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Best Market Research on Stem Cell Therapy Market, Industry / Sector Analysis Report, Regional Outlook & Competitive Market Share & Forecast,…

Trusted Business Insights presents the Updated and Latest Study on Stem Cell Therapy Market 2019-2025. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Abstract, Snapshot, Market Analysis & Market Definition: Stem Cell Therapy MarketIndustry / Sector Trends

Stem Cell Therapy Market size was valued at USD 7.8 billion in 2018 and is expected to witness 10.2% CAGR from 2019 to 2025.

U.S. Stem Cell Therapy Market Size, By Type, 2018 & 2025 (USD Million)

Rising prevalence of chronic diseases will positively impact the stem cell therapy market growth. Cardiovascular diseases, neurological disorders and other chronic conditions have resulted in high mortality over past few years. Conventional therapeutic methods and treatments are currently replaced due to lack of efficiency and efficacy. Recently developed stem cell therapies are capable of replacing defective cells to treat diseases that has reduced morbidity drastically. Therefore, people have now started relying on stem cell therapy that has long term positive effects.

Advancements in stem cell therapy in developed regions such as North America and Europe have boosted the industry growth. Since past few years, there have been several researches carried out for stem cell therapy. Currently developed stem cell therapies have shown positive outcomes in treatment of leukemia. Similarly, due to advancements in regenerative medicine, several other chronic conditions such as muscular dystrophy and cardiovascular diseases also have been cured. Aforementioned factors have surged the industry growth. However, high cost of allogenic stem cell therapy may hamper the industry growth to some extent.

Market Segmentation, Outlook & Regional Insights: Stem Cell Therapy Market

Stem Cell Therapy Market, By Type

Allogenic stem cell therapy segment held around 39% revenue share in 2018 and it is anticipated to grow substantially during analysis timeframe. Allogenic stem cell is available as off the shelf therapy and it is easily scalable that helps in providing treatment without delay. Moreover, the procedure includes culturing donor-derived immunocompetent cells that are highly effective in treatment of several diseases. Stem cells obtained in allogenic therapy are free of contaminating tumor cells. This reduces risk for disease recurrence that will surge its demand thereby, stimulating segment growth.

Autologous stem cell therapy segment is estimated to witness 10.1% growth over the forthcoming years. People usually prefer autologous stem cell therapy as it has minimum risk of immunological rejection. However, on introduction of allogenic stem cell therapy, demand for autologous stem cell therapy has declined as it is difficult to scale up. However, there are concerns regarding risk of cross contamination during large scale manufacturing of autologous stem cell lines that will impede segmental growth to some extent.

Stem Cell Therapy Market, By Application

Neurology segment was valued around USD 1.6 billion in 2018 and it is estimated that it will witness significant growth over the forthcoming years. Stem cells are used to replenish the disrupted neurological cells that helps in quick patient recovery. Pluripotent stem cells provide replacement for cells and tissues to treat Alzheimers, Parkinsons disease, cerebral palsy, amyotrophic lateral sclerosis and other neurodegenerative diseases. Thus, pivotal role of stem cells in treating life threatening neurological condition will escalate segment growth.

Cardiovascular segment will witness 10% growth over the analysis timeframe. Considerable segmental growth can be attributed to development in stem cell therapies that have enhanced recovery pace in patients suffering from cardiovascular diseases. Recently developed allogenic stem cell therapies are efficient and easily available that have reduced the mortality rates in cardiovascular patients. Above mentioned factors will propel cardiovascular segment growth in near future.

Germany Stem Cell Therapy Market Size, By Application, 2018 (USD Million)

Stem Cell Therapy Market, By End-users

Hospitals segment held over 56% revenue share in 2018 and it is anticipated to grow significantly in near future. Rising preference for stem cell therapies offered by hospitals proves beneficial for the business growth. Hospitals have affiliations with research laboratories and academic institutes that carry out research activities for developing stem cell therapies. On introduction and approval of any novel stem therapy, hospitals implement it immediately. Associations with research and academic institutes further helps hospitals to upgrade its stem cell treatment offerings that positively impacts the segmental growth.

Clinics segment is expected to grow at around 10% during the forecast timeframe. Clinics specializing in providing stem cell therapies are well-equipped with advanced medical devices and superior quality reagents required for imparting stem cell therapies. However, as clinics offer specialized stem cell therapies, their treatment cost is much higher as compared to hospitals that may reduce its preference.

Stem Cell Therapy Market, By Region

North America stem cell therapy market held around 41.5% revenue share in 2018 and it is estimated to grow substantially in near future. Increasing adoption of novel stem cell therapies will prove beneficial for regional market growth. Moreover, favourable government initiatives have positive impact on the regional market growth. For instance, government of Canada has initiated Strategic Innovation Fund Program that invests in research activities carried out for stem cell therapies enabling development in stem cell therapy. Above mentioned factors are expected to drive the North America market growth.

Asia Pacific stem cell therapy market is anticipated to witness 10.8% growth in near future owing to increasing awareness amongst people pertaining to benefits of advanced stem cell therapies. Additionally, favourable initiatives undertaken by several organizations will promote industry players to come up with innovative solutions. For instance, according to Pharma Focus Asia, members of Asia-Pacific Economic Cooperation collaborated with Life Sciences Innovation Forum to involve professionals having expertise in stem cell therapies from academia and research centers to promote developments in stem cell research. Thus, growing initiatives by organizations ensuring availability of new stem cell therapies will foster regional market growth.

