelease platform is unique in the industry. Current processes rely on bulk isolation, whereas Claras capture and release technology isolates pure exosomes. This allows researchers to easily isolate and target specific exosomes including cardiac, neurological, cancer and others and use them for therapeutic treatments and drug delivery platforms.

Im very excited about the work that Clara Biotech is doing to improve exosome purification, said Kathryn Zavala, managing director of BioTools Innovator. Their technology has the potential to significantly impact how we diagnose and treat diseases by advancing the field of exosome research and development.

Clara Biotech launched in 2018 with a Small Business Innovation Research grant from the National Cancer Institute and received training through the National Science Foundations Innovation Corps (I-Corps) program on how to transfer knowledge into products and processes that benefit society. It has seven full-time employees, and its lab is housed in the KU Innovation Park.

Clara Biotech is an example of how KU innovation provides the foundation to form a company that addresses societal needs and creates Kansas jobs, said Tricia Bergman, KUs director of strategic partnerships. It also illustrates how technology developed in KU labs can transition into the KU Innovation Park, where the company can continue to develop through ongoing partnerships with the university.

Until now, Clara Biotech has provided lab services to its customers. Now, its moving toward packaging its technology so other companies, labs and researchers can leverage it to complete the isolation process themselves.

Were trying to democratize access to these exosomes, West said.

Clara Biotech is beta-testing kits containing its isolation technology with promising results from early adopters and hopes to launch its first product by the end of the year.

Building a company is probably the hardest thing Ive ever done in my life, but its also super rewarding, West said. The work were doing is really important.

Photo: Jim West, CEO of Clara Biotech, holds the two checks his company won at MedTech Innovators Biotools Innovator program in San Diego in October.

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Biotech company with KU roots wins national competition, secures funding to help move research 'from bench to bedside' | The University of Kansas - KU...

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Findings Continue to Change the Treatment of Blood Cancers

HACKENSACK, N.J., Dec. 9, 2021 /PRNewswire/ -- Researchers from Hackensack Meridian Health John Theurer Cancer Center (JTCC), a part of the Georgetown Lombardi Comprehensive Cancer Center, will present updates on treatment advances in multiple myeloma, lymphoma, leukemia, and bone marrow transplantation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition, to be held virtually and live at the Georgia World Congress Center in Atlanta from December 11-14, 2021.

"John Theurer Cancer Center is a world leader in the care of people with hematologic malignancies and a pioneer in clinical research related to blood cancers. The acceptance of 47 studies from our investigators demonstrates our expertise in this area and our commitment to improving outcomes not only for our own patients, but people affected by these diseases everywhere," said Andre Goy, MD, MS, chairman and executive director of the John Theurer Cancer Center.

This year's presentations will include a plenary session as the #2 ranked abstract for the entire conference with data that will change the paradigm in the treatment of relapsed aggressive lymphoma for the FIRST TIME in 40 years. Dr. Lori Leslie, MD, director of the Indolent Lymphoma and Chronic Lymphocytic Leukemia Research Programs at JTCC will be co-presenter of the phase III international ZUMA-7 clinical trial (abstract #2), which compared axicabtagene ciloleucel (axi-cel) CAR T-cell therapy with standard of care (SOC) in patients with relapsed / refractory diffuse large B-cell lymphoma (DLBCL) after initial therapy. For decades the SOC has been high dose therapy followed by autologous stem cell transplant (ASCT) but patients with high risk disease and / or early relapse still do very poorly. Axi-cel is now used to treat DLBCL that have failed two prior regimens of treatment, including standard salvage chemoimmunotherapy (CIT) followed by ASCT.

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Bringing axi-cel earlier as second line therapy resulted in a 2.5-fold increase in median event-free survival (defined as the time without any cancer progression or any related complications) and doubled the complete response rate (65% vs 32%).

"This study is the first to change the paradigm for relapsed and refractory DLBCL that was established decades ago, demonstrating significant and clinically meaningful improvements in outcome," said Dr. Leslie. "Axi-cel may replace chemoimmunotherapy and autologous stem cell transplantation as the standard of care for people with DLBCL that relapses or persists after initial treatment. It is a game-changer."

The JTCC presentations address new developments in the treatment of multiple myeloma, lymphoma, leukemia, and bone marrow transplantation, as well as a study assessing gene therapy for sickle cell disease in pediatric patients.

Multiple Myeloma Research

Adding a PI3K inhibitor improved duration of CAR T-cell response. (Abstract #548, David S. Siegel, MD, PhD) In this phase I clinical trial, researchers showed that adding a PI3 kinase inhibitor called bb007 to bb2121 CAR T-cell therapy (forming a combined therapy called bb21217) in relapsed/refractory multiple myeloma (MM) patients who had three or more regimens of treatment resulted in a duration of response of 17 months (compared with 10 months for bb2121 alone in a prior study), and CAR T cells were detectable longer.

Study shows feasibility of "off the shelf" donated CAR T cells. (Abstract #651, David S. Siegel, MD, PhD). Current CAR T-cell therapies involve expensive modification of a patient's own T cells. Allogeneic (donated) CAR T cells represent a potentially more accessible, less expensive option but carry the risk of rejection and complications such as graft-vs-host disease. The phase I UNIVERSAL study demonstrated the safety of donated anti-BCMA CAR T cells in heavily pretreated MM patients, with mild to moderate side effects as expected for this type of immunotherapy.

Novel targeted MM therapies. Three abstracts provided additional data on novel targeted agents for relapsed/refractory MM. Selinexor was FDA approved in December 2020 and is being assessed in combination with other agents. A study of once-weekly oral selinexor with pomalidomide and dexamethasone (abstract #2748, Noa Biran, MD) showed an overall response rate of more than 60% in relapsed/refractory MM, including patients whose disease persisted after CAR T-cell therapy or after anti-CD38 antibody treatment. This is important because patients with MM after CAR T-cell therapy usually do not respond to additional treatment.

Study shows patients fare better if treated in high-volume academic medical centers. (Abstract #2996, David Vesole, MD, PhD, with Lombardi Comprehensive Cancer Center researchers) An analysis of data from the National Cancer Database of nearly 175,000 patients with MM treated at all types of facilities showed that the median overall survival was 75.5 months at high-volume centers versus 50.2 months at low-volume centers. Academic/research cancer programs with high volumes have the best outcomes in MM and are more likely to use chemotherapy, immunotherapy, and autologous stem cell transplantation than low-volume centers, particularly community cancer centers.

Lymphoma Research

Long-term data confirm durability of CAR T-cell benefit in indolent lymphoma. (Abstract #93, Lori Leslie, MD) An update of the pivotal ZUMA-5 clinical trial, which led to the approval of axi-cel CAR T-cell therapy for relapsed/refractory follicular lymphoma, confirmed continued benefit in patients with indolent lymphoma. In follicular lymphoma (most common subtype of indolent lymphoma), high response rates translated to durable responses, with a median duration of response of 38.6 months and 57% of patients free of cancer progression at last follow-up.

