Category Archives: Stem Cell Treatment


Mesoblast shares fall on trials mixed results with heart injection treatment – Stockhead

Mesoblast (ASX:MSB) shares have dropped more than 12 per cent to a nearly one-month low after a six-year trial investigating using its stem cell treatment to treat advanced chronic heart failure delivered confusing results.

The injection of stem cells into the heart appeared to prevent deaths in patients with mild symptoms but not hospitalisations. That was the primary goal of the study, which Mesoblast says is the largest and most rigorous cell therapy trial ever performed.

In the Phase III trial involving 537 patients with advanced chronic heart failure, the group treated with an injection of rexlemestrocel-L had 60 per cent fewer heart attacks and strokes than those treated with a sham treatment.

Also, in the 206 patients with mild heart failure symptoms, those who received rexlemestrocel-L suffered 60 per cent fewer deaths than those in the placebo group.

But the trial failed to meet its predefined primary endpoint in that the treatment failed to reduce hospitalisations during the follow-up period, which lasted an average of 30 months.

At 11.36am, MSB shares were down 12.3 per cent to $3.98, their lowest level since mid-November.

But Mesoblast said that overall the study was incredibly positive and that it would seek regulatory approval in the United States as well as a large commercial partner to meet what it sees as a huge market opportunity.

On a conference call with analysts this morning, Mesoblast chief executive Dr Silviu Itescu said that rexlemestrocel-L had delivered a dramatic reduction in mortality that may change the paradigm of how patients with advanced cancer may be treated.

He said Mesoblast planned to meet again shortly with the US Food and Drug Administration and ask for drug approval of rexlemestrocel-L, (REVASCOR), which consists of an injection of 150 million mesenchymal precursor cells directly into the heart.

This is a very surprising finding and quite dramatic, he said. And I think that any therapy that has this degree of reduction in mortality in a well-conducted randomized placebo-controlled study of this size has to be taken seriously in terms of its ability to completely change the natural course of the disease.

So, we will be having those discussions. I think there are clear pathways towards accelerated approval.

Dr Itescu said that the rexlemestrocel-L injection may not have reduced recurrent hospitalisations because advanced heart failure patients are brittle and may need hospital treatment to be stabilised.

It appears our therapy does not have an impact on those types of outcomes, he said.

Mesoblast believes that the stem cell injection works to reduce severe inflammation in the heart muscle.

Other drugs in this patient population have reduced mortality by less than 20 per cent, Dr Itescu said.

The drug would be aimed at patients with class 2 heart disease mild symptoms such as shortness of breath or angina, but who did not yet have problems walking short distances.

That would be our target market in the patient journey, in terms of that being a very large market opportunity, Dr Itescu said.

Clearly, we will be looking for a strategic commercial partner that has strength and commitment in the commercial channels that will be required to make this product available to these large numbers of patients. We have ongoing discussions with a number of large pharmaceutical companies in this space.

Dr Itescu said that a rexlemestrocel-L injection would not displace other therapies, but would be a single delivery of a therapeutic that gives three o=o four years at least, of mortality benefit and reduction in major vascular events like heart attacks and stroke.

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Mesoblast shares fall on trials mixed results with heart injection treatment - Stockhead

Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade – GlobeNewswire

December 17, 2020 11:00 ET | Source: DelveInsight Business Research LLP

Los Angeles, USA, Dec. 17, 2020 (GLOBE NEWSWIRE) -- Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade

Glioblastoma Multiforme Clinical Trial Analysis and Pipeline Assessment report offers an analysis of 120+ companies working for the development of therapies for Glioblastoma Multiforme

DelveInsights Glioblastoma Multiforme (GBM) Pipeline Insight, 2020 report proffers a comprehensive coverage of all the companies under pipeline and growth prospects across Glioblastoma Multiforme pipeline, collaborations and agreements taking place, in-depth commercial assessment, and competitive analysis.