Latin America Stem Cell Therapy Market Size, By Country, 2025 (USD Million)

Key Players, Recent Developments & Sector Viewpoints: Stem Cell Therapy Market

Key industry players in stem cell therapy market include Astellas Pharma Inc, Cellectis, Celyad, Novadip Biosciences, Gamida Cell, Capricor Therapeutics, Cellular Dynamics, CESCA Therapeutics, DiscGenics, OxStem, Mesoblast Ltd, ReNeuron Group and Takeda Pharmaceuticals. Chief industry players implement several initiatives such as mergers and acquisitions to sustain market competition. Also, receiving approvals for stem cell therapy products from regulatory authorities fosters companys growth. For instance, in March 2018, European Commission approved Takedas Alofisel that is off-the-shelf stem cell therapy. Product approval will help company to gain competitive advantage and capture market share.

Stem Cell Therapy Industry Viewpoint

Stem cells industry can be traced back to 1950s. In 1959 first animals were made by in-vitro fertilization by preserving the stem cells. Till 2000, research was being carried out on stem cells to study its therapeutic effect. In 2000, fund allocations were made to research on cells derived from aborted human foetuses. In the same year, scientists derived human embryonic stem cells from the inner cell mass of blastocytes. Later, in 2010, clinical trials for human embryonic stem cell-based therapy were initiated. As technology progressed, stem cell therapy for treating cancer were developed. However, due to ethical issues, the use of stem cells for curing diseases witnessed slow growth for few years. But as the regulatory scenario changed, people started preferring stem cell therapies due to its better efficacy. Stem cell therapy is in developing stage and has numerous growth opportunities in developing economies with high prevalence of chronic diseases.

Key Insights Covered: Exhaustive Stem Cell Therapy Market1. Market size (sales, revenue and growth rate) of Stem Cell Therapy industry.2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Stem Cell Therapy industry.3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Stem Cell Therapy industry.4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Stem Cell Therapy industry.

Research Methodology: Stem Cell Therapy Market

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Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsUS: +1 646 568 9797UK: +44 330 808 0580

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Best Market Research on Stem Cell Therapy Market, Industry / Sector Analysis Report, Regional Outlook & Competitive Market Share & Forecast,...

The Inside Scoop on Northwest Biotherapeutics, Inc (OTCMKTS: NWBO) – MicroCap Daily

Northwest Biotherapeutics, Inc (OTCMKTS: NWBO) is an exciting little biotech that has managed to attract legions of shareholders who continue to accumulate. The stock does have a history of big moves up recently seeing another significant pop.

There is a lot to get excited about on NWBO, the Company is developing cancer vaccines designed to treat a broad range of solid tumor cancers more effectively than current treatments, and without the side effects of chemotherapy drugs.

Northwest Biotherapeutics, Inc (OTCMKTS: NWBO) is a clinical stage biotechnology company focused on the development of personalized immunotherapy for a broad range of solid tumor cancers. The company has over 190 issued patents and more than 65 pending patent applications worldwide, grouped into 12 patent families. The Company is led by Linda Powers, a successful entrepreneur who worked for many years in corporate finance and restructurings, mergers and acquisitions, joint ventures and intellectual property licensing. Ms. Powers has served as the Chairman of NW Bio since 2007, and as CEO since 2011. NWBO platform technology is DCVax, which uses activated dendritic cells to mobilize a patients own immune system to attack their cancer.

Northwest lead product is DCVax-L for Glioblastoma multiforme (GBM), the most lethal form of primary brain cancer. The Company has completed two Phase I/II trials and are now well under way with a large Phase III trial, as described below. Northwest is currently conducting a 348-patient double blind, randomized, placebo controlled Phase III clinical trial with DCVax-L for newly diagnosed GBM. The primary endpoint of the trial is Progression Free Survival, meaning the length of time that a patient continues without disease progression (i.e., recurrence of the tumor). Secondary endpoints include overall survival and other measures. The trial is under way at 51 sites (medical centers) across the US. The sites and the eligibility criteria are listed in the profile of the trial at ClinicalTrials.gov. The trial is also under way in Europe. The lead site is Kings College Hospital in London. Approximately 30 trial sites are also in varying stages of preparation in the U.K. and Germany.

DCVax Direct offers a potential new treatment option for the wide range of clinical situations in which patients tumors are considered inoperable because the patient has multiple tumors, or their tumor cannot be completely removed, or the surgery would cause undue damage to the patient and impair their quality of life. DCVax-Direct is administered by direct injection into a patients tumors. It can be injected into any number of tumors, enabling patients with locally advanced disease or with metastases to be treated. DCVax-Direct can also be injected into tumors in virtually any location in the body: not only tissues at or near the surface of the body but also, with ultra-sound guidance, into interior tissues. Northwest is currently conducting a 60-patient Phase I/II trial of DCVax-Direct for all types of inoperable solid tumors. The trial is under way at MD Anderson in Houston, TX and MD Anderson in Orlando, FL, with additional sites in varying stages of preparation. The Phase I stage of the trial involves dose escalation, testing 3 different dose levels of DCVax-Direct, and confirmation of the optimal dose.

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DCVax-Prostate is designed specifically for late stage, hormone independent prostate cancer. Such cancer involves the spread of micro-metastases beyond the prostate tissue. In most patients, there is no focal tumor which can be surgically removed and used to make lysate, or into which dendritic cells can be directly injected. Instead, the cancer cells are diffuse. We have developed a DCVax product line using a particular proprietary antigen PSMA (Prostate Specific Membrane Antigen) which is found on essentially all late stage (hormone independent) prostate cancer. The PSMA is produced through recombinant manufacturing methods, and is then combined with the fresh, personalized dendritic cells to make DCVax-Prostate.