Study confirms benefit of CAR T-cell therapy for mantle cell lymphoma (MCL). (Abstract #744, Andre Goy, MD) ZUMA-2 led to the first approval of CAR T-cell therapy for MCL. An analysis of real-world data of MCL patients who received this treatment, 73% of whom would not have been eligible for ZUMA-2, demonstrated similar effectiveness, with an overall response rate of 86% and 64% achieving a complete response. The results support the paradigm-shifting benefit of this therapy in a heavily pretreated patient population where the median overall survival would have otherwise been very poor.

Molecular biomarkers predictive of CAR T-cell response. (Abstract #165, Andrew Ip, MD, Andre Goy, MD) Researchers performed whole exome and transcriptome sequencing to show that patients with DLBCL who had genetic signatures of high-risk disease with standard initial therapy do well with CAR T-cell therapy. Some mutations predicted good versus poor outcomes after CAR T-cell therapyreflecting differences in the tumor or its microenvironmentand may provide the rationale for choosing the most appropriate treatment for each patient and augmenting the response to CAR T-cell therapy.

Value of adding brentuximab to standard chemotherapy for peripheral T-cell lymphoma (Abstract #133, Tatyana Feldman, MD, Lori Leslie, MD) Non-anaplastic subtypes of T-cell lymphoma have poor outcomes and require new options. This study showed that adding brentuximab to conventional combination chemotherapy was tolerable and effective in patients with non-anaplastic CD30-positive peripheral T-cell lymphoma.

Machine learning useful for stratifying lymphoma patients. (Abstract #2395, Andre Goy, MD) Using machine learning and data on 380 patients with DLBCL with expression levels of 180 genes, researchers used machine learning to develop a model to reliably stratify patients with DLBCL treated with R-CHOP combination therapy into four survival subgroups. The model can be used to identify which patients may not respond well to R-CHOPa standard DLBCL treatmentand instead be considered for other therapies or clinical trials.

Lymphoma/CLL adversely affects COVID-19 outcomes. (Abstract #184, Lori Leslie, MD) A study of electronic medical record data on 500 patients with lymphoma, chronic lymphocytic leukemia (CLL), or other lymphoid cancers who tested positive for SARS-CoV-2 showed that those with aggressive non-Hodgkin lymphoma and CLL and patients who had received recent cytotoxic chemotherapy or anti-CD20 antibody treatment (such as rituximab) may be at risk for poor COVID-19 outcomes. JTCC researchers are now working with investigators in the Center for Discovery and Innovation to study T-cell immunity in people with cancer.

Other studies focused on adding ublituximab and umbralisib to ibrutinib in people with CLL (Abstract #395, Lori Leslie, MD) and assessing cerdulatinib as monotherapy for patients with relapsed/refractory peripheral T-cell lymphoma (Abstract #622, Tatayana Feldman, MD).

Leukemia Research

Oral therapy for low-risk myelodysplastic syndrome (MDS) (Abstract #66, James McCloskey, MD) People with MDS are at risk for developing acute leukemia. Those with low-risk MDS may receive supportive care for low blood counts. Patients with high-risk MDS have received inconvenient injections with drugs such as azacitidine and decitabine. This study showed that oral decitabine and cedazuridine was pharmacokinetically equivalent to intravenous decitabine; in patients with low-risk MDS, the oral treatment was well tolerated with prolonged treatment and may be useful for preventing the progression of this disease to leukemia.

Effectiveness of adding venetoclax to gilteritinib effective for FLT3-mutated acute leukemia (Abstract #691, James McCloskey, MD) Acute myeloid leukemia (AML) with FLT3 mutations initially responds to FLT3 inhibitors but frequently becomes resistant to these drugs. This study showed that giving venetoclax (a BCL2 inhibitor) with the FLT3 inhibitor gilteritinib was very effective, clearing the FLT3 mutation in most patients, and was associated with longer overall survivaleven in patients with high-risk subtypes.

Liquid biopsy for detecting molecular abnormalities in AML (Abstract #3463, Jamie Koprivnikar, MD, James McCloskey, MD, and others) This study assessed next-generation sequencing (NGS) to detect molecular abnormalities in AML using liquid biopsies. The data show that this approach is reliable for detecting structural chromosomal abnormalities in myeloid neoplasms. It could potentially replace the need for conventional cytogenetic testing, be much more convenient (replacing bone marrow biopsies for materials), and be more cost-effective.

Bone Marrow Transplantation Research

Next-generation sequencing and liquid biopsy valuable for detecting early relapse after stem cell transplantation. (Abstract #1828, Scott Rowley, MD, Michele Donato, MD, Maher Albitar, MD, and others) Cell-free DNA was isolated from the peripheral blood post-allogeneic transplant in patients treated for AML, acute lymphocytic leukemia, chronic myelogenous leukemia, chronic myelomonocytic leukemia, MDS, MM, and lymphoma. Researchers showed that NGS and liquid biopsy are useful for detecting residual disease. The data suggest that this approach, which examines cancer DNA in peripheral blood rather than a sample from a bone marrow biopsy, may be effective for detecting and managing minimal residual disease (MRD)the next frontier in oncologyenabling doctors to modify therapy to achieve MRD negative status or, during transplantation, to adjust immunosuppressors or use additional T cells to prevent relapse.

Use of NGS and machine learning after transplant to predict graft-vs-host disease (GVHD) (Abstract #2892, Scott Rowley, MD, Michele Donato, MD, Maher Albitar, MD, and others) Using NGS RNA sequencing plus a machine learning approach, researchers looked at over 1,400 genes in 46 patients who had an allogeneic bone marrow transplant and developed a model based on 7 genes to predict acute GVHD, one of the most significant complications of receiving a transplant from a bone marrow donor. There are currently no valid ways to predict acute GVHD and intervene early until patients become symptomatic. The ability to identify molecular markers of this complication while patients are asymptomatic may allow for early intervention to prevent GVHD.

Sickle Cell Disease Research

Sustained quality of life in patients receiving gene therapy for sickle cell disease (Abstract #7, Stacey Rifkin-Zenenberg, DO, Hackensack University Medical Center) LentiGlobin gene therapy (bb1111) has been under study in a clinical trial as a one-time treatment and cure for sickle cell disease. This study presented long-term quality of life data for one group in the study, demonstrating an improvement in hematologic parameters and complete resolution of veno-occlusive events and related pain as well as sustained and clinically meaningful improvement in quality of life 6 and 24 months post-treatment. Even patients with the worst baseline quality of life scores experienced a benefit. LentiGlobin is the first gene therapy for sickle cell disease and the results of this study are very promising, with the potential to change patient outcomes for this chronic debilitating disease.