Some of the key highlights of Glioblastoma Multiforme Pipeline Report

Glioblastoma Multiforme pipeline is robust and possesses multiple potential drugs in late and mid-stage developments, which are yet to be launched. 120+ companies are dedicatedly working in advancing the GBM pipeline in the foreseeable future.

Some of the Glioblastoma Multiforme Pipeline Therapies

Regorafenib: Bayer Healthcare

Regorafenib is an orally-administered inhibitor of multiple kinases. Sold under the brand name, Stivarga, Regorafenib is approved for use in patients with hepatocellular carcinoma. The drug is currently under investigation for the treatment of Glioblastoma Multiforme which is mentioned. The molecule is in the phase III stage of clinical development.

CYNK 001: Celularity

CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. The U.S. Food and Drug Administration (FDA) recently cleared Celularitys Investigational New Drug (IND) Application for CYNK-001 in patients with glioblastoma multiforme (GBM).

Know more about GBM pipeline therapies and product profiles, request @ https://www.delveinsight.com/sample-request/glioblastoma-multiforme-gbm-pipeline-insight

Upcoming Glioblastoma Multiforme Therapies

Scope of Glioblastoma Multiforme Pipeline Therapeutics report

Key Questions regarding Current Glioblastoma Multiforme Treatment Landscape and Emerging Therapies Answered in the report

Table of Contents

Browse Detailed TOC, Emerging Drugs and Key Companies @ Glioblastoma Multiforme Drug Pipeline Landscape

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Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade - GlobeNewswire

Mesoblast shares savaged on poor COVID trial results – Sydney Morning Herald

Stem cell biotech Mesoblast has had its share price smashed after the company emerged from a trading halt on Friday to reveal its COVID-19 treatment trial was unlikely to meet its primary goal.

Shares in the biotech plunged 41 per cent at the start of trading before recovering some lost ground during the day to close 36 per cent lower at $2.41, shaving its market value by $800 million to $1.4 billion.

Mesoblast chief executive Silviu Itescu. The company was hoping to show its flagship product, remestemcel-L, would help patients with moderate to severe respiratory distress because of coronavirus.Credit:Credit: Arsineh Houspian

In a single-page ASX release, Mesoblast told investors that an independent data safety monitoring board had reviewed interim results from its coronavirus trial. The company was hoping to show its flagship product, remestemcel-L, could help patients with moderate to severe respiratory distress because of coronavirus.

On the same morning that Sydneysiders were thrown into major uncertainty due to a new outbreak of the virus on the northern beaches, Mesoblast noted the trial is not likely to meet the 30-day mortality reduction endpoint for the study.

The company will still complete the study, which had been aiming to recruit 300 patients, using the 223 patients already enrolled.

Chief executive Silviu Itescu insisted the news, while disappointing, was not the end of the road for the project.

He claimed that recruiting patients for the trial, which was run with the US National Institutes of Health, had been tough and that the most recent patients recruited to the trial were older than the earlier recruits.

He argued that as the pandemic wore on, many critically ill patients had also already received a number of experimental treatments and this could have impacted the results.

We have no ability to prevent other experimental drugs to be given to these patients, he said.

Its the second piece of tough news for Mesoblast, which is backed by high-profile investors including billionaire Alex Waislitz, to be released this week, after phase 3 trial results of its heart failure drug caused the stock to plunge at the start of the week. The company released data on Tuesday showing its other key product, Revascor, showed reduction in mortality of up to 60 per cent in heart failure patients.

However, the study missed its primary goal, which was to show that the drug reduced the incidence of trips to hospital for non-fatal heart failure events.

Mesoblast is also continuing to negotiate with the US Food and Drug Administration over approvals for its remestemcel-L product for the treatment of graft-versus-host disease. The product was blocked at the final hurdle this year when US regulators requested more data on the treatments effectiveness prior to giving the green light.

Shares in the company are still above where they started 2020, at $2.05 on January 2. The share price has swung wildly throughout the year, gaining or losing more than 30 per cent of its share price in a number of individual sessions as investors reacted quickly to corporate updates, particularly around COVID-19.