Northwest is led by Linda Powers, her linkedin page describes her as: Ms. Powers served as Chairman of NW Bio for the last 4 years, and brings more than 25 years experience in corporate transactions and operations, including more than a decade specializing in building biotech companies through Toucan Capital. Ms. Powers is particularly well known for her experience in building biotech companies that are developing cell therapies, including both immune cell therapies (such as NWBios DCVax) and adult stem cell therapies. Such products consisting of living human cells require fundamentally different manufacturing, storage, distribution and handling than do pharmaceutical drugs (pills in bottles). Such living cell products also involve different clinical and regulatory requirements, and different business and cost/pricing models, than traditional drugs. The cell therapy companies which Ms. Powers has been involved in building over the last decade, both in the US and abroad (in Asia, Europe and Israel), are at the forefront of clinical trials and early commercialization. Ms. Powers has served for years on a number of related boards, including the M2Gen Board of the Moffitt Cancer Center, the Board of the Trudeau Institute (a world leader in immunology research) and others. As Chairman of NW Bio, she has brought her lengthy experience to bear in helping to shape NW Bios overall strategy and programs. As CEO, she will now undertake operational responsibilities in addition to continuing her duties as Chairman.

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Currently trading at a $129 million market valuation NWBO has $1.9 million in the treasury, manageable debt and fast growing sales reporting $593k in revenues for the 3 months ended September 30, 2019. NWBO is an exciting story developing in small caps; this little biotech is sitting on drugs in development that could potentially turn the multi-billion dollar drug-resistant cancers market upside down. NW Bio lead product, DCVax-L, is currently in a 331-patient Phase III trial for patients with newly diagnosed Glioblastoma multiforme (GBM), the most aggressive and lethal brain cancer. The stock has a significant shareholder base that continues to bid this one higher. We will be updating on NWBO when more details emerge so make sure you are subscribed to Microcapdaily so you know whats going on with NWBO.

Disclosure: we hold no position in NWBO either long or short and we have not been compensated for this article.

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The Inside Scoop on Northwest Biotherapeutics, Inc (OTCMKTS: NWBO) - MicroCap Daily

3D beating heart tissue experiment heads to Space Station – UW Medicine Newsroom

Note to editors and reporters: Live coverage on NASA Television of the SpaceX CRS-20 cargo launch carrying this experiment is scheduled at 8:30 p.m. EST, 11:30 p.m. PST March 6 and will be replayed twice on March 7. Coverage of the rendezvous with the International Space Station will be at 5:30 a.m. EST Monday, March 8, with installation at 8:30 a.m. All times are subject to change due if weather or launch conditions are unfavorable

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Web-embeddable video

Photos of tissue loading

Soundbite Log

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Youtube link

Space exploration can take a toll on the human heart. Astronauts are at risk for changes in their cardiac function and rhythm. To learn how microgravity and other physical forces in space exact their effects on heart muscle, a Tissue Chips in Space project has now been packed and is awaiting launch to the International Space Station.

The experimental equipment consists of small, compact devices, a little bit larger than cell phone cases. The holders contain a row of tiny, 3-D globs of beating heart tissue grown from pluripotent stem cells, generated from human adult cells. The heart muscle tissue is supported between two flexible pillars that allow it to contract freely, in contrast to the rigid constraints of a Petri dish.

The devices also house a novel invention from the University of Washington. It automatically senses and measures the contractions of the heart tissues, and reduces the amount of time the astronauts will need to spend conducting this study.

The flexible pillars contain tiny magnets, explained UW graduate student Ty Higashi, one of the inventors. When the muscle tissue contracts, the position of the embedded magnets changes, and the motion can be detected by a sensor, he said. That information is then sent down to a laboratory on Earth.

This model will recapitulate, on a miniature scale, what might be happening to the architecture and function of heart muscle cells and tissues in astronauts during a space mission.

The project head is Deok-Ho Kim, a professor in bioengineering, who recently joined the Johns Hopkins University faculty in Baltimore. He and co-investigator, Nathan Sniadecki, a professor in mechanical engineering, began this study two years at the UW Medicine Institute for Stem Cell and Regenerative Medicine (ISCRM). Jonathan Tsui, a postdoc in bioengineering, Ty Higashi, a graduate student in mechanical engineering , and other members of the UW project team, continue the cross-country collaboration in Seattle. The team is working with several NASA and National Institutes of Health groups, and researchers at other universities, on this effort.

Sniadecki said that each of the tissues heading to the International Space Center contain about a half million heart cells.

They act like a full tissue, he explained. They contract, they beat and you can actually see them physically shorten in the dish. Were actually able to see little heart beats from these tissues.

The SpaceX shuttle delivering this scientific payload is expected to leave from Cape Canaveral no earlier than 8:50 p.m. PST (11:50 p.m. EST) Friday, March 6. The exact departure schedule depends on the weather and other factors.

Once on board, the experiment will run for 30 days before being returned to Earth for further analysis. A related space-based experiment will follow skyward later, to see if medications or mechanical interventions can offset what the heart muscle endures during extended space missions.

The space program is looking at ways to travel longer and farther, Sniadecki said. To do so, they need to think about protecting their crews. Having treatments or drugs to protect astronauts during their travel would make long term space travel possible.

Guarding against cardiac problems would be especially critical during space travel at distances never attempted before, such as a mission to Mars, said Sniadecki. This opportunity to really kind of push the frontier for space travel is every engineers dream.

He added, We also hope to gather information that will help in preventing and treating heart muscle damage in people generally, as well as in understanding how aging changes heart muscle.

Microgravity is known to speed up aging, and likely influence other cell or tissue properties. Because aging is accelerated in space, studies on the International Space Station is a way to more quickly assess this process over weeks, instead of years.

I think the medicine side of it is extremely helpful on Earth, too, because what we discover could potentially lead to treatments for counteracting aging, Sniadecki said.

This space medicine research project is funded by the National Center for Advancing Translational Sciences and the National Institute of Biomedical Imaging and Bioengineering. This heart tissue study is part of the national Tissue Chips in Space program.

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3D beating heart tissue experiment heads to Space Station - UW Medicine Newsroom

Shingles vaccine makes an impact around the world and right here in Rockford – Rockford Register Star

Although most people are aware that the College of Medicine graduates 55 physicians each year, many of whom eventually practice in Winnebago County, the research enterprise of the campus is less well known. In particular, a remarkable breakthrough in the prevention of shingles occurred two years ago which should make every Rockfordian proud. Shingles is a painful skin rash that can cause itching and excruciating pain, and occasionally blindness.