The full set of ASH data presentations by JTCC researchers is as follows:

Abstract #

Type

Title

Authors

Presenting (PST)

2

Plenary Scientific Session

Primary Analysis of ZUMA-7: A Phase 3 Randomized Trial of Axicabtagene Ciloleucel (Axi-Cel) Versus Standard-of-Care Therapy in Patients with Relapsed/Refractory Large B-Cell Lymphoma

Lori A. Leslie

Sunday, December 12, 2021: 2:00 PM-4:00 PM

7

Oral

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Stacey Rifkin

Saturday, December 11, 2021: 9:30 AM-11:00 AM

50

Oral

A Large Multicenter Real-World Evidence (RWE) Analysis of Autoimmune (AI) Diseases and Lymphoma: Histologic Associations, Disease Characteristics, Survival, and Prognostication

Tatyana A. Feldman, Jason Lofters

Saturday, December 11, 2021: 9:45 AM

66

Oral

Oral Decitabine/Cedazuridine in Patients with Lower Risk Myelodysplastic Syndrome: A Longer-Term Follow-up of from the Ascertain Study

James K McCloskey

Saturday, December 11, 2021: 10:45 AM

93

Oral

Long-Term Follow-up Analysis of ZUMA-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL)

Pashna N. Munshi, Lori A. Leslie,

Saturday, December 11, 2021: 10:00 AM

133

Oral

Brentuximab Vedotin Plus Cyclophosphamide, Doxorubicin, Etoposide, and Prednisone (CHEP-BV) Followed By BV Consolidation in Patients with CD30-Expressing Peripheral T-Cell Lymphomas

Tatyana A. Feldman, Lori A. Leslie

Saturday, December 11, 2021: 12:00 PM-1:30 PM

165

Oral

Impact of Molecular Features of Diffuse Large B-Cell Lymphoma on Treatment Outcomes with Anti-CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy

Andrew Ip, MD, Andre Goy

Saturday, December 11, 2021: 12:30 PM

184

Oral

A Multi-Center Retrospective Review of COVID-19 Outcomes in Patients with Lymphoid Malignancy

Lori A. Leslie

Saturday, December 11, 2021: 12:00 PM-1:30 PM

307

Oral

Post Hoc Analysis of Responses to Ponatinib in Patients with Chronic-Phase Chronic Myeloid Leukemia (CP-CML) By Baseline BCR-ABL1 Level and Baseline Mutation Status in the Optic Trial

James K McCloskey

Saturday, December 11, 2021: 4:00 PM-5:30 PM

395

Oral

A Phase 2 Study Evaluating the Addition of Ublituximab and Umbralisib (U2) to Ibrutinib in Patients with Chronic Lymphocytic Leukemia (CLL): A Minimal Residual Disease (MRD)-Driven, Time-Limited Approach

Lori A. Leslie

Sunday, December 12, 2021: 10:30 AM

548

Oral

Updated Clinical and Correlative Results from the Phase I CRB-402 Study of the BCMA-Targeted CAR T Cell Therapy bb21217 in Patients with Relapsed and Refractory Multiple Myeloma

David S. Siegel

Sunday, December 12, 2021: 4:30 PM-6:00 PM

561

Oral

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Stacey Rifkin-Zenenberg

Sunday, December 12, 2021: 4:30 PM-6:00 PM

622

Oral

Phase 2a Study of the Dual SYK/JAK Inhibitor Cerdulatinib (ALXN2075) As Monotherapy in Patients with Relapsed/Refractory Peripheral T-Cell Lymphoma

Feldman

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BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that BESREMi (ropeginterferon alfa-2b-njft) is now commercially available in the U.S. to eligible patients with polycythemia vera (PV). BESREMi was approved by the FDA in November as the only interferon for adults with polycythemia vera. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

Today marks the beginning of a new chapter in the treatment of PV. Our team is delivering on our goal to bring an innovative solution that may help more people manage not only the symptoms of PV, but target the disease itself to gain durable control with potential to reduce progression over time, said Meredith Manning, U.S. General Manager. We look forward to working closely with U.S. providers to raise awareness of this therapy and help advance treatment goals.

PharmaEssentia SOURCE Now Available to Support People with PV in the U.S.

With the commercial availability of BESREMi, PharmaEssentia is also launching a comprehensive patient support program, which can be found at http://www.pharmaessentiaSOURCE.com.

The SOURCE program is available for patients prescribed BESREMi and offers a full suite of services designed to help patients start and stay on therapy. Services include insurance navigation support, titration and injection training, and ongoing adherence guidance. The program also includes physician resources, including guides to help patients get started on treatment and ordering processes.

As part of this program, PharmaEssentia will help patients with financial barriers to starting therapy. The company is offering co-pay and co-insurance programs to assist eligible patients who experience financial need. Programs include a $0 copay card for commercially insured patients, temporary product supply in case of insurance delays and/or gaps in coverage, free drug for the uninsured and under-insured as well as assistance identifying additional support as needed.

Weve designed SOURCE with active input from the PV community to simplify the process for appropriate patients to initiate and maintain access to BESREMi and to benefit from its effects over the long-term, added Manning. Our goal is to ensure that any appropriate person with PV who is prescribed BESREMi is able to receive the therapy.

About Polycythemia Vera

Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.1

About BESREMi

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. After one year, patients with stable complete hematologic response (CHR) can be treated with BESREMi every four weeks.

BESREMi has orphan drug designation for treatment of PV in the United States. The product was approved by the European Medicines Agency (EMA) in 2019 and has received approval in Taiwan and South Korea. BESREMi was invented and is manufactured by PharmaEssentia.

Important Safety Information

IMPORTANT SAFETY INFORMATION AND INDICATIONS

WARNING: RISK OF SERIOUS DISORDERS

Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy.

CONTRAINDICATIONS

WARNINGS AND PRECAUTIONS

Other central nervous system effects, including suicidal ideation, attempted suicide, aggression, bipolar disorder, mania and confusion have been observed with other interferon alfa products.

Closely monitor patients for any symptoms of psychiatric disorders and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue BESREMi therapy.

ADVERSE REACTIONS

The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%), fatigue (12%), myalgia (11%), and influenza-like illness (11%).

DRUG INTERACTIONS

Patients on BESREMi who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive myelosuppression. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity.

USE IN SPECIFIC POPULATIONS

Please see accompanying full Prescribing Information, including Boxed Warning.

About PharmaEssentia

PharmaEssentia Corporation (TPEx: 6446), based in Taipei, Taiwan, is a rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, the company aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today the company is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung. For more information, visit our website or find us on LinkedIn and Twitter.

Forward Looking Statement

This press release contains forward looking statements, including statements regarding the timing of BESREMis availability in the United States, the commercialization plans and expectations for commercializing BESREMi in the United States, and the potential benefits or competitive position of BESREMi. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management 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 PharmaEssentias ability to launch BESREMi in the United States, whether BESREMi is successfully commercialized and adopted by physicians and patients, the extent to which reimbursement is available for BESREMi, and the ability to receive FDA and other regulatory approvals for additional indications for BESREMi. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. The information found on our website, and the FDA website, is not incorporated by reference into this press release and is included for reference purposes only.

1 Cerquozzi S, Tefferi A. Blast Transformation and Fibrotic Progression in Polycythemia Vera and Essential Thrombocythemia: A Literature Review of Incidence and Risk Factors. Blood Cancer Journal (2015) 5, e366; doi:10.1038/bcj.2015.95.

