CytomX Therapeutics to Present at Guggenheim Healthcare Talks 2021 Oncology Day
By Dr. Matthew Watson
SOUTH SAN FRANCISCO, Calif., Feb. 04, 2021 (GLOBE NEWSWIRE) -- CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage, oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody® technology platform, today announced that Sean McCarthy, D.Phil., president, chief executive officer, and chairman, will participate in a virtual fireside chat at the Guggenheim Healthcare Talks 2021 Oncology Day on February 11th at 3:30 p.m. ET. In addition, management will be available for one-on-one meetings with investors.
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CytomX Therapeutics to Present at Guggenheim Healthcare Talks 2021 Oncology Day
Gain Therapeutics Announces Three Late-Breaker Presentations at the 17th Annual WORLDSymposium
By Dr. Matthew Watson
BETHESDA, Md., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (“Gain”) a biotechnology company focused on redefining drug discovery by identifying and optimizing allosteric binding sites that have never before been targeted, today announced that it will present three late-breaker abstracts at the 17th annual WORLDSymposium, a research conference dedicated to lysosomal diseases being held virtually February 8-12, 2021. The poster presentations will highlight data supporting Gain’s Gaucher disease, GM1 gangliosidosis and Morquio B programs.
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Gain Therapeutics Announces Three Late-Breaker Presentations at the 17th Annual WORLDSymposium
Replimune Reports Third Fiscal Quarter Financial Results and Provides Corporate Update
By Dr. Matthew Watson
RP1: Initial data in new indications expected in 2021 in anti-PD1 failed NSCLC, anti-PD1 failed CSCC and CSCC solid organ transplant recipient patients; further updates expected to be provided across all studies
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Replimune Reports Third Fiscal Quarter Financial Results and Provides Corporate Update
Turning Point Therapeutics to Participate in Guggenheim Healthcare Talks 2021 Oncology Days
By Dr. Matthew Watson
Company to Participate in Two Q&A Sessions, Including a Joint Discussion with Zai Lab Company to Participate in Two Q&A Sessions, Including a Joint Discussion with Zai Lab
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Turning Point Therapeutics to Participate in Guggenheim Healthcare Talks 2021 Oncology Days
Agenus Announces Positive Preliminary Results of iNKT Cell Therapy Trial in COVID-19
By Dr. Matthew Watson
LEXINGTON, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, today announced positive preliminary results from its Phase 1 trial of iNKT cell therapy in patients with moderate to severe symptoms of COVID-19 through its subsidiary, AgenTus Therapeutics.
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Agenus Announces Positive Preliminary Results of iNKT Cell Therapy Trial in COVID-19
Outlook Therapeutics’ Recent Financing Secures Funding to Support ONS-5010 / LYTENAVA™ (bevacizumab-vikg) Through Planned BLA Submission
By Dr. Matthew Watson
Overallotment option on recent public offering partially exercised by underwriter
Intrommune Receives IND Clearance From U.S. Food and Drug Administration for INT301
By Dr. Matthew Watson
Milestone Marks Advance for Oral Mucosal Immunotherapy for Peanut Allergies Milestone Marks Advance for Oral Mucosal Immunotherapy for Peanut Allergies
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Intrommune Receives IND Clearance From U.S. Food and Drug Administration for INT301
Alphageneron Pharmaceuticals Signed Exclusive License Agreement With Multimmune GmbH For Natural Killer Cell Platform Therapy Worldwide
By Dr. Matthew Watson
CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Alphageneron Pharmaceuticals, Inc., (Alphageneron) signed an Exclusive License Agreement with Multimmune GmbH, of Munich, Germany, (Multimmune) on October 1st, 2020, for its clinical stage autologous Natural Killer (“NK”) cell therapy and companion diagnostic platform, for all fields worldwide.