Without a preventive vaccine it will impact 1 out of every 3 people and can be particularly serious for those who are elderly or have impaired immune systems. The exciting news is that a vaccine developed right here in Rockford is providing protection to millions of people around the world.

Two years ago, the U.S. Food and Drug Administration approved a new vaccine to prevent herpes zoster or shingles, a painful condition caused by the chickenpox virus. After childhood chickenpox, the virus becomes dormant in neurons and may reactivate in adults to cause shingles. The vaccine, marketed by GSK under the brand name ShingrixTM, is now the recommended vaccine for adults over age 50. It is not only safer, but is more effective than the existing live vaccine.

Abbas Vafai, Ph.D., the scientist who developed the Shingrix vaccine, worked on its development when he was an associate professor of microbiology at the University of Illinois College of Medicine Rockford from 1990 to 1997 and also had worked on the vaccine at the University of Colorado. The U.S. Food & Drug Administration approved the vaccine for use in October 2017. Since then, the Centers for Disease Control has recommended Shingrix for all adults over age 50.

Because of this, its use for all adults 50 years and older is warranted the only problem has been getting enough of it made to meet the worldwide demand. Vaccine shortage have been felt in Rockford and throughout the country.

We are incredibly excited about Dr. Vafais success with vaccine development and are extremely proud that a vaccine developed on the Rockford campus of the University of Illinois College of Medicine is now impacting the health of people worldwide.

Because some of the work on the vaccine was conducted on the Rockford campus, a portion of the profits will go to the College of Medicine Rockford. The total of what the College of Medicine Rockford and its Department of Biomedical Sciences may receive in royalties over the course of the seven years is predicted to be over $10 million. These are dollars that allow us to continue to educate tomorrows scientists and physicians and support cutting-edge research.

As the UIC Health Sciences Campus-Rockford on Parkview Avenue continues to thrive and grow, so does the Rockford community. In fact, an economic impact study conducted in 2016 indicated the campus had an impact of over $58.2 million to the Rockford Metro Area the equivalent of 898 jobs.

For those whose doctor recommends the Shingrix vaccine, they can expect the vaccine, given in two doses, will prevent what could be a serious and painful condition. The vaccine is also extremely effective in preventing a consequence of shingles called postherpetic neuralgia. Postherpetic neuralgia occurs in 10% to 18% of individuals who have shingles and is characterized by a severe chronic pain condition that can last for years.

Biomedical research at the College of Medicine continues to explore new ways to improve health and prevent disease. State-of-the-art research in the areas of prostate cancer, lung cancer, eradication of parasitic infections, improving the longevity of joint replacements and stem cell treatment of severe debilitating neurologic diseases, occurs daily on campus. With the support of our community, the next major medical breakthrough could again come from the University of Illinois College of Medicine Rockford and its team of researchers in the Department of Biomedical Sciences and other academic departments.

Dr. Alex Stagnaro-Green is the regional dean of the University of Illinois College of Medicine at Rockford.

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Shingles vaccine makes an impact around the world and right here in Rockford - Rockford Register Star

Omeros Corporation Reports Updated Results from Narsoplimab HSCT-TMA Clinical Trial and Highlights from Recent Clinical and CMC Meetings with FDA -…

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today reports an update on clinical data from its pivotal trial of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), markedly exceeding the FDA-agreed threshold for the primary efficacy endpoint. While an overview of preliminary data submitted to FDA was made public on December 4, 2019 in a press release from the company, all patients have now completed treatment and trial enrollment has been closed. Narsoplimab is Omeros human monoclonal antibody targeting mannan-binding lectin-associated serine protease 2 (MASP-2).

In recent meetings with FDA focused on clinical as well as chemistry, manufacturing and controls (CMC) data, FDA confirmed important aspects of Omeros rolling Biologics License Application (BLA) for narsoplimab in HSCT-TMA. The BLA continues on its clear path to completion.

The efficacy threshold agreed with FDA, the updated results from the 28-patient trial, and highlights of the recent FDA meetings are the following:

Primary Endpoint

Secondary Endpoints

Safety

The treated population had multiple high-risk features that portend a poor outcome, including the persistence of HSCT-TMA despite modification of immunosuppression (which was a criterion for entry into the trial), graft-versus-host disease, significant infections, non-infectious pulmonary complications and neurological findings. Patients in the trial had a high expected mortality rate, with 93% of them having multiple risk factors.

The efficacy and safety data from the pivotal trial with narsoplimab are encouraging, said Miguel-Angel Perales, M.D., Deputy Chief of the Adult Bone Marrow Transplantation Service and Director of the Adult Stem Cell Transplantation Fellowship at Memorial Sloan Kettering Cancer Center. Given the trials stringent response criteria across laboratory markers and organ function, the complete response rate seen with narsoplimab is remarkable, as is the 100-day survival. There currently is no approved treatment for HSCT-TMA. Current therapy is generally limited to supportive care and withdrawal of drugs critical for GVHD prophylaxis. Not only could narsoplimab become central to the treatment of HSCT-TMA, it might well allow us to maintain that needed GVHD prophylaxis.

Complete clinical trial data will be presented by Dr. Perales later this month at the Annual Meeting of the European Society for Blood and Marrow Transplantation in Madrid.

Recent FDA Meeting Highlights and CMC Updates

The non clinical sections of our BLA have been submitted, our CMC campaign is progressing well with process validation and commercial lots already manufactured, and our pivotal trial is complete, stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. The efficacy threshold agreed with FDA reflects both the primary endpoints stringent response criteria and the poor outcomes expected in the patients enrolled in our trial. Of course, were very pleased that the response rates and confidence intervals seen with narsoplimab are well above that efficacy threshold. We look forward to continuing to work closely with regulators to make the drug commercially available to transplanters and their patients in the U.S. and internationally as quickly as possible.