2021 PharmaEssentia Corporation. All rights reserved. US-BSRM-2100225 11/21

BESREMi and PharmaEssentia are registered trademarks of PharmaEssentia Corporation, and the PharmaEssentia logo and PharmaEssentia SOURCE are trademarks of PharmaEssentia Corporation.

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PharmaEssentia's BESREMi (ropeginterferon alfa-2b-njft) Now Available for the Treatment of People With Polycythemia Vera in the United States -...

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On International Day of Disabled Persons, TheHealthSite spoke to Dr. Sunil Bhat, Director and Clinical Lead, Pediatric Hematology, Oncology, and Blood & Marrow Transplantation, Mazumdar Shaw Cancer Centre, Narayana Health City, to discuss the condition and understand the ways one can manage it.

Written by Satata Karmakar | Updated : December 3, 2021 5:31 PM IST

3rd December every year is observed as UN-designated International Day for persons with disabilities. The observance of the Day aims to promote an understanding of disability issues and mobilize support for the dignity, rights, and well-being of persons with disabilities. This year's theme is "not all disabilities are visible" since some of the disabilities are non-visible but they cause significant challenges for people living with such conditions for day-to-day participation in society.

Such non-visible disabilities include some of the rare blood disorders such as Thalassemia, Aplastic Anemia, Sickle cell Anemia, Fanconi Anemia, Hemophilia, and so on. The Rights of People with Disability Bill passed by the Parliament of India in December 2016 included newer disabilities like blood disorders sickle cell anemia, thalassemia, and hemophilia. Today, on International Day of Disabled Persons, TheHealthSite spoke to Dr. Sunil Bhat, Director and Clinical Lead, Pediatric Hematology, Oncology, and Blood & Marrow Transplantation, Mazumdar Shaw Cancer Centre, Narayana Health City, to discuss the condition and understand the ways one can manage it.

In India, the burden of blood disorders and blood cancer is huge. India is even called as Thalassemia capital of the world with over 10,000 new cases every year. Thalassemia is a disabling condition not just because of chronic anemia but other co-morbidities like organ damage, bone damage, and cardiac complications.

People with thalassemia may need lifelong blood transfusions and other therapies (like iron removal medications). With the advances in the medical field, blood stem cell transplant plays an important role in the treatment of various blood disorders like thalassemia, aplastic anemia, and blood cancers as well. For a blood stem cell transplant to be deemed successful, the human leukocyte antigens (HLA) of the donor should match the antigens present in the cells of the patient. Only 30% of the patients find a matching donor in the family and the rest 70% depending on an unrelated donor. Such unrelated donors are being registered by stem cell registries like DKMS BMST Foundation India.

However, despite such a huge disease burden, Indian stem cell donors only form a tiny fraction, about 0.04% of the total listed unrelated donors globally. The main reason is the lack of awareness and prevailing myths around the stem cell donation process deny many patients a second chance at their lives in the country. It is high time that healthy people understand blood stem cell donation is a safe process and come forward to register as a donor. There is only a 1 in a million chance that someone comes as a match for a patient!

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Shahid Akhter, editor, ETHealthworld, spoke to Ramesh Ramadurai, MD, 3M India, to know more 3M collaborations that can improve and impact cancer care, besides cutting down on the costs in a big way.

How has the technological and infrastructural facilities impacted Bone Marrow Transplantation in India ?Every year nearly 20,000 Indian patients, including many children, who suffer from blood and solid cancers require bone marrow transplantation (BMT). However, only 2,000 of these patients are fortunate enough to receive this therapy, as the cost of bone marrow transplantation can vary from INR 10 Lakhs to 30 Lakhs. It is imperative to reduce the cost of cancer treatment while maintaining a stringent focus on sterilization and quality. It is indeed the need of the hour for us to address this issue.

What are the various technological advancement required for the better functioning of the facilities?Despite the increasing demand for bone marrow transplantation, the number of bone marrow doners in India is astonishingly low. India conducts stem cell transplant procedure for approximately 2,000 every year, while around 80,000 to 100,000 annual transplants are required to tackle the burden of blood cancers and fatal blood disorders. Finding a matching donor is very difficult. This option is exercised only after the alternative options have failed.

India has only about 400,000 donors registered on the bone marrow registry. Chances of finding a donor match are as low as 10% to 15% compared to the West where the chances of matching are as high as 60% to 70% due to higher rates of donations.

What are the major and significant developments in treating bone marrow cancer in the country?A bone marrow registry collects information on individuals willing and able to donate bone marrow and gathers the donor information into a database. In India, organisations like Datri are helping to create a pool of donors to help people who do not have blood-related donors by finding an unrelated match for life-saving treatment.

Infusion of a memory cell is another advancement. This involves taking out the cells, sorting the good cells and the memory cells or the fighting cells, which can fight infections, sorting them out, capturing them and putting them back into the body after giving the requisite chemotherapy. This is also called T-cell depletion with memory cell infusion. It is now available in India and is affordable.

Through this partnership with United Way Bengaluru and Sri Shankara Cancer Hospital and Research Centre (SSCHRC), how does 3M aim to foster accessibility for people from different sections of the society in treating cancer. 3M India was brought into the SSCHRC family through United Way of Bengaluru, and this is our second round of engagement with the hospital. Last year, 3M India had donated equipment for cancer research and diagnostics like the Sanger sequencer and QPCR, made enhancements to the childrens play area at the long-stay Lakshmi Childrens center with child-friendly wall graphics, and provided kitchen utensils and cooking counters for the resident families of paediatric cancer patients.

We have donated several critical equipments for the research labs which contribute to the successful treatment of the BMT patients. As on date the BMT unit at SSCHRC has treated and discharged 5 patients and currently 4 are undergoing treatment. This wing of the hospital is accessible by few staff nurses and specialists like Dr K N Nataraj who is the Chief of Adult and Paediatric Haematology at the hospital. For a successful bone marrow transplantation, there are several requisites, some of which include, successful donor matching, extremely technique-sensitive harvesting and transplantation processes and robust infection control. With this essential, life-saving equipment, the cost of the treatment will reduce to approximately 50% (between Rs 8-12 Lacs as against the actual cost of Rs 15- 30 lacs) and help the hospital treat many more cancer patients.

How do 3M India and Sri Shankara Hospital plan to take this initiative ahead in the future for the growth and enhancement of bone marrow transplantation in the facility? It is matter of pride for 3M India and United Way of Bengaluru that we are associated with SSCHRC, an institution at the forefront of providing comprehensive cancer treatment to the needy, through CSR interventions.

By complying with the Foundation for the Accreditation of Cellular Therapy (FACT) Guidelines, the BMT Centre will be a one-of-a-kind medical facility where people of all economic status can receive treatment. Being a growing facility, the hospital is committed to continuing its responsibility towards expansion of multiple hospital beds and medical care. We are inspired by the commitment of the doctors and Sri Shankara Board of Trustees, led by Dr. B.S. Srinath and other dedicated professionals who developed a multifaceted approach to establishment a state of the art, affordable cancer hospital that is accessible to all irrespective of caste, creed, religion, gender or socioeconomic status.