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Alphageneron Pharmaceuticals Signed Exclusive License Agreement With Multimmune GmbH For Natural Killer Cell Platform Therapy Worldwide
AGTC Executives Awarded First Place in the BioProcess International Reader’s Choice Awards, Cell & Gene Therapies Category
By Dr. Matthew Watson
Article reflects Company’s leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies
Monthly information related to total number of voting rights and shares composing the share capital – January 31, 2021
By Dr. Matthew Watson
To Read More: Monthly information related to total number of voting rights and shares composing the share capital – January 31, 2021PRA Health Sciences to Report Fourth Quarter and Full Year 2020 Earnings
By Dr. Matthew Watson
RALEIGH, N.C., Feb. 04, 2021 (GLOBE NEWSWIRE) -- PRA Health Sciences, Inc. (NASDAQ: PRAH) will release its fourth quarter and full year 2020 results after the market closes on Thursday, February 25, 2021. The Company will also host a conference call on Friday, February 26, 2021 at 9:00 a.m. (ET) to discuss the results with members of the investment community.
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PRA Health Sciences to Report Fourth Quarter and Full Year 2020 Earnings
Stem cells efficacy confirmed in treating ototoxic hearing loss – Korea Biomedical Review
By daniellenierenberg
Researchers at the Catholic University of Korea St. Marys Hospital have recently proved the efficacy of bone marrow-derived stem cells to treat ototoxicity hearing loss, the hospital said Thursday.
The team, led by Professor Park Kyoung-ho of the Department of Otolaryngology, conducted an experiment on animal models with ototoxic sensorineural hearing, or sudden hearing loss.
They utilized Catholic MASTER cells, bone marrow stem cells developed by the Catholic Institute of Cell Therapy, to compare the stem cell injection group with the controlled group.
The result showed that animals started to recover their hearing after three weeks. Five weeks later, they recovered normal hearing at 8000Hz, 16000Hz and 32000Hz frequency.
Ototoxic hearing loss is caused when a person ingests chemicals or certain medications that adversely affect the inner ear functions. Major symptoms related to the illness are dizziness, false hearing, and hearing loss, which permanently defects hearing functions. Elders with such symptoms should have medical consultations as they are a high-risk group, the hospital said.
We have proved the efficacy of our bone marrow stem cells in recovering hearing, said Professor Park, who doubles as the director of the Stem Cell Institute. Through the results, we expect to provide new treatment opportunities for patients with hearing loss.
The test results were published in the Korean Journal of Otorhinolaryngology-Head and Neck Surgery.
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Stem cells efficacy confirmed in treating ototoxic hearing loss - Korea Biomedical Review
Understanding bone marrow transplant: The guidelines and the protocols – The New Indian Express
By daniellenierenberg
The outbreak of the Covidpandemic has made many patients reluctantto undergotreatments. While their apprehension seems to overpower them, doctors need to ensure thatstrict guidelines and protocols which assure the best quality service are followed.
Among elective surgeries andtransplants, bone marrow transplant cases have increased substantially in the past few months. Adhering to guidelines for pre-transplant evaluation and the management of a common complication, graft versus host disease (GVHD)is essential.
With the diversity of practice and expertise, the following guidelines will provide a pivotal tool for learning about the rapidly updated therapy landscape in Hematopoietic stem cell transplantation (HSCT).
The guidelines intended to provide a systematic approach for transplantation and help streamline clinical practices and educate new generations of physicians-in-training. Additionally, guidelines can help to evaluate a potential transplant recipient anddetermine if the patient is an eligible candidate for the procedure.
Types and selection of transplantation:
Selection of the type of transplantation for a patient depends on factors such as the type of malignancy, availability of a suitable donor, age of the recipient, the ability to collect a tumor-free autograft, the stage, the malignancy's susceptibility to the GVM effect, and status of disease -- bone marrow involvement, the bulk of disease, chemosensitivity to conventional chemotherapy. This method is particularly applicable for Autologous or Allogeneic Transplantation where one can have a sibling donor or a matched unrelated donor. In the case of a matched unrelated donor, ensure that the collection is adequate and stem cells are available well in time especially if they are imported from countries in Europe.
A haploidentical transplant is another type of transplant that uses healthy, blood-forming cells from a half-matched donor to replace the unhealthy ones. The ideal donor in this case is a family member.
That said, for bone marrow transplant blood products are the backbone and it is important to ensure to have adequate supply before you begin with the transplant.