In addition to its HSCT-TMA program, Omeros is enrolling its narsoplimab Phase 3 clinical trials for immunoglobulin A (IgA) nephropathy and atypical hemolytic uremic syndrome (aHUS). Narsoplimab has been granted, for both HSCT-TMA and IgA nephropathy, FDAs breakthrough therapy designation as well as orphan drug designations from FDA and the European Medicines Agency. The drug also holds FDAs fast-track designation for aHUS.

Primary Efficacy Endpoint

To be considered a responder, a patient must achieve the primary endpoint of complete HSCT-TMA response defined by improvement in laboratory markers and improvement in clinical status.

Laboratory Markers

Clinical Status

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, the company has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, can, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, expectations regarding regulatory exclusivities, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K for the year ended December 31, 2019, filed with the Securities and Exchange Commission on March 2, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

Dr. Miguel-Angel Perales has received compensation from Omeros for advisory services.

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Omeros Corporation Reports Updated Results from Narsoplimab HSCT-TMA Clinical Trial and Highlights from Recent Clinical and CMC Meetings with FDA -...

Stem Cell Alopecia Treatment Market 2020 Size, Shares, Key Players, Demand, Supply, Growth and Forecast to 2026 – 3rd Watch News

New Jersey, United States,-The Stem Cell Alopecia Treatment Market report was created with experience and knowledge by market analysts and researchers. It is a phenomenal compilation of important studies that examine the competitive landscape, segmentation, geographic expansion and sales growth, production and consumption of the Stem Cell Alopecia Treatment market. Players can use the reports accurate market data and numbers, as well as statistical studies, to understand the current and future growth of the Stem Cell Alopecia Treatment market. The report includes CAGR, market share, sales, gross margin, value, volume and other key market numbers that provide a clear picture of the growth of the Stem Cell Alopecia Treatment market.

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Top Key Players of the Stem Cell Alopecia Treatment Market Research Report:

Our analysts have used advanced primary and secondary research techniques and tools to prepare this report. The sources and research tools we use are extremely reliable. New players can also use this research study to develop business strategies and learn about future market challenges. We offer a complete competitive analysis that includes a detailed company profile of the main players, a study of the nature and characteristics of the supplier landscape and other important studies.

Leading Regions

The research study contains important results and insights from our monitoring and analysis of the market for Stem Cell Alopecia Treatment industry. We have provided key data points, including divestments, launches, enhancements, partnerships, mergers, acquisitions, and other strategic initiatives by players in the Stem Cell Alopecia Treatment market. The report also includes price developments for regional markets and an analysis of important market events at regional and global levels. Our analysis enables you to make informed decisions in the market for Stem Cell Alopecia Treatment in terms of acquisitions, inventory, prices and production. We enable you to offer your opponents tough competition by providing fast, actionable market information in real time.

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Table of Content

1 Introduction of Stem Cell Alopecia Treatment Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Alopecia Treatment Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Alopecia Treatment Market, By Deployment Model

5.1 Overview

6 Stem Cell Alopecia Treatment Market, By Solution

6.1 Overview

7 Stem Cell Alopecia Treatment Market, By Vertical

7.1 Overview

8 Stem Cell Alopecia Treatment Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Alopecia Treatment Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Ethan Zohn Talks Survivor: Winners at War and Life After Cancer – menshealth.com

Survivor fans have been waiting for an all-winners season of the hit CBS show for a very long time, and when the official cast list for Survivor: Winners at War was finally released, everyone was excited to see Ethan Zohn's name among the list of returnees. With his positive attitude, he quickly became a fan favorite when he played (and won) Survivor: Africa back in 2002, and when he later appeared on Survivor: All-Stars in 2004.

Zohn's been busy in the 16 years between All-Stars and Winners at War, from the nonprofit organization he co-founded to his appearance on The Amazing Race in 2011. Zohn played professional soccer prior to Survivor, and after the show, he reported for ESPN during the 2002 FIFA World Cup. Zohn has also been open about his post-show health struggles, as he battled a rare type of cancer called CD20-positive Hodgkin's lymphoma in 2009 and 2011. In 2012, Zohn received two stem cell transplants from his brother, and he's been in remission ever since.

While Zohn started off strong on Winners of War by forming an alliance with big-name players, he ended up getting majorly blindsided on the season's fourth episode, and it also looks like he might be in some trouble during tonight's episode. Zohn spoke with Men's Health about his time on the show, how he prepped for his long-awaited return, and how his experience on the Edge of Extinction paralleled his battle with cancer.

This interview has been edited and condensed for clarity.

"I had no idea whatsoever that I was about to be voted off that night. Definition of blindside ... I had a good thing going with my alliance (Rob and Parvati), and I had a little side thing going with Adam, and so when Adam revealed the plan to go against Parvati, we were just like, 'Uhh no,' because both Rob and I were working with Parvati, more than Adam. So we had to switch the script and go after Adam and it backfired for a lot of different people, most importantly me."

"I was so discombobulated. Watching it back is actually fun for me, because it was such a blur ... Once I saw my name the second time up there, I was like, 'This is the end for me.' I had put in a lot of time and energy and motivation in getting ready to go play the game. And after 16 years, two cancer diagnoses, for me to get to the starting point was such a huge, huge deal. I dont think people understand the complexity of my health challenges leading up to this point. So to be kicked off early and get sent to the Edge of Extinction was very disappointing, I had very high expectations for myself. I'm a former professional soccer player, a marathoner, an athlete and I like to win, so I would have liked to play the game of Survivor. The Edge of Extinction, we get to play but its a different game."