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Shares hit a high of $5.50 at the end of September on optimism for the COVID trial and its research pipeline.

Mesoblast raised $138 million from investors earlier this year in anticipation of scaling up manufacturing capabilities for the COVID-19 project.

It also entered into a partnership with pharmaceuticals giant Novartis for the development of its products for treating COVID-19 and other respiratory diseases, which included a $US50 million ($65.6 million) upfront payment, including $US25 million in equity in the business. Mesoblast reported a $US24.5 million loss for the September quarter.

Mr Itescu said nobody likes to not achieve an endpoint in a research project, but he expected the company to keep working with Novartis to comb through the research data and collaborate on future work on the treatment.

Novartis has partnered with us for many indications [uses of remestemcel-L], he said.

Analysts were reviewing their models for the stock on Friday morning, with Bell Potters team, which had a speculative buy recommendation on the stock, labelling the news very disappointing.

A concise wrap of the day on the markets, breaking business news and expert opinion delivered to your inbox each afternoon. Sign uphere.

Emma reports on healthcare companies for The Age and Sydney Morning Herald. She is based in Melbourne.

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Mesoblast shares savaged on poor COVID trial results - Sydney Morning Herald

Creative Medical Technology Holdings Announces Successful Application of ImmCelz Immunotherapy for Treatment of Stroke – KPVI News 6

PHOENIX, Dec. 16, 2020 /PRNewswire/ --Creative Medical Technology Holdings Inc., (OTC CELZ) announced today positive preclinical data supporting the utilization of its ImmCelz cell based immunotherapy for treatment of stroke. In an animal model of ischemia stroke, the middle cerebral artery ligation model, administration of ImmCelz resulted in 34% reduction in infarct volume, whereas control bone marrow mesenchymal stem cells reduced infarct volume by 21%. Additionally, improvements in functional recovery where observed using the Rotarod test. At 28 days after induction of stroke the animals receiving ImmCelz had superior running time (92% of non-stroke controls) compared to animals which received bone marrow mesenchymal stem cells (73% of non-stroke control). Animals that received saline had a running time that was 50% of non-stroke controls.

"The regenerative potential of immune cells that have been programmed by stem cells is a fascinating and novel area of research." Said Dr. Amit Patel, coinventor of ImmCelz, and board member of the Company. "Conceptual advantages of using reprogrammed T cells include higher migratory ability due to smaller size, as well as ability to replicate and potentially form "regenerative memory cells."

"This data, which is covered by our previous filed patents, such as no. 15/987739, Generation of autologous immune modulatory cells for treatment of neurological conditions, demonstrate that immune modulation via this stem cell based method may be a novel and superior way of addressing the $30 billion dollar market for stroke therapeutics1." Said Dr. Thomas Ichim, coinventor of the patent and Chief Scientific Officer of the Company. "The fact that this technology, which has priority back to 2017, is demonstrating such stunning results, motivates us to consider filing an Investigational New Drug Application for use in stroke."

Creative Medical Technology Holdings possesses numerous issued patents in the area of cellular therapy including patent no. 10,842,815 covering use of T regulatory cells for spinal disc regeneration, patent no. 9,598,673 covering stem cell therapy for disc regeneration, patent no. 10,792,310 covering regeneration of ovaries using endothelial progenitor cells and mesenchymal stem cells, patent no. 8,372,797 covering use of stem cells for erectile dysfunction, and patent no. 7,569,385 licensed from the University of California covering a novel stem cell type.

"While stroke historically has been a major area of unmet medical need, the rise in stroke cases , as well as the fact that younger people are increasingly falling victim to stroke, strongly motivates us to accelerate our developmental programs and to continue to explore participation of Big Pharma in this space." Said Timothy Warbington, President and CEO of the Company. "We are eager to replicate the existing experiments start compiling the dossier needed to take ImmCelz into humans using the Investigational New Drug Application (IND) route through the FDA."