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Las Vegas, USAPolymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes , Dec. 08, 2021 (GLOBE NEWSWIRE) -- DelveInsights Polymyositis Pipeline Insight 2021 report offers exhaustive global coverage of available, marketed, and pipeline therapies in different phases of clinical development, major pharmaceutical companies working to advance the pipeline space, and future growth potential of the Polymyositis pipeline domain.

Some of the essential takeaways from thePolymyositis Pipelinereport:

Request a sample and discover more about the report offerings @ Polymyositis Emerging Therapies

The Polymyositis pipeline report lays down detailed profiles of the pipeline assets, comparative analysis of clinical and non-clinical stage Polymyositis products, inactive and dormant assets, comprehensive assessment of driving and restraining factors, as well as the opportunities and risks in the Polymyositis pipeline landscape.

Polymyositis Overview

Polymyositis is a type of inflammatory myopathy, which refers to a group of muscle diseases characterized by chronic muscle inflammation and weakness. Polymyositis (PM), an autoimmune disorder, develops due to abnormal activation of cytotoxic T lymphocytes (CD8 cells) and macrophages against muscular antigens as well as the strong extrafusal muscular expression of major histocompatibility complex 1 causing damage to the endomysium of skeletal muscles. Polymyositis develops gradually over time, and it rarely affects persons younger than age 18.

Find out more about the disease and recent developments @Polymyositis Pipeline Assessment

Polymyositis Pipeline Drugs

Learn more about the novel and emerging Polymyositis pipeline therapies @ Polymyositis Pipeline Analysis

Polymyositis Therapeutics Assessment

ThePolymyositis Pipelinereport proffers an integral view of the Polymyositis emerging novel therapies segmented by Stage, Product Type, Molecule Type, Mechanism of Action and Route of Administration.

By Product Type

By Stage

By Route of Administration

By Molecule Type

By Mechanism of Action

Scope of the Polymyositis Pipeline Report

Dive deep into rich insights for emerging therapies and assessment, visit @ Polymyositis Emerging Therapies

Table of Contents

For further information on the Polymyositis current pipeline therapeutics, reach out @ Polymyositis Ongoing Clinical Trials

Track and assess a candidates clinical development journey through Actionable Intelligence and Comparative Therapeutic Assessment

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SEATTLE, Nov. 18, 2021 /PRNewswire/ -- According to Latest Report, The global cell and gene therapy marketis estimated to account for 47,095.2 Mn in terms of value by the end of 2028.

Genetic mutations can lead to a wide range of serious malfunctions at the cellular level, including diseases such as cancer. These treatments use "living drugs" to repair damaged tissues and replace diseased organs, and they have the potential to cure a wide variety of ailments. In addition to regenerating damaged organs, cell and gene therapy can cure cancer, and the treatment process is fast-paced, with significant progress made in recent years. For the cell and gene therapy industry to reach its full potential, early interaction with payers and regulators is crucial. This will facilitate a fast-tracked clinical trial. While embracing new platform technologies is challenging, early collaboration with other industries will ensure a faster path to market for the new therapies. In addition to this, a play-to-win attitude is critical to success in this field. The success of gene and cell therapies will depend on achieving clinical and research goals.

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Market Drivers

1. Increasing incidence of cancer and other target diseases is expected to drive growth of the global cell and gene therapy market during the forecast period

With growing incidence of cancer and target diseases such as measles and tuberculosis, the adoption of gene and cell therapy has increased. According to the World Health Organization (WHO), in 2019, around 1.4 million people died from tuberculosis worldwide with around 10 million people being diagnosed with the same. According to the same source, in 2018, around 9.6 million died due to cancer with over 300,000 new cases of cancer being diagnosed each year among children aged 0-19 years across the globe. Gene therapy uses genes to treat or prevent disease, where it allows doctors to insert a gene into a patient's cells instead of using drugs or surgery. Therefore, it has the potential to completely treat genetic disorders.

2. Growing investments in pharmaceutical R&D activities are expected to propel the global cell andgene therapy market growth over the forecast period

Key pharmaceutical companies in the market are focused on research and development activities pertaining to gene therapy. Currently, gene therapy is being widely researched for various diseases including cancer, cystic fibrosis, hemophilia, AIDS, and diabetes. For instance, in November 2021, Sio Gene Therapies reported positive interim data for gene therapy trial of Phase I/II of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis, a genetic disorder that progressively destroys nerve cells in the brain and spinal cord.

Market Opportunity

1. Increasing demand for cell and gene therapies can present lucrative growth opportunities

The demand for cell and gene therapies is increasing with growing cases of genetic disorders, chronic diseases, etc. According to the Cystic Fibrosis Foundation (CFF), in the U.S., over 1,000 new cases of cystic fibrosis are diagnosed each year. Moreover, According to the WHO, the number of people with diabetes has increased from 108 million in 1980 to 422 million in 2014. According to the same source, in 2016, around 1.6 million deaths were directly caused due to diabetes. Cell and gene therapies have the potential to treat the aforementioned diseases.

2. Growing regulatory approval can provide major business opportunities

Key companies are focused on research and development activities, in order to gain regulatory approval and enhance market presence. For instance, in March 2021, Celgene Corporation, a subsidiary of Bristol Myers Squibb, received the U.S. Food and Drug Administration (FDA) approval for the first cell-based gene therapy Abecma indicated for the treatment of multiple myeloma.

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Market Trends

1. Stem cell therapy

In the recent past, stem cell therapies have gained significant importance across the healthcare sector. Stem cell therapy has the potential to treat tissue damage and have low immunogenicity. Furthermore, it can enhance the growth of new healthy skin tissues, improve collagen production, stimulate hair development after loss, and can be used in the treatment of various diseases including Parkinson's disease, Alzheimer's disease, cancer, spinal cord injury, etc.

2. North America Trends

Among regions, North America is expected to witness significant growth in the global cell and gene therapy market during the forecast period. This is owing to ongoing clinical trials combined with key companies focusing on R&D activities pertaining to cell and gene therapy. Moreover, the presence of key market players such as Thermo Fisher Scientific, Takara Bio Inc., Catalent Inc., and more are expected to boost the regional market growth in the near future.

Competitive Section

Major companies operating in the global cell and gene therapy market are Thermo Fisher Scientific, Merck KGaA, Lonza, Takara Bio Inc., Catalent Inc., F. Hoffmann-La Roche Ltd, Samsung Biologics, Wuxi Advanced Therapies, Boehringer Ingelheim, Novartis AG, and Miltenyi Biotec.

For instance, in July 2021, Minova Therapeutics Inc. entered into a collaboration and license agreement with Astellas Pharma Inc. for the research, development, and commercialization of novel cell therapy programs for diseases caused by mitochondrial dysfunction.

Global cell and gene therapy Market, By Region:

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Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights - PRNewswire

Spinal Cord Stem Cells Comments Off on Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights – PRNewswire | November 22nd, 2021

WHEN it comes to health, the news in recent times has been sombre.

It has been another rollercoaster year battling Covid, with the UK emerging from a third lockdown in spring.

13

Millions of people have since had their jabs, and boosters are being rolled out as winter looms large.

But Covid is not the only big health story to come out of the past two years.