"I like to think that Im generally positive. I think whats interesting about the Edge of Extinction is that its this weird world between life and death. So I think the parallels between living on the Edge of Extinction and going through cancer is kind of similar. Youre here on this island, youre caught between life in the game and life out of the game, youre isolated from your friends and family, youre tired youre hungry, youre thirsty, youre lethargic."

"But the incredible thing about the Edge of Extinction is that you may have a couple chances to get back into the game, and that was all I needed. All you need is a tiny bit of hope. And that hope can come in a multitude of different ways: winning challenges, fire tokens, other ways to get back in the game. With cancer there is a clear parallel. You gotta just stay alive long enough because you just never know whats around the corner. A new cure, a new drug, cancer research, whatever it is. But as long as you keep that hope alive, theres a way out of this thing."

"I was extremely excited as you can imagine, but I was also like, 'I have to get off the couch and start getting into shape again.' I was so excited, I knew this probably going to happen at some point in my life, that there was going to be an all-winners edition. And I remember, I was literally in my second stem transplant watching Heroes vs. Villains, and I was fairly confident that I would have been asked to play that season if I hadnt been sick. So here I am, literally surviving for my life here in this hospital room watching my friends play another game, and I would have done anything to be out there, because to me that represents adventure and life and health. Being a healthy person in this world to me, is going out there and being able to compete at the highest level in a game that touches you physically, mentally, socially, spiritually, environmentally. And so I was like if I get out of this thing alivewhich I had a 50% shot ofthen Im going to do everything in my power to get myself ready to go play this game again."

"Yes. I was completely dumbfounded to see Amber out there. They had separated us prior to the game as boys and girls, and then we get out there and we see the women show up. I expected that, but then I saw Amber. I was standing next to Rob and I tapped him on the back and was like, 'Hey dude, you got your wife out here? Well done, well played.'"

"I was researching on the internet pretty heavily, and there was a lot of fan theories and fans analyzing for us, and I love when people do that [laughs]. I was watching all the seasons of the people that I thought might be in there."

"Parvati and I are friends outside of the game so we definitely spoke, but other than that I didnt speak to anyone ... Theres a lot of activity with the new schoolers now. They go to parties, charity events, social media-ing. So theres a lot of pre-existing relationships that I think may or may not have played a factor in the game. But thats not part of my game. When you get to the island the game starts, thats when the game starts."

"I live in New Hampshire, so when I got the call, there was snow up to my thighs outside. So my wife [interior designer Lisa Heywood] and I left New Hampshire, packed the house, moved to a ridiculously amazing health and wellness community called Serenbe near Atlanta so I could be outside and train and light fires. My wife was hiding idols in the forest for me, I was tying and untying knots, doing puzzles.

"Im playing 16 years of catch up, and the game is moving at a rocket pace, and I am used to game where ... there was no idols, there were no clues, there was no way to get back in the game, no fire tokens or currency, no Edge of Extinction, nothing. So I really had to play a lot of catch up and get used to this new gameplay and I just tried to do the things that I could.

"I read some body language books and some lip reading stuff. I really took this seriously, but it may not have looked it, because I got voted off on the fourth episode [laughs]. But I thought if Im really going to do this and this is my last hurrah and Im going to win this money, then I have to put in the time and effort."

"I hired Aaron Leventhal, hes the strength and conditioning coach for the Minnesota Thunder. Hes also a former professional soccer player and we played together back in the day. So I hired him to give me a training plan, an eating plan, everything.

"This time, I really focused on eating. I cut out coffee, sugar, I stopped using cannabis. So that was a big thing for me because I use CBD everyday and cannabis is part of my recovery from going through cancer and dealing with all the anxiety. I started going to bed when the sun went down, I tried to rise when the sun came up. I did everything I could to get myself on a cycle and on a system where my body wasnt going through shock.

"I actually asked the Survivor doctors if I could bring it out there, because I use it as a medicine, and its not illegal in all states. But its illegal in Fiji!"

"Cancer is fairly easy to go throughwhen the doctor tells you to do something or youre going to die, you pretty much do it, you dont have any choice. But with surviving cancer and being a young adult with this whole world in front of you, thats when it really gets tough. Theres uncertainty, theres scars that need healing, you dont know if the cancer is going to come back. There's anxiety that your whole life is going to be different, whos going to want to marry you, am you going to have kids, can you get a job or health insurance.

"All that stuff is just going through your head on a daily basis and I just got sucked into that life of fear and anxiety and it was hard for me to break out of that. But then I finally did and now I have to go on Survivor and be away from everything. I had gotten to a point in my life where everything was all good, so that was really difficult for me to be honest, behind the scenes. I might look calm and collected out there, but on the inside I was...it was war of the worlds going on.

"I became an investor in a farm in upstate Vermont thats called MontKush, and we produce certified organic, high altitude outdoor hemp. I actually have a line of CBD tinctures that will be coming out in the summer. Theres also a TV show coming out that's going to be about the farm, and the leads are Anthony Sullivan from OxiClean and Dave Christian [Ed: Zohn will also appear on the show, but he won't be a main character]. I moved up there this past summer when I got back from Survivor and I lived on the farm and did everything a farmer would do."

"Lets put it this way: I would like to see an old schooler win this game."

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Low back and neck pain is costing us a fortune. Here’s how to stop – jacksonprogress-argus

If you're constantly seeking relief for bothersome back and neck pain, you're likely not alone, according to a study published Tuesday in JAMA.

In 2016, Americans and their insurance companies spent an estimated $134.5 billion on lower back and neck pain -- more than all forms of cancer combined.

Researchers estimated US public, private and out-of-pocket spending on health care for 154 health conditions from 1996 to 2016 and low back and neck pain was first, followed by other musculoskeletal conditions including joint and limb pain, then spending for diabetes, heart disease, falls and urinary diseases.

"In terms of health and wellness, I think the study highlights [that] a lot of the issues could be prevented with proper wellness and nutrition balance in our lives," said Dr. Sheldon Yao, chair and professor of Osteopathic Manipulative Medicine at the New York Institute for Technology's College of Osteopathic Medicine. Yao was not part of the study.