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

Timothy Warbington, CEO CEO@ CreativeMedicalHealth.com

Creativemedicaltechnology.com http://www.StemSpine.com http://www.Caverstem.com http://www.Femcelz.com

1Stroke Management Market Size Forecasts 2026 | Statistics Report (gminsights.com)

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Creative Medical Technology Holdings Announces Successful Application of ImmCelz Immunotherapy for Treatment of Stroke - KPVI News 6

In COVID-19 Clinical Trials, Experts from Baptist Health’s Cancer Institutes Treat Patients With Mild or Severe Symptoms – Baptist Health South…

Offering more hope to patients with COVID-19, cancer experts throughout Baptist Health South Florida are launching new clinical trials. The trials, developed as a result of promising initial emergency and experimental COVID-19 therapies at Miami Cancer Institute on the Baptist Hospital campus in Miami and at the Eugene M. & Christine E. Lynn Cancer Institute at Boca Raton Regional Hospital, span the range of care from the most critically ill to those with mild symptoms.

Guenther Koehne, M.D., Ph.D., director and chief of Stem Cell Transplantation, Hematologic Oncology and Benign Hematology at Miami Cancer Institute.

Its exciting to lead these next-generation clinical trials, said Guenther Koehne, M.D., Ph.D., principal investigator of two of the studies, and director and chief of Stem Cell Transplantation, Hematologic Oncology and Benign Hematology at Miami Cancer Institute. We have learned much about COVID-19 since the pandemic began last winter and our hope is that these trials will lead to tremendous treatment options for patients.

Specialists at both centers are accomplished in many of the techniques and technologies that are doing double-duty as oncology and COVID-19 treatments and pivoted at the start of the pandemic to lead numerous COVID-19 clinical trials.

Trial to Save the Most Critical Patients

Miami Cancer Institute is leading a phase I/IIa clinical trial using mesenchymal stem cells for critically ill patients with COVID-19 induced respiratory failure. Mesenchymal stem cells are derived from umbilical cord lining tissue and aid in healing by regenerating damaged lung tissue. The trial is for hospitalized patients who are receiving oxygen therapy or who are on ventilation support and are not showing improvement.

Early in the pandemic, Miami Cancer Institute treated several patients with mesenchymal stem cells through single-use emergency approval from the U.S. Food and Drug Administration (FDA). The patients, who were among the most ill COVID-19 patients, recovered.

In our early experience with these umbilical cord lining stem cells, we had very promising results, Dr. Koehne said. We are very hopeful that the clinical trial will give us evidence that this treatment can save the lives of those who experience respiratory failure due to COVID-19.

Low-dose Radiation may Reverse Pneumonia

Both Lynn Cancer Institute and Miami Cancer Institute are participating in the PREVENT trial, Low-Dose Radiotherapy for Patients with SARS-COV2 (COVID-19) Pneumonia. With a single, low-dose of thoracic radiation, researchers hope that inflammation in the lungs is reduced and that patients with pneumonia associated with COVID-19 may not need to be placed on a ventilator.

Minesh Mehta, M.D., deputy director and chief of Radiation Oncology at Miami Cancer Institute.

We have seen this treatment option benefit many cancer patients, and the hope is that it also helps those affected by the virus, said Minesh Mehta, M.D., co-principal investigator, deputy director and chief of Radiation Oncology at Miami Cancer Institute.

Patients eligible for the study include hospitalized men and women ages 50 and up who are diagnosed with COVID-19 and pulmonary pneumonia but who are not on ventilators.

This trial gives us the opportunity to administer potentially effective treatment before the need for ICU placement or mechanical ventilation, said Michael Kasper, M.D., co-principal investigator and director of Radiation Oncology at Lynn Cancer Institute.

Radiation therapy has shown a reduction in inflammation in a number of conditions, including viral pneumonia, autoimmune disorders and degenerative joint disorders. At much higher doses, it is also used to treat cancer.

Trial to Shorten Recovery and Reduce Symptoms For Those With Mild Disease

Michael Kasper, M.D., director of Radiation Oncology at Lynn Cancer Institute.