Behind the scenes, scientists around the world have been working on medical trials in the hope of finding cures for major illnesses.

And there have been dozens of major breakthroughs that could save billions of lives and change the way diseases are treated forever.

Just this month it emerged the vaccine for the human papillomavirus virus (HPV) could eradicate cervical cancer within the next few years.

From asthma to Alzheimers and cancer to infertility, CLARE OREILLY looks at the new treatments, vaccines and medicines that could put an end to some of the most common and deadly conditions.

CERVICAL cancer kills more than two women every day in the UK, claiming around 850 lives every year.

Yet a new study has found the disease could soon be a thing of the past.

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Kings College London scientists found the human papillomavirus (HPV) vaccine cut cases by 90 per cent.

The jab, which was first rolled out to teenage girls in the UK in 2008 then to boys in 2019, prevents HPV, which is responsible for nearly all cases of cervical cancer.

The study, in the Lancet, tracked women who received some of the first doses and found it prevented an estimated 17,200 pre-cancers and 450 cases in women in their twenties.

Cancer Research UKs chief executive Michelle Mitchell said: Its a historic moment to see the first study showing that the HPV vaccine has and will continue to protect women from cervical cancer.

A NEW antibody-based treatment developed by scientists in the UK and Germany could soon yield a vaccine to prevent Alzheimers.

The degenerative condition is thought to be caused by a type of protein that sticks to brain cells.

13

The scientists were able to trigger the immune system to make antibodies, which targeted the protein before it was deposited.

Professor Mark Carr, who led a team at the University of Leicester, said: It has the real potential to provide an effective treatment for Alzheimers using a therapeutic antibody and highlights the potential of a simple vaccine.

Meanwhile, a year-long study has started in Norway where Alzheimers patients will receive a transfusion of blood taken from runners.

It is hoped the chemicals released in the blood after running have a rejuvenating effect to slow disease progression.

THREE new drugs are being put through trials in the hope they could end the misery of hot flushes for menopausal women.

Hot flushes are thought to be caused by changes in hormone levels affecting the bodys temperature control.

13

But the medicines fezolinetant, elinzanetant and pavinetant can block the receptors which are responsible for the common symptom.

London GP Dr Zoe Watson says it could be years before the treatment is available on the NHS, though.

She says: It looks interesting in theory, but there are question marks over its efficacy, its side-effect profile and its cost.

Certainly if it does this well then it could be extremely useful for women whose most troubling menopausal symptom is hot flushes.

However, menopause is much more than just hot flushes and halting periods."

A BRAND new injection could reverse spinal cord injuries and allow patients to walk again just four weeks after treatment.

Developed by a team at Northwestern University in the US, the jab encourages nerves to regrow.

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It gave paralysed mice the ability to walk and human trials are expected to begin next year.

For decades, this has remained a major challenge for scientists because our bodys central nervous system, which includes the brain and spinal cord, doesnt have any significant capacity to repair itself after injury.

Professor Samuel Stupp said: Our research aims to find a therapy that can prevent individuals from becoming paralysed after major trauma or disease.

We are going straight to the FDA [the US Food and Drug Administration] to get this approved for use in patients.

DEMENTIA affects around 850,000 people in the UK and costs 26.3billion a year, but scientists at Durham University have made a breakthrough.

They are working on a treatment that could boost memory and muscle control in patients with the killer disease.

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Using infrared light to zap the brain improved the memory and thought processing in trials of healthy people.

And the next step is to enlist dementia patients to test the therapy.

Its delivered by a specially equipped helmet, which beams invisible light waves into the brain and forces cells to boost levels, improving blood flow too.

Dr Paul Chazot, who led the study, said: While more research is needed, there are promising signs that therapy involving infrared light might also be beneficial for people living with dementia and this is worth exploring.

ANYONE with asthma knows how debilitating it can be to receive a diagnosis.

Yet more than five million people in the UK are asthmatic. But a brand new drug, already approved for use on the NHS, is set to transform the lives of many with the condition, making attacks less frequent and less severe.

13

Dupilumab is prescribed to treat eczema and rhinosinusitis a type of sinusitis where the nasal cavity as well as sinuses become inflamed.

Its from a family of drugs used to treat Covid.

Currently only patients with very serious asthma who have had at least four severe asthma attacks in the last year and are ineligible for other biological treatments will be considered for a prescription.

But the drug is set to change the lives of many asthma sufferers across the country.

HALF of us will get cancer at some point in our lives. But new jab Survivin could change the landscape dramatically, scientists say.

The first clinical trials are already under way, and the injection works to boost the bodys immune system. It supercharges the immune cells, helping them seek out and destroy cancerous cells while leaving healthy cells alone.

13

Currently there are 36 terminally ill patients taking part in the trials, which are focused on ovarian, prostate and lung cancers.

Michelle Mitchell, chief executive of Cancer Research UK, said: Just this month we heard the HPV vaccine has likely prevented hundreds of women from developing cervical cancer.

This is a new and exciting frontier in cancer medicine and if this trial and others are successful, we could see thousands more lives saved.

AROUND seven per cent of all men are affected by infertility.

And while treatments currently focus on solutions rather than cures, scientists at the University of Georgia, in the US, are looking to reverse male infertility altogether.

13

The researchers have used primate embryonic stem cells the building blocks of all cells in the body to grow sperm cells in the earlier stages of development in a petri dish.

These spermatids, which lack a head and tail for swimming, were capable of fertilising a rhesus macaque egg in vitro.

Lead researcher and associate professor Dr Charles Easley says: This is a major breakthrough towards producing stem cell-based therapies to treat male infertility in cases where the men do not produce any viable sperm cells.

It is the first step that shows this technology is potentially translatable.

GETTING through the blood/brain barrier to target treatments for brain cancer is complex.

But now a team of scientists in Toronto, Canada, have found a way to use ultrasound beams.

13

They help open the barrier and can help facilitate drug delivery, which could change the way the disease is treated.

A trial this year saw four women with breast cancer that had spread to their brains treated with magnetic resonance-guided focused ultrasound (MRgFUS).

It allowed the antibody therapy herceptin to pass into their brain tissue, and caused the tumours to shrink without damaging any healthy tissue.

Dr Nir Lipsman, who led the study, said: It has long been theorised that focused ultrasound can be used to enhance drug delivery, but this is the first time we have shown we can get drugs into the brain.

A DRUG taken in pill form is to be trialled to combat the deadliest form of cancer.Auceliciclib is already used to treat brain tumours.

But now scientists hope it can help fight pancreatic cancer, which is often first diagnosed when it is at a late stage.

13

Professor Shudong Wang and her team at the University of South Australia are also working on new ways to detect the disease.

She said: Pancreatic cancer is extremely difficult to diagnose at an early stage because there are very few symptoms.

If it is caught early the malignant tumour can be surgically removed, but once it spreads into other organs it is lethal.

Chemotherapy and radiotherapy only buy patients a little extra time.

The team hopes the drug will be more effective and with fewer side-effects than current treatment options.

THE heroic scientists who developed the Covid vaccine did not stop there.