What are the guidelines and protocols that can be adopted in current times?
Some measures for consideration are: Minimize face-to-face visits including monitoring and consider shifting to telehealth where feasible. Some adaptive community measures like the hospital in the home services, community practices for blood collection, imaging, and support services. For radiation oncology treatment, consider reducing fractions when supported by evidence Consider alternative and less resource-intensive treatment regimes. Minimize unnecessary visitors to cancer centers, for instance, limiting to only patients and their essential caregivers based on frailty and language needs Screen for possible symptoms of COVID-19 and triage patients for admission. If necessary, the admission has to be directed to oncology/hematology departments rather than emergency departments. Immunocompromised patients are likely to have atypical presentations of COVID-19 For suspected checkpoint inhibitor-related pneumonitis prioritizes COVID-19 testing for an early decision regarding corticosteroid therapy.
These are some guidelines that you should heed during a bone marrow transplant. While it is imperative to be updated about the guidelines, timely intervention can reduce the other possible complications during the process.
(The author is the Director, Medical Oncology and Hemato Oncology, atFortis Cancer Institute, Bangalore)
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Understanding bone marrow transplant: The guidelines and the protocols - The New Indian Express
World Cancer Day 2021: DKMS Announces The Milestone Of Giving 90,000 Blood Cancer Patients Worldwide A Second Chance At Life – PR Newswire India
By daniellenierenberg
- DKMS-BMST continues to urge Indians to step up to be a potential lifesaver!
BENGALURU, India, Feb. 4, 2021 /PRNewswire/ -- February 4th is marked as World Cancer day every year. This day is observed to spread awareness about the disease and its increasing burden. In line with the theme for this year "Create a futurewithout cancer. The time to act isnow", DKMS BMST Foundation India, a non-profit organization with a mission to provide a second chance at life to blood cancer and blood disorder patients in India makes an appeal to people to come forward and register as potential blood stem cell donors.
On World Cancer Day 2021, DKMS is also celebrating a milestone of providing more than 90,000 blood cancer patients across 57 countries with a second chance at life, since it was founded almost 30 years ago in 1991. DKMS is an international non-profit organization that helps provide patients with lifesaving blood stem cell transplants. DKMS has presence in India, Germany, USA, Poland, UK, Chile, and South Africa.
Patrick Paul, CEO, DKMS BMST Foundation India, says, "DKMS is proud to be the world's leading donor center, accounting for nearly 30% of the total donor pool. While, this is a global milestone, when it comes to India, the fact is that the Indian donors are highly underrepresented in the global database. This is why it becomes difficult for doctors to find a matching blood stem cell donor for Indian patients. While DKMS has registered over 10.5 million donors and has provided over 90,000 patients with a second chance at life globally, it is critical to highlight that only over 43,000 Indian donors are part of this donor pool."
In India, every year, over one lakh people are diagnosed with a form of blood cancer and it remains one of the leading causes of cancer-related deaths among children. Most people are unaware that a life-threatening disease like blood cancer can be treated and in most of the cases, a stem cell transplant is the patient's only chance for survival. For instance, 15-year-old Maheer from Gujarat, India, is one of the blood cancer survivors who had received a lifesaving blood stem cell donation in 2012. He was able to find his matching blood stem cell donor, Dr. Sita, who hails from Germany. Today, he leads a normal, healthy and happy life. He is in grade 9 and loves to travel, read and swim.
Today, more than 37 million potential unrelated donors are listed worldwide with stem cell donor centers and registries, of which only 0.03% are Indians. Currently, in India, the biggest challenge is the lack of awareness about blood stem cell transplant and the importance of registering as a potential blood stem cell donor. The entire procedure is safe and secure. Once the blood stem cells are collected from a donor, they are infused into the patient through a transplant process which then moves through the bloodstream and settles in the bone marrow. These new blood stem cells begin to increase in numbers and produce red blood cells, white blood cells, and platelets, resulting in the replacement of the patient's diseased cells and that's how a blood cancer patient gets a second chance at life. This situation can only be improved by recruiting many more potential stem cell donors from India.