Miami Cancer Institute is also enrolling patients in a phase 2 clinical trial known as BLAZE-4, which continues previous work using a monoclonal antibody, bamlanivimab, to treat patients with milder cases of COVID-19. The Institute participated in the phase 1 study, BLAZE-1, which resulted in Emergency Use Authorization (EUA) status by the FDA.

Bamlanivimab is for patients who are COVID-19 positive but are not hospitalized and have mild symptoms. It must be administered within 72 hours of a positive test result.

The BLAZE-1 study showed a lower subsequent hospitalization rate among those who received the drug versus those who received a placebo, and may reduce the viral load, leading to better outcomes. The BLAZE-4 trial will evaluate the efficacy and safety of bamlanivimab both on its own and in combination with another monoclonal antibody. The drugs work by prohibiting the spread of the virus to additional cells in the body.

We have reached a new level of sophistication trying to treat the virus before it makes you really sick, Dr. Koehne said. Despite the prospect of having COVID-19 vaccines, which are intended to prevent us from getting sick, we need to stay focused on the treatment of those individuals who are symptomatic from the virus.

Tags: COVID-19, Lynn Cancer Institute, Miami Cancer Institute

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In COVID-19 Clinical Trials, Experts from Baptist Health's Cancer Institutes Treat Patients With Mild or Severe Symptoms - Baptist Health South...

1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive – NPR

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says. Victoria Gray hide caption

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says.

The last thing a lot of people want to do these days is get on a plane. But even a pandemic would not stop Victoria Gray. She jumped at the chance to head to the airport this summer.

"It was one of those things I was waiting to get a chance to do," says Gray.

She had never flown before because she was born with sickle cell disease. She feared the altitude change might trigger one of the worst complications of the devastating genetic disease a sudden attack of excruciating pain.

But Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

About a year after getting the treatment, it was working so well that Gray felt comfortable flying for the first time. She went to Washington, D.C., to visit her husband, who has been away for months on deployment with the National Guard.

"It was exciting. I had a window. And I got to look out the window and see the clouds and everything," says Gray, 35, of Forest, Miss.

Gray wore a mask the whole time to protect herself against the coronavirus, kept her distance from other people at the airport, and arrived happily in Washington, D.C., even though she's afraid of heights.

"I didn't hyperventilate like I thought I would," Gray says, laughing as she recounts the adventure in an interview with NPR.

NPR has had exclusive access to follow Gray through her experience since she underwent the landmark treatment on July 2, 2019. Since the last time NPR checked in with Gray in June, she has continued to improve. Researchers have become increasingly confident that the approach is safe, working for her and will continue to work. Moreover, they are becoming far more encouraged that her case is far from a fluke.

At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. "Patients appear to be cured of their disease, which is simply remarkable."

Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven't been followed long enough to report any results, officials say.

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.

Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray and the two other sickle cell patients haven't had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven't needed the regular blood transfusions that had been required to keep them alive.

"It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life," says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray's doctor and is helping run the study.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.

The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

"It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia but potentially can be used in other disorders," Frangoul says.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

The researchers stress that they will have to follow Gray and many other patients for a lot longer to be sure the treatment is safe and that it keeps working. But they are optimistic it will.

Gray hopes so too.

"It's amazing," she says. "It's better than I could have imagined. I feel like I can do what I want now."

The last year hasn't always been easy for Gray, though. Like millions of other Americans, she has been sheltering at home with three of her children, worrying about keeping them safe and helping them learn from home much of the time.

"I'm trying to do the things I need to do while watch them at the same time to make sure they're doing the things they need to do," Gray says. "It's been a tough task."

But she has been able do other things she never got to do before, such as watch her oldest son's football games and see her daughter cheerleading.

"This is really a life-changer for me," she says. "It's magnificent."

She's now looking forward to going back to school herself, learning to swim, traveling more when the pandemic finally ends, and watching her children grow up without them worrying about their mother dying.