Drivers of back and neck pain

Back pain can be debilitating, removing people from enjoying the activities of everyday life. This area of the body is composed of complex system of muscles, ligaments, tendons, disks and bones, which all coordinate to support the body.

Obesity, sedentary lifestyles, technology and poor diet have all been linked to back and neck pain.

"Obesity is a giant epidemic that plays a part into back pain," Yao said, explaining that a loss of core strength due to obesity can put someone at increased risk for back pain.

Poor posture and a lack of core- and neck-strengthening exercises -- such as planks, neck-tilts, yoga and lifting weights -- also contribute to increased incidence of low back and neck pain, because weak muscles fail to properly support bones and are more prone to injury.

The amount of time a person spends sitting at their desk or bending over their cell phone can also be to blame.

"Everyone's on their smart phone now, constantly connected 24/7 and your posture is just looking forward," Yao said. The strain on a person's spine from constantly bending one's neck to text or browse can feel like the equivalent of 20 or 30 pounds, according to a 2018 study.

"Those are things where, as a society, we are not balanced," Yao said. "I'm not saying you can't eat any of those things, but just be aware of how much we're taking in in terms of those inflammatory foods."

How to alleviate pain at home

Health care spending on neck and back pain has increased each year since 1996, the study found, including newer and more expensive treatments such as stem cell and plasma injections, and an increase in surgeries instead of outpatient treatment.

The dollars don't appear to be well-spent, said Dr. Joseph Dieleman of the Institute for Health Metrics and Evaluation at the University of Washington's School of Medicine.

"The big picture trend suggests that all of the spending isn't essentially leading to fewer cases," said Dieleman, lead author of the study.

"In fact, we see that the health burden essentially hasn't changed at all over time, despite the huge increases in spending," he said. "It suggests that we have increased our spending a huge amount but we're not necessarily getting a lot more for it."

There are ways to mitigate back and neck pain at home before it becomes a larger problem.

It might seem counterintuitive, but staying moderately active by going for a walk can help reduce pain and prevent muscles from weakening.

"One of the biggest misconceptions is, 'I hurt myself. I need to go on complete bed rest and lie in bed and do nothing,' " Yao said. "That's been shown to really not be effective and ideally they need to try to maintain some form of activity as much as they can, and that's been shown to have positive results."

Eating healthier can not only reduce the inflammation that can lead to chronic back pain; it can also help someone lose excess weight, another factor of back pain.

Chronic back pain can be emotionally straining in addition to the physical symptoms.

To manage frustration, depression and other psychological side effects, Johns Hopkins Medicine recommends mindfulness and meditation as two nonsurgical methods for easing back pain. Taking breaks from activity when needed and being patient with yourself can prevent the condition from advancing.

Lower back pain can stem from a range of causes, from a mild strain to a traffic accident. If pain becomes something more serious, it's important to seek additional care from a doctor instead of self-medicating, Yao said. Doctors can recommend multiple treatments including muscle relaxants, injections and physical therapy.

Yao said the study highlights the extent to which society as a whole can improve on their muscle and joint health and ensure that patient care is at the forefront.

"Exercise is the last thing we do, eating right is the last thing we do," Yao said. "Society as a whole is so stressed and overworked and taxed out that health becomes really on the back burner.

"Patients have to take care of and responsibility for their own health. The more that a doctor can help facilitate that, the better."

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Low back and neck pain is costing us a fortune. Here's how to stop - jacksonprogress-argus

10 things to know about stem cell therapy – Newsd.in

New Delhi: The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions.

Stem cell treatments for brain or neural diseases like Parkinsons and Alzheimers disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way.

Undifferentiated cells that are able to differentiate and transform into any type of cells of the body when and where needed. They have an enormous potential to repair, heal and regenerate. Stem cells come from blood, bone marrow, umbilical cord blood and adipose tissue.

Autologous stem cell therapy: Patient receives stem cells from his/her own body

Allogeneic stem cell therapy: Patient receives the stem cells donated by another individual

Autologous stem cell therapy is better than allogeneic stem cell therapy as chances of mismatching are not there and they pose the minimum risk of immune rejection. Also, no side effects or adverse effects are seen as a persons own blood cells are used. They start the healing process immediately in a natural way.

The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. Stem cells can be obtained from the bone marrow, adipose tissues etc. Due to their tremendous potential to prevent and to treat various health conditions and to repair the injured tissues global research investigation is continuously being done as to explore the maximum advantage of these cell lines.

The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions. Stem cell treatments for brain or neural diseases like Parkinsons and Alzheimers disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way.

Depending upon the disease, different stem cell source can be used in a specific condition. The procedure may involve the extraction of stem cells from adipose tissue-derived stem cells with the combination of PRP (Platelet-rich plasma) or can be obtained from bone marrow that can differentiate into progenitor cells that differentiate into various other tissues which can help in the therapy.

The stem cells are isolated from the bone marrow or adipose tissues followed by their processing and enrichment under sterile conditions. These activated stem cells are placed back into the patients body at the target site for repairing the damaged tissue. It is necessary that the stem cells are injected in the specific area of injury as only then the desired results will be achieved.

Adipose stem cells are preferred over bone marrow stem cells as they are easy to isolate and contain a higher number of stem cells.

The stem cells injections are gaining much interest because it is devoid of the painful procedure, takes less time in comparison to surgery, there are no host and recipient rejections as stem cells are harvested from the patients body itself and a targeted delivery system is available.

The stem cells obtained are processed in a sophisticated stem cell lab and after activation is inserted back into the host with the help of intravenous, intramuscular, intraarterial, intradermal and intrathecal injections as per the requirement of the treatment process.