This World Cancer Day, one can take a pledge to become a potential lifesaver. Registration takes only 5 minutes. If one between 18 and 50 years and in good health, the first step to register as a blood stem cell donor by ordering the home swab kit at http://www.dkms-bmst.org/register.
SOURCE DKMS BMST Foundation India
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World Cancer Day 2021: DKMS Announces The Milestone Of Giving 90,000 Blood Cancer Patients Worldwide A Second Chance At Life - PR Newswire India
Man who wants to live for 180 years spends Rs 18 lakh to re-inject his own stem cells – Times Now
By daniellenierenberg
Dave Asprey  |  Photo Credit: Twitter
A man who is determined to live until he is 180 years old says that his bizarre methods will soon be as popular as mobile phones. American millionaire tech entrepreneur Dave Asprey, 47, believes he will live to the year 2153 'at least' by using techniques such as sitting in a cold cryotherapy chamber and intermittent-fasting.
Dave coined the term 'Biohacking' to describe his methods of turning back the biological clock.
He got parts of his bone marrow removed to have the stem cells injected back into his body for $25,000 (Rs 18 lakh).
He speculated that people who are under 40 years of age will be "happy and highly functional" at 100 after applying his methods.
When This Morning's Holly Willoughby asked him why he wants to live so long, he replied, "I'm curious, I think there's a lot of things we can fix and improve in the world and I don't feel like I'm at all done yet."
Dave believes that he won't be the only one to be live for so long.
"The things I am working to pioneer, some of them are expensive, some of them are free like fasting. This will be like cell phones, everyone has cell phones - everyone will have anti-ageing. Change can happen rapidly in society," he said. "There will be many people who are under 40 right now who [will be] walking around under their own power, perfectly happy, highly functional, who are more than 100 years old."
Dave has spent an estimated $1 million on techniques and hacks to try and improve his body's overall functioning.
He added, "I set myself up to have much less inflammation than most people do, by controlling what I eat and how I sleep and a lot of other anti-ageing treatments."
He explained why he re-introduced his stem cells in his body, saying, "When we're young, we have a ton of stem cells and we heal like young people. As we age our stem cells get exhausted, so I do things like intermittent fasting which give me more stem cells and then I take my own stem cells and move them around the body so I heal and move like a young person."
Dave also believes in the benefits of cryotherapy, also known as cold therapy, which is the use of low temperatures in medical therapy to treat a variety of tissue lesions. He has been having cold showers for over ten years.
Another technique that Dave uses to live a long life is intermittent fasting, which involves restricting times that you eat meals to create periods of fasting over a certain period.
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Man who wants to live for 180 years spends Rs 18 lakh to re-inject his own stem cells - Times Now
Hematopoietic Stem Cell Transplantation (HSCT) Market Incredible Possibilities, Growth With Industry Study, Detailed Analysis And Forecast To 2027 …
By daniellenierenberg
Up Market Research (UMR) recently published a report entitled, the Hematopoietic Stem Cell Transplantation (HSCT) Market, describing the crucial aspects of the market by conducting an in-depth analysis of the current trend, emerging threats, and future market assessment. This report takes into account the adverse impact of the COVID-19 pandemic on the market for the period of 2020-2020 and provides a detailed information about how the market will perform during the forecast period, 2020-2027. Our research team presents the report in a simplistic manner supported by fact and actual figures that will assist clients to arrive an informed decision about their investment plans and business strategies.
Request Free Exclusive Sample on Hematopoietic Stem Cell Transplantation (HSCT) Market Report @ https://www.upmarketresearch.com/home/requested_sample/30593
The report provides a holistic view of the market offering key insights of the market behavior over last four years and key assessment of the future market performance. It includes a systematic analysis of historical data for the period, 2015-2020 and draws upon assessment of the market performance for the forecast period, 2020-2027 by observing 2020 as the base year. With the reference to the available data, it provides vital insights on key factors such as drivers, restrains, trends, challenges, and opportunities of the Hematopoietic Stem Cell Transplantation (HSCT) market growth.