The team at the University of Oxford has also developed a malaria jab that will save billions of lives.

13

A trial showed 77 per cent of volunteers who were vaccinated stayed malaria-free over the following 12 months.

More than 100 malaria vaccines have been developed in recent decades, but the Oxford jab is the first to have such a high success rate.

Halidou Tinto, professor of parasitology and the principal investigator on the trial, said: These are very exciting results showing unprecedented efficacy levels from a vaccine that has been well-tolerated in our trial programme.

We look forward to the upcoming phase III trial to demonstrate large-scale safety and efficacy data for a vaccine that is greatly needed.

A DRUG that repairs cancerous cells could revolutionise the way breast cancer is treated.

Patients given olaparib as part of a two-and-a-half year trial were 42 per cent less likely to see their cancer return.

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There was also a 43 per cent dEcrease in the risk of the disease spreading.

Until the breakthrough earlier this year, the drug was mainly used for late-stage cancers, but the new findings suggest it is effective as an early treatment.

Professor Andrew Tutt, professor of oncology at the Institute of Cancer Research who led the study, said: Women with early-stage breast cancer who have inherited BRCA1 or BRCA2 mutations are typically diagnosed at a younger age.

Up to now, there has been no treatment that specifically targets the unique biology of these cancers to reduce the rate of recurrence, beyond initial treatment such as surgery.

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From asthma to cancer to infertility, the new treatments, jabs and meds making us healthier... - The Sun

Spinal Cord Stem Cells Comments Off on From asthma to cancer to infertility, the new treatments, jabs and meds making us healthier… – The Sun | November 22nd, 2021

PRC Clinical to Provide Start-up CRO Services for BRTX-100 Phase 2 Clinical Trial

MELVILLE, N.Y., Nov. 19, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company" or BioRestorative) (NASDAQ:BRTX), a life sciences company focused on adult stem cell-based therapies, today announced that it has entered into a letter of intent with PRC Clinical, a CRO specializing in clinical trial management, with regard to PRC Clinical providing startup clinical project management activities for the Companys BRTX-100 Phase 2 clinical trial to treat chronic lumbar disc disease.

We are pleasedto announce that we have entered into a letter of intent for PRC Clinical to provide startup activities for our Phase 2 study. PRC has extensive experience and expertise in managing clinical studies in the stem cell and regenerative medicine space. They also have theexperienced and professionalnetwork of clinicians and study sites streamlining patient enrollment, site monitoring and management. Additionally, we have been working with and familiarizing ourselves with PRCs team and capabilities since 2019. We are thrilled to finally be in a position to begin the process of validating our technology through the FDA process, while keeping shareholders updated along the regulatory pathway, said Lance Alstodt, CEO of BioRestorative.

PRC Clinical has provided specialty CRO services for nearly 20 years. Their innovative approach to executing studies for biotech and pharmaceutical companies combines high-touch human elements and cutting-edge technology with extensive experience and deep therapeutic knowledge. PRC Clinical is an all inclusive CRO and has specialized expertise across regenerative medicine, CNS, ophthalmology, pulmonary and COVID-19, rare and orphan disease and more complex indications.

PRC Clinical is pleased to begin start-up CRO activities for BRTX-100. We look forward to being able to bring our stem cell experience to this trial. We are committed to supporting BioRestoratives development of BRTX-100 and its clinical application, said Curtis Head, CEO of PRC Clinical.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:

Email: ir@biorestorative.com

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BioRestorative Therapies Enters into Letter of Intent with PRC Clinical - GlobeNewswire

Bone Marrow Stem Cells Comments Off on BioRestorative Therapies Enters into Letter of Intent with PRC Clinical – GlobeNewswire | November 22nd, 2021

Jack Stevens was diagnosed with juvenile myelomonocytic leukaemia and underwent stem cell treatment but has sadly lost his battle as his father's former clubs pay tribute

The young son of former England footballer Gary Stevens has died tragically aged four.

Stevens son Jack was diagnosed with a rare form of blood cancer.

Just a year ago his family were hopeful of a recovery after Jack underwent a stem cell transplant following his diagnosis with juvenile myelomonocytic leukaemia.

The stem cells Jack received from older brother Oliver had engrafted, meaning they had entered Jacks bone marrow, enabling him to produce his own white blood cells.

The transplant procedure was brought forward after Jack responded to two rounds of preparatory chemotherapy, negating an original plan for five cycles of treatment.

He was discharged to a nearby apartment and during the week lived with mum Louise.

Stevens a brilliant right-back in Howard Kendalls magical mid-1980s Everton team and the couples other sons, Oliver and Josh, would join Jack and Louise at weekends.

This morning Everton said: "Everyone at Everton is deeply saddened to learn that Gary Stevens four-year-old son, Jack, has passed away following his courageous battle with leukaemia.

"Our thoughts are with Gary and his family at this incredibly sad time."

Image:

Stevens, 59, began his football career with Everton, representing the Toffees for six seasons and making over 200 appearances between 1982-1988.

He then transferred to Glasgow Rangers where he played a part in their treble-winning season in 1993.

After another six-year stint, he returned to Merseyside to finish his career plying his trade for Tranmere Rovers.

Stevens earned 46 caps for England during his playing days before moving into physiotherapy after his retirement.

Tranmere shared Everton's post and issued their own touching message, which read: "The thoughts of everyone at Tranmere Rovers are with Gary Stevens and his family at this sad time."

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Former England and Everton footballer Gary Stevens' son tragically dies aged four - Mirror.co.uk

Bone Marrow Stem Cells Comments Off on Former England and Everton footballer Gary Stevens’ son tragically dies aged four – Mirror.co.uk | November 22nd, 2021

November 19, 2021 Stem cell therapy helped to reduce the number of heart attacks, strokes and death among people with chronic, high-risk, NYHA class II or III heart failure with reduced ejection fraction (HFrEF), especially among those who have higher levels of inflammation, yet hospitalization was not reduced, according to late-breaking research presented at the American Heart Associations Scientific Sessions 2021.

Heart failure is a condition when the heart is unable to adequately pump blood to meet the bodys need for oxygen and nutrients. In heart failure with reduced ejection fraction (HFrEF), the heart muscle enlarges and weakens, resulting in a decrease in pumping ability and fluid buildup in the bodys tissues. Inflammation plays a significant role in the progression of heart failure over time.

This study set out to examine the effects of using stem cells (mesenchymal precursor cells) injected into the heart to target inflammation and treat chronic heart failure. Researchers hypothesized that a single injection of stem cells from healthy adult donors in addition to guideline-directed medical therapy (GDMT) for heart failure would affect the number of times participants were hospitalized for heart failure events and reduce heart attacks, strokes, and/or death.

Cell therapy has the potential to change how we treat heart failure, said Emerson C. Perin, M.D., Ph.D., the studys lead author, the director of the Center for Clinical Research and medical director of the Texas Heart Institute in Houston. This study addresses the inflammatory aspects of heart failure, which go mostly untreated, despite significant pharmaceutical and device therapy development. Our findings indicate stem cell therapy may be considered for use in addition to standard guideline therapies.