What is the use of anaesthetics and why? Usually, local anaesthetics are used during a stem cell procedure to numb the area but sometimes general anaesthesia is also given while extracting the stem cells from bone marrow. But it is necessary to find out what anaesthetic your doctor uses during orthopaedic stem cell treatments.

A number of anaesthetics have been found to kill the stem cells thus; the treatments end result will greatly depend on the use of anaesthetics. Some anaesthetics very well sync with the stem cell and hence, aid in the treatment.

Stem cells are to be extracted and processed in a clean room, under aseptic conditions maintaining a controlled environment. The doctor should explain the entire process and the number of viable stem cells infused into the patient during the process. Also, the precision of the injections to provide good quality of stem cells at the site of injury will help in better and faster recovery of the patients damaged area.

Cost of the treatment and its duration varies from one patient to another. The disease which needs to be cured, the severity, age factor, health condition, etc, define the duration of the therapy. One may respond during the treatment phase itself while the other may show results after a few sessions or weeks. Depending upon the disease diagnosed, the stem cells extracted, duration of the therapy, other adjuvants used in the process, the cost of the stem cell therapy can vary.

It is essential that after the stem cell therapy the patient should visit the stem cell doctor for recuperation therapies. The primary goals of such therapy is the prevention of secondary complications, analysis of the recovery of motor, sensory and all the bodily functioning, psychological support/counselling for depression, mood swings or anxiety etc. and reintegration into the community.

There can be different sets of precautions which need to be followed at various steps for the recovery of the damaged tissues. The treatment and post-treatment conditions may vary from person to person depending upon the disease and the severity.

Stem cell therapy has shown results in treating serious ailments like leukaemia, grafting tissues, autism, orthopaedic conditions and skin problems etc. Stem Cell Therapy has been successfully used in the treatment of around 80 serious disorders.

Survival rates among patients who received stem cell treatment are significantly high, whether cell donors are related or unrelated to them. With the ongoing research around the world, scientists are exploring new possibilities in which a number of life-threatening diseases can be prevented and cured hence, the stem cells have proved to be promising in the near future as many aspects are yet to be revealed.

Read: Egg stem cells do not exist, says researchers

-IANS

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10 things to know about stem cell therapy - Newsd.in

From Scorpion to Immunotherapy: City of Hope Scientists Repurpose Nature’s Toxin for First-of-Its Kind CAR T Cell Therapy to Treat Brain Tumors -…

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope scientists have developed and tested the first chimeric antigen receptor (CAR) T cell therapy using chlorotoxin (CLTX), a component of scorpion venom, to direct T cells to target brain tumor cells, according to a preclinical study published today in Science Translational Medicine. The institution has also opened the first in-human clinical trial to use the therapy.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

Glioblastoma (GBM), the most common type of brain tumor, is also among the most deadly of human cancers, according to the American Cancer Society. It is particularly difficult to treat because the tumors are disseminated throughout the brain. Efforts to develop immunotherapies, including CAR T cells, for GBM must also contend with a high degree of heterogeneity within these tumors.

For the study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets, including IL13R2, HER2 and EGFR. They found that CLTX bound to a greater proportion of patient tumors and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells in cell-based assays and in animal models without off-tumor targeting and toxicity.

Our chlorotoxin-incorporating CAR expands the populations of solid tumors potentially targeted by CAR T cell therapy, which is particularly needed for patients with cancers that are difficult to treat such as glioblastoma, said Christine Brown, Ph.D., City of Hopes Heritage Provider Network Professor in Immunotherapy and deputy director of T Cell Therapeutics Research Laboratory. This is a completely new targeting strategy for CAR T therapy with CARs incorporating a recognition structure different from other CARs.

Michael Barish, Ph.D., City of Hope professor and chair of the Department of Developmental and Stem Cell Biology, initiated the development of a CAR using chlorotoxin to target GBM cells. The peptide has been used as an imaging agent to guide GBM resection surgery, and to carry radioisotopes and other therapeutics to GBM tumors.

Much like a scorpion uses toxin components of its venom to target and kill its prey, were using chlorotoxin to direct the T cells to target the tumor cells with the added advantage that the CLTX-CAR T cells are mobile and actively surveilling the brain looking for appropriate targets, Barish said. We are not actually injecting a toxin, but exploiting CLTXs binding properties in the design of the CAR. The idea was to develop a CAR that would target T cells to a wider variety of GBM tumor cells than the other antibody-based CARs.

The notion is that the higher the proportion of tumor cells that one can kill at the beginning of treatment, the greater the probability of slowing down or stopping GBM growth and recurrence, Barish added.

Dongrui Wang, a doctoral candidate in City of Hopes Irell & Manella Graduate School of Biological Sciences, was the lead scientist to establish and optimize the CLTX-CAR T cell platform and to determine that cell surface protein matrix metalloprotease 2 is required for CLTX-CAR T cell activation. He added that while people might think the chlorotoxin is what kills the GBM cells, what actually eradicates them is the tumor-specific binding and activation of the CAR T cells.

Based on the promising findings of this study, the study team intends to bring this therapy to patients diagnosed with GBM with the hope of improving outcomes against this thus far intractable cancer. With recently granted Food and Drug Administration approval to proceed, the first-in-human clinical trial using the CLTX-CAR T cells is now screening potential patients.

This work was supported by the Ben & Catherine Ivy Foundation of Scottsdale, Arizona, and the clinical trial will be supported by The Marcus Foundation of Atlanta.

City of Hope, a recognized leader in CAR T cell therapies for glioblastoma and other cancers, has treated nearly 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 29 ongoing CAR T clinical trials, including CAR T trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent glioblastoma combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is the highest ranked cancer hospital in the West, according to U.S. News & World Reports Best Hospitals: Specialty Ranking. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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From Scorpion to Immunotherapy: City of Hope Scientists Repurpose Nature's Toxin for First-of-Its Kind CAR T Cell Therapy to Treat Brain Tumors -...