This report provides a comprehensive outlook on the key segments and sub-segmentations that includes the complete information about the product types, applications, end-users and regions. It offers latest information regarding the growth rate, volume, and size of the market in respect to each segment and also explains the market performance of these segments in the respective five regions. Moreover, it discusses a wide range of the emerging market scope and potential drawbacks present in the segments.
Hematopoietic Stem Cell Transplantation (HSCT) Market Report Includes:
The Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report is segmented into:
By Types:
AllogeneicAutologous
By Applications:
Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)
By Regions:
For More Information on This Report Visit: https://www.upmarketresearch.com/home/enquiry_before_buying/30593
The report covers the competitive landscape of various major global players, their current market positions, and key business strategies adopted to mark their major footprint in the market. This includes information about the product launch, expansion of the production facilities or plants, adoption of new technologies, latest merger & acquisition, partnership, and collaboration of the key players. It furthers provides concrete information about the existing market scope for the new entrants and the current competitive levels and scenario for the emerging players in the global market.
The Hematopoietic Stem Cell Transplantation (HSCT) Market Report Covers the Following Companies:
Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord I
Up Market Research (UMR) also offers customized report for a particular product, application, and region as per the requirement of clients and provides additional companies profiles as per the clients request.
Regarding the methodology, the report is prepared by relying on primary and secondary sources including interviews of the company executives & representatives and accessing official documents, websites, and press release of the companies related to the Hematopoietic Stem Cell Transplantation (HSCT) market. It also includes comments and suggestions from the experts in the market especially the representatives from government and public organizations as well as international NGOs. The report prepared by Up Market Research (UMR) is known for its data accuracy and precise style, which relies on genuine information and reliable data source. Moreover, customized report can be available as per the clients wishes or specific needs. It takes into account of various research tools and methods including predictive analysis, Porters 5 force analysis, SWOT analysis, and real-time analytics.
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This report includes the estimation of market size in terms of value (USD) and volume (K MT), with applying top-down and bottom-up approaches to estimate and validate the overall scope of the market. The report is presented with a group of graphical representations, tables, and figures that shows a clear picture of the developments of the products and its market performance over the last few years. With this precise report, it can be easily understood the growth potential, revenue growth, product range, and pricing factors related to the Hematopoietic Stem Cell Transplantation (HSCT) market. This report includes information on the latest government policies, norms, and regulations that can affect the dynamics of the market.
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Harnessing the Potential of Cell and Gene Therapy – OncLive
By daniellenierenberg
Excitement took wing in the scientific community in the early 1990s, when the first gene therapy trial showed significant success, only to crash at the end of the decade with a patients tragic death.
Twenty years later, the excitement is back and greater than before. Although safety remains a concern, investigators are breaking ground in cell and gene therapy, and many believe that ultimately, a string of cured cancers will follow.
In 2017, the excitement over these therapies returned in spades when the FDA signed off on a cell-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for patients with B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.
Were entering a new frontier, said Scott Gottlieb, MD, then-FDA commissioner, in announcing the groundbreaking approval.
Gottlieb was not exaggerating. The growth in CAR T-cell research is exploding. Although only a handful of cell and gene therapies are on the market, the FDA predicted in 2019 that it will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.
We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.
Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells.
Community oncologists will need to be increasingly familiar about the various products, including their immediate and longer-term risks, Bo Wang, MD, and Deepu Madduri, MD, recently wrote in OncologyLive.1 It is key to understand the optimal time for referring these patients to an academic institution, as well as how to manage the requisite post CAR T-cell therapy in the community setting. Madduri is an assistant professor of medicine, hematology and medical oncology, as well as associate director of cellular therapy service, and director of clinical operations with the Center of Excellence for Multiple Myeloma at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai in New York, New York. Wang is a third-year clinical fellow in hematology/oncology at Mount Sinai.
Early referral to academic centers and hospitals equipped to deliver therapies is crucial for patients eligible for therapy. However, as advances continue in the field, community practices may be called upon to administer therapies in their clinic.
The Community Oncology Alliance (COA) envisions a broader role for the settings in which CAR T-cell therapies can be administered. When the Centers for Medicare & Medicaid Services (CMS) was considering coverage for CAR T-cell therapies in 2019, COA officials argued against limiting approvals to hospitals.