The Randomized Trial of Targeted Transendocardial Delivery of Mesenchymal Precursor Cells in High-Risk Chronic Heart Failure Patients with Reduced Ejection Fraction study also called the DREAM-HF trial, is the largest stem cell therapy study to date among people with heart failure. In this multi-center, randomized, sham-controlled, double-blind trial, researchers enrolled 537 participants (average age 63, 20% female) with heart failure and reduced ejection fraction, which is when the left side of the heart, its main pumping chamber, is significantly weakened.

Heart failure was defined using the New York Heart Association (NYHA) functional classification system. This classification system places patients in one of four categories based on how much they are limited during physical activity. Class I heart failure means no limitation of physical activity, with class IV heart failure meaning an inability to have any physical activity without discomfort.

Participants were randomly divided into two groups: 261 adults received an injection of 150 million mesenchymal precursor cells, commonly known as stem cells, directly into the heart using a catheter. The remaining 276 adults received a scripted, or sham, procedure. Healthy adult donors provided the mesenchymal precursor cells.

The study participants were discharged from the hospital the day after the procedure, and researchers followed these participants for an average of 30 months. The studys focus was to examine if the stem cell treatment affected the likelihood of participants returning to the hospital for treatment of worsening heart failure. They also tracked whether participants had a heart attack or stroke, or died, and measured levels of high-sensitivity C-reactive protein (CRP), a measure in the blood indicating inflammation.

While researchers did not see a decrease in hospitalizations due to the stem cell treatment, they did notice several other significant results. The findings include:

We were impressed to learn that stem cell treatment effects were additive to current standard heart failure treatments, Perin said. For the first time, the known anti-inflammatory mechanism of action of these cells may be linked to a cause-and-effect benefit in heart failure. The stem cells acted locally in the heart, and they also helped in blood vessels throughout the body.

Perin and colleagues believe further research is needed to better understand how these stem cells may affect the course of progression of heart failure and how these therapies may be directed to the patient groups that could see the most benefits.

Limitations to the research include the selection of endpoints commonly used in heart failure studies. The studys results suggest that traditional endpoints associated with recurrent heart failure hospitalization do not fully reveal the benefits or mechanisms of these stem cells on heart attack, stroke and death in patients with chronic heart failure.

Co-authors are Barry H. Greenberg, M.D.; Kenneth M. Borow, M.D.; Timothy D. Henry II, M.D.; Farrell O. Mendelsohn, M.D.; Les R. Miller, M.D.; Elizabeth Swiggum, M.D.; Eric D. Adler, M.D.; Christopher A. James, P.A.; and Silviu Itescu, M.D. Authors disclosures are listed in the abstract.

The study was funded by Mesoblast Inc.

https://www.dicardiology.com/article/late-breaking-science-presentations-aha-2021-meeting

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Stem Cell Therapy for Heart Failure Reduced Major Cardiac Events and Death - Diagnostic and Interventional Cardiology

Cardiac Stem Cells Comments Off on Stem Cell Therapy for Heart Failure Reduced Major Cardiac Events and Death – Diagnostic and Interventional Cardiology | November 22nd, 2021

DUBLIN--(BUSINESS WIRE)--The "Induced Pluripotent Stem Cell (iPSC) Market Share, Size, Trends, Industry Analysis Report By Application (Manufacturing, Academic Research, Drug Development & Discovery, Toxicity Screening, Regenerative Medicine); By Derived Cell; By Region, Segment & Forecast, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

The global Induced Pluripotent Stem Cell (iPSC) market size is expected to reach $2,893.3 million by 2028

The ability to model human diseases in vitro as well as high-throughput screening has greatly propelled market growth. Companies have effectively overcome market hurdles faced in the recent past such as proper culturing and differentiation of derived cells at a commercial scale and have developed state-of-the-art manufacturing processes that can achieve scalability and can achieve stringent quality parameters. Such trends are propelling the overall industry growth.

Companies have also developed advanced platforms for Induced pluripotent stem cells that guarantee close connection with a host of in-house technologies that are useful in the proper definition of disease signatures as well as relationships between genetic mutations as well as that properly describe perturbation of specific molecular pathways. This has resulted in the creation of human translational models that are aiding better target identification of diseases that have high unmet medical needs.

Many companies have developed transfection kits, reprogramming vectors, differentiation media, live staining kits, immunocytochemistry, among others to aid the smooth workflow of iPSC production.

However, it has been observed in the recent past that the demand for cells for screening and other purposes is significant and that there are significant challenges that pose a significant hurdle in large-scale iPSC production and differentiation.

Heavy investment in R&D activities pertaining to the development and optimization of iPSC reprogramming process in order to achieve sufficient production is a key industry trend. In the recent past, companies focused more on hepatic, cardiac, pancreatic cells, among others.

However, with the advent of a number of new participants as well as advancements and breakthroughs achieved, it is anticipated that the application portfolio will further increase in the near future.

Industry participants operating in the industry are:

Key Topics Covered:

1. Introduction

2. Executive Summary

3. Research Methodology

4. iPSC Market Insights

4.1. iPSC - Industry Snapshot

4.2. iPSC Market Dynamics

4.2.1. Drivers and Opportunities

4.2.1.1. Increasing demand for body reconstruction procedures and tissue engineering

4.2.1.2. Rising Investments across the globe

4.2.2. Restraints and Challenges

4.2.2.1. Scalability Issues

4.3. Porter's Five Forces Analysis

4.4. PESTLE Analysis

4.5. iPSC Market Industry trends

4.6. COVID-19 Impact Analysis

5. Global iPSC Market, by Derived Cell

5.1. Key Findings

5.2. Introduction

5.3. Hepatocytes

5.4. Fibroblasts

5.5. Amniotic Cells

5.6. Cardiomyocytes

6. Global iPSC Market, by Application

6.1. Key Findings

6.2. Introduction

6.2.1. Global iPSC Market, by Application, 2017 - 2028 (USD Million)

6.3. Manufacturing

6.4. Academic Research

6.5. Drug Development & Discovery

6.6. Toxicity Screening

6.7. Regenerative Medicine

7. Global iPSC Market, by Geography

7.1. Key findings

7.2. Introduction

7.2.1. iPSC Market Assessment, By Geography, 2017 - 2028 (USD Million)

8. Competitive Landscape

8.1. Expansion and Acquisition Analysis

8.1.1. Expansion

8.1.2. Acquisitions

8.2. Partnerships/Collaborations/Agreements/Exhibitions

9. Company Profiles

9.1. Company Overview

9.2. Financial Performance

9.3. Product Benchmarking

9.4. Recent Development

For more information about this report visit https://www.researchandmarkets.com/r/ykewbe

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Global Induced Pluripotent Stem Cell (iPSC) Market Report 2021-2028 - Increasing Demand for Body Reconstruction Procedures and Tissue Engineering -...

Cardiac Stem Cells Comments Off on Global Induced Pluripotent Stem Cell (iPSC) Market Report 2021-2028 – Increasing Demand for Body Reconstruction Procedures and Tissue Engineering -… | November 22nd, 2021