It is important to understand that there are state-of-the-art community oncology practices that have significant experience and capabilities in administering highly complex treatments, COA officials wrote in a letter to CMS. For example, stem cell transplants, which are similar in complexity to CAR T therapy, are performed successfully in the community oncology practice setting.2
Broader use of gene therapies depends on several factors, including navigating the logistics of gene therapies, addressing the high costs, and managing toxicities.3
Autologous CAR T-cell therapies involve a manufacturing process that requires coordination between the treating facility and the processing facility. Following leukapheresis, patients may require maintenance therapy to control disease progression during the manufacturing time, which can take 3 to 5 weeks.
In terms of cost, gene and cell therapies can cost from $375,000 to $475,000 per dose and they may face coverage restrictions from payers. Approvals could take weeks to obtain.3,4
Because of cytokine release syndrome and neurotoxicities associated with CAR T-cell therapy, the FDA mandates risk evaluation and mitigation strategy training for centers.
Further, providers may find that real-world experiences with gene therapies are different from those seen in the clinical trial setting, according to Ankit J. Kansagra, MD.
In a presentation at the 2020 American Society of Clinical Oncology Virtual Education Program, Kansagra, an assistant professor of medicine and Eugene P. Frenkel, MD, Scholar in Clinical Medicine at Harold C. Simmons Comprehensive Cancer Center in Dallas, Texas, said that in practice patients may be older and have more aggressive disease, with double- and triple-hit lymphomas.4
Specifically, Kansagra noted that medications such as steroids and/or tocilizumab (Actemra) to prevent or treat cytokine release syndrome or other toxicities were more frequently used in the real-world setting than what had been seen in clinical trials.
As it stands now, only a fraction of eligible patients are receiving CAR T-cell therapies, Kansagra said. Potentially, 9750 patients a year may be eligible for CAR T-cell therapies in approved and upcoming hematologic indications. From 2016 to 2019, a total of 2058 patients received CAR T-cell infusion.4
Next steps for transplanting these novel therapies to clinical practice will require changes in key areas, Kansagra said, such as supply chain management, patient support, and financial systems (Figure).4
Figure. Next Steps for Effective Delivery of Gene and Cell Therapies4
Meanwhile, multiple myeloma experts advise providers to be ready for change. As commercially available myeloma CAR T-cell therapies are approved, it will be even more important for community oncologists to better understand these therapies so they can offer them to their patients, Wang and Madduri wrote.1
Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.
Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.
For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.
Safety has been the main brake around adeno-associated virus vector [AAV] gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by 2015 Giants of Cancer Care winner in immuno-oncology Carl H. June, MD, and others in CAR T-cell therapy. Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.
In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed/ refractory mantle cell lymphoma.
Another CD19-directed therapy under FDA review for relapsed/refractory large B-cell lymphoma, is lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application for ide-cel seeks approval for the B-cell maturation antigendirected CAR therapy to treat adult patients with multiple myeloma who have received at least 3 prior therapies.5
The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials. gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.6 Natural killer (NK) cells are the research focus of Dean A. Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital in Columbus, Ohio. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA investigational new drug application. Lee is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His efforts are geared toward pediatric sarcomas.
The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.
However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.
Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.
The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.
For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio (NPR) did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.7
Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.
She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.8
Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.
Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.
CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off-the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.
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Harnessing the Potential of Cell and Gene Therapy - OncLive
Two Studies Shed Light on How and Where the Body Can Add New Fat Cells – Technology Networks
By daniellenierenberg
Gaining more fat cells is probably not what most people want, although that might be exactly what they need to fight off diabetes and other diseases. How and where the body can add fat cells has remained a mystery - but two new studies from UT Southwestern provide answers on the way this process works.
The studies, both published online in Cell Stem Cell, describe two different processes that affect the generation of new fat cells. One reports how fat cell creation is impacted by the level of activity in tiny organelles inside cells called mitochondria. The other outlines a process that prevents new fat cells from developing in one fat storage area in mice - the area that correlates with the healthy subcutaneous fat just under the skin in humans. (Both studies were done in mice.)
In the second study, a commonly used cancer drug was able to jump-start healthy fat cell creation in mice, a finding that raises the possibility of future drug treatments for humans.
While fat isn't popular, as long as people overeat they will need a place to store the excess calories, explains Philipp Scherer, Ph.D., director of the Touchstone Center for Diabetes Research at UT Southwestern and senior author of the first study focusing on mitochondria. There are two options, he says: squeezing more lipids (fat) into existing fat cells and ballooning their size, leading to problems such as inflammation and, eventually, diabetes; or creating new fat cells to help spread the load. Fat stored properly - in fat cell layers under the skin (subcutaneous fat) that aren't overburdened instead of around organs (visceral fat) or even inside organs - is the healthy alternative, he says.
Problems follow if existing fat cells are left on their own to become engorged, adds Rana Gupta, Ph.D., associate professor of internal medicine and senior author of the second study. "When these cells are so overwhelmed that they can't take it anymore, they eventually die or become dysfunctional, spilling lipids into places not intended to store fat."
Those lipids may move into the liver, leading to fatty liver disease; to the pancreas, resulting in diabetes; or even to the heart, causing cardiovascular disease, Gupta says. Visceral, or belly fat, may surround the organs, creating inflammation.
The healthiest place to store fat is in subcutaneous fat, adds Gupta. Ironically, that is where mice in his study were least able to create new fat cells, despite the fact that stem-cell-like progenitor cells primed to become fat cells were present there as well, he says.
Gupta's study identified a process that prevents progenitor cells from developing into fat cells in mouse subcutaneous inguinal fat.
The protein HIF-1a (short for hypoxia-inducible factor-1 alpha) is central to the process. It kicks off a series of cellular actions that ultimately inactivate a second protein called PPARgamma, the key driver of fat cell formation.
These proteins are found in both humans and mice. In fact, in a culture of human subcutaneous fat cell progenitors, HIF-1a also inhibited new fat cells from being created, according to Gupta.
In Gupta's mouse study, researchers used a genetic approach to inhibit HIF-1a and found that the progenitor cells could then make subcutaneous inguinal fat cells and fewer were inflamed or fibrotic.
Next, they tested the cancer drug imatinib (brand name Gleevec) and found it had the same effect. The cancer drug was tried because it was known to have beneficial effects against diabetes in cancer patients with both diseases, Gupta says.
In Scherer's study, researchers manipulated a protein called MitoNEET in the outer membrane of the precursor cells' mitochondria, organelles known as the cells' power plants. The resulting mitochondrial dysfunction and drop in cell metabolism caused precursor cells to lose the ability to become new fat cells and increased inflammation.
"This study shows we can manipulate the precursor cells' willingness to become fat cells," Scherer says. "The ability to recruit new fat cells by tickling these pre-fat cells to become fat cells is very important and has profound beneficial effects on health, particularly in the obesity-prone environment that we all live in."
He says his goal is now to design a drug that could stimulate mitochondrial activity.
"Understanding the mechanism is an important first step," Scherer says, referring to the findings from the two studies. "We will have to learn in the future how to manipulate these processes pharmacologically."
Reference: Joffin N, Paschoal VA, Gliniak CM, et aI. Mitochondrial metabolism is a key regulator of the fibro-inflammatory and adipogenic stromal subpopulations in white adipose tissue. Cell Stem Cell. doi:doi.org/10.1016/j.stem.2021.01.002
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Two Studies Shed Light on How and Where the Body Can Add New Fat Cells - Technology Networks
Heidi Klum loves to pamper her skin with goodies from Drunk Elephant and Glossier – woman&home
By daniellenierenberg
With her sun-kissed hair and flawless golden skin, it's easy to forget that Heidi Klum is in her mid 40s. Genetics certainly help. But so do Heidi Klum's favourite skincare products by Glossier and Drunk Elephant, plus a reminder of home courtesy of German beauty brand The Cream by Augustinus Bader.
In a rare selfie, Klum showed off the Perfectil Hair Skin And Nails Vitamins she takes daily, alongside this smorgasbord of beauty products, which proves the supermodel takes her skincare seriously.
Here are some of her favourite products:
More from womanandhome:
Drunk Elephant T.L.C. Framboos Glycolic Resurfacing Night Serum
This night serum contains a hefty dose of glycol acid to exfoliate built-up dead skin cells and resurface dull, uneven skin. Expect brighter, smoother skin when you wake.
Drunk Elephant T.L.C. Sukari Babyfacial 25% AHA + 2% BHA Mask
If you like to feel a product working, you'll positive love this mask. Like an AHA/BHA facial in a bottle, it resurfaces skin to reveal greater clarity and improved skin tone.
Drunk Elephant C-Firma Day Serum
This vitamin C day serum is packed with antioxidants to protect skin from urban aggressors.
Glossier Solution
A liquid exfoliator that you apply a bit like toner, it buffs away dead dulling skin with a single swipe.
Mario Badescu Drying Lotion
We know, we know, even supermodels get spots. This cult zit lotion contains salicylic acid, sulfur, and zinc oxide to dry up blemishes - fast.
Augustinus Bader The Rich Cream
The brainchild of a stem cell scientist no less, this overnight treatment uses amino acids and vitamins to re-energise cells to repair damage more effectively.
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Heidi Klum loves to pamper her skin with goodies from Drunk Elephant and Glossier - woman&home
I Tried MAC’s Virtual Try-On Tool & Here’s What Happened – GLAMOUR UK
By daniellenierenberg
There's no better time than a national lockdown to test out the coolest new skincare/makeup/hair trends and snazzy innovations in beauty tech from the comfort of your own home. That said, there's nothing more frustrating than spending upwards of 100 on a snazzy new hair treatment or at-home facial system, only to find out it doesn't *actually* work.
Enter GLAMOUR Tries: the weekly Instagram series which sees GLAMOUR editors do all of the time-consuming (and expensive) work for you.
We've been busy trying out all of the (sometimes) wacky but always wonderful beauty crazes to take the internet by storm - from the FOREO's UFO 2 Smart Mask Treatment to Sarah Chapman's 138 Meso-Melt Infusion at-home facial and Toni&Guy's Hello Day! Secret Volumising Crimper. These are the products that every veteran beauty sleuth is talking about - but that you want to do a bit of research into before buying.
Thanks to GLAMOUR Tries, you won't need to waste your hard-earned pennies on testing these innovations yourself. We're getting in their first, giving you the lowdown and making sure you invest in products that genuinely work. Like what you see? You can shop all of the products seen on GLAMOUR tries, with the click of a button, down below. Don't say we don't treat you.
On GLAMOUR Tries this week, our Beauty Editor Lottie Winter tried MAC's brand new virtual try-on tool, in an attempt to find her perfect shade of the classic Studio Fix foundation - from the comfort of her sofa. But was the tool accurate? Was the selected foundation *actually* Lottie's exact match? Here's what happened...
First thing's first: how can you access the tool? "All you have to do is go to the MAC website, find the Studio Fix foundation and simply click on "find your shade match" - and it'll take you straight through to the virtual try-on tool," said Lottie. Click "start now", "allow access to your camera" and then - strike a pose!
"Within just a few seconds, it has selected not only my best match but options for a more golden, more rosy, darker and lighter finish," Lottie continued. "N6 is the shade of Studio Fix Fluid foundation that the virtual try-on tool has chosen for me. So, let's put it to the test..."
Buy It Now
"I'm using a MAC 170 brush to apply. I've just half of my face so we can compare the results - but it couldn't be a better match. It has blended so effortlessly and it has knocked out all the redness around my nose and my eyes.
"What I love most about this foundation is that it's super long-wearing and it helps to control shine throughout the day which is great if you're like me and you have very oily skin. But also the coverage is buildable which means you can get that believable finish."
Any final thoughts? "It couldn't have been easier to get my perfect shade match. It just makes shopping for foundation online so much easier and totally reliable," said Lottie. We're sold.
Would you MAC's virtual try-on tool a go? Have you already tried it? Let us know your thoughts over on Instagram @glamouruk.
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I Tried MAC's Virtual Try-On Tool & Here's What Happened - GLAMOUR UK