"I want to see them graduate high school and be able to take them to move into dorms in college. And I want to be there for their weddings just everything that the normal people get to do in life. I want to be able to do those things with my kids," she says. "I can look forward now to having grandkids one day being a grandmama."

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1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive - NPR

Impact Analysis of Covid-19 On Stem Cell Therapy Market to Witness Steady Growth 2027 | Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon…

Global Stem Cell Therapy Market Growth To Increase Manifold By 2027 A fundamental overview of the Stem Cell Therapy Market niche is provided in the Stem Cell Therapy Market report accompanying definitions, classifications, applications along with industry chain framework. The Stem Cell Therapy market report provides a broad assessment of the required market dynamics and the latest trends. It also highlights regional markets, prominent market players, multiple market segments [products, applications, end users, and key regions] and subsectors that broadly consider numerous departments along with applications.

NOTE:Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Request a sample copy of the report: https://www.coherentmarketinsights.com/insight/request-sample/2848

Top Key Players Global Stem Cell Therapy Market Competition: Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Anterogen Co. Ltd., Pharmicell Co., Inc., and Stemedica Cell Technologies, Inc.

The market can be segmented into: By Cell Source:Adult Stem CellsInduced Pluripotent Stem CellsEmbryonic Stem CellsOthersGlobal Stem Cell Therapy Market, By Application:Musculoskeletal DisordersWounds and InjuriesCancerAutoimmune disordersOthers

Furthermore, the report acknowledges that in this growing and immediately intensifying market situation, the most recent advertising and marketing details are critical to determining the performance of the forecast period and making an essential choice for the profitability and growth of the Stem Cell Therapy market. Do it. Additionally, the report covers various factors influencing the growth of the Stem Cell Therapy market during the forecast period. Additionally, this particular analysis will also determine its impact on individual segments of the market.

To identify the growth opportunities in the market, the report has been segmented into regions that are growing faster than the overall market. This region was focused on regions with slower growth rates than the global market. Each geographic segment of the Stem Cell Therapy market has been independently investigated, with price, distribution and demand data specifically for North America (US, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), and Asia-region markets. . Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia, etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).

Research purpose:

Provides strategic profiling of key players in the market, comprehensively analyzes core competencies, and derives the competitive landscape of the market.

Provides insight into factors influencing market growth. Stem Cell Therapy market analysis based on various factors such as price analysis, supply chain analysis, Porter Five Force analysis, etc.

It provides detailed analysis of the market structure with forecasts for various segments and sub-segments of the global Stem Cell Therapy market.

Provides a country level analysis of the market in relation to its current market size and future outlook.

Provides country-level analysis of the market by application, product type and sub-segment.

Provides historical and forecast revenue for the market segment and sub-segments in relation to the four major regions and countries in North America, Europe, Asia and other countries.

Track and analyze competitive developments such as joint ventures, strategic alliances, new product development and research and development in the global Stem Cell Therapy Market.

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The chapters covered in the research report are as follows:

Chapter 1, 2: Targets of the Global Stem Cell Therapy Market, encompassing market introduction, product images, market summary, and development scope.

Chapter 3, Chapter 4: Global Market Competition, Sales Volume, and Market Profit by Manufacturer.

Chapters 5, 6, 7: Global Supply (Production), Consumption, Exports, Imports by Regions such as USA, Asia Pacific, China, India, Japan. From 2015 to 2024, we conduct regional market research based on regional sales rate and market share.

Chapters 8, 9, 10: Global Market Analysis by Application, Cost Analysis, Marketing Strategy Analysis, Distributor/Trader

Chapters 11, 12: Market Information and Research Conclusions, Appendix and Data Sources.

The market report also primarily identifies additional useful and useful information about the industry, including the Stem Cell Therapy market development trends analysis, return on investment, and feasibility analysis. Additionally, SWOT analysis is distributed in the report to analyze the growth of key global market players in the Stem Cell Therapy market industry.

In addition, the research report investigates:

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Impact Analysis of Covid-19 On Stem Cell Therapy Market to Witness Steady Growth 2027 | Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon...

Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma – Targeted Oncology

High-dose melphalan (Evomela) followed by autologous stem cell transplant (ASCT) has demonstrated significant efficacy for the treatment of patients with multiple myeloma. The agent has improved progression-free survival (PFS) in patients when administered as upfront therapy to patients aged 66 years or younger.

The median PFS shown with melphalan plus ASCT in a phase IFM/DFCI 2009 study was 50 months versus 26 months when compared with the standard of care treatment regimen lenalidomide (Revlimib), bortezomib (Velcade), and dexamethasone (RVD) alone hazard ratio for disease progression or death, 0.65;P<.001). As the treatment strategy continues to be explored in patients with multiple myeloma, researchers have now begun to investigate an outstanding biological question of whether the alkylating agent causes an increased amount of DNA damage.

An analysis presented during the virtual 2020 American Society of Hematology (ASH) Annual Meeting showed that between the time of diagnosis and relapse, patients treated with high-dose melphalan had an increased number of point mutations. It was unclear from this research how this result translated to treatment selection and sequencing.

In an interview with Targeted Oncology, Mehmet Samur, PhD, senior research scientist, Dana-Farber Cancer Institute, discussed the investigation of high-dose melphalan following ASCT in patients with multiple myeloma and shared insights into how the ongoing questions can be explored in the future.

TARGETED ONCOLOGY: Can you explain what was demonstrated prior with high-dose melphalan followed by ASCT in patients with multiple myeloma?

Sumar: The clinical part of the phase 3 study was published previously. It showed that when you do RVD plus high-dose melphalan following by stem cell treatment, patients do significantly better than patients who get RVD alone. Adding high-dose melphalan increased the PFS benefit by around 12 months.

TARGETED ONCOLOGY: Can you provide background on your analysis of Melphalan for patients with multiple myeloma?

Sumar: Melphalan is an alkylating agent. Because of the way the agent works, we always think that it creates more DNA damage. The study that we presented at ASH was questioning whether this was true or not.

We collected DNA sequencing data from patients who were treated with RVD followed by high-dose Melphalan and a bone marrow transplant. We had a total of 25 patients, and we collected data at the time of diagnosis and the time of relapse. To compare this compilation, we also collected data from 43 patients from the IFM/DFCI 2009 study who only received RVD. We also collected data at diagnosis and relapse in the 43 patients. Genomic alterations were compared at diagnosis and relapse for patients who were injected with high-dose Melphalan and RVD versus patients who were only treated with RVD.

TARGETED ONCOLOGY: What were the findings from this study?

Sumar: We found that patients who got high-dose melphalan plus RVD followed by transplant accumulated more point mutations. To be precise, they accumulated around 10,000 new mutations between diagnosis and relapse at 5 years. For RVD patients, there were around 4500 new point mutations. The study showed that treating patients with high-dose Melphalan is increasing the mutational load by about 2.9-fold at the time of relapse.

TARGETED ONCOLOGY: What are the implications of these findings?

Sumar: There are a couple of things that we see from our study. One point is that we only saw point mutations. We didnt see any large-scale DNA alterations. This suggests that our patients who are treated with high-dose melphalan are more likely to experience changes.

In terms of the pathways that are mutated, the DNA damage repair pathway is more frequently mutated between diagnosis and relapse in patients treated with high-dose melphalan. We think that if we combine inhibitors that can overcome the selection of DNA damage repair pathway mutations, those patients may get additional benefit from the treatment.

We dont have clear data yet on whether this increased mutational load is something that is bad for patients. Even though these patients have more mutations, overall survival times are similar between the 2 arms. C outcomes are not impacted by the increased number of mutation so far.

TARGETED ONCOLOGY: What plan are underway to further this research?

Sumar: We are expanding our study in multiple ways. There is no clear data set we can get answers to our ongoing questions yet. We have reached out to our partners around the world to see if we can come up with a cohort to investigation. Also, we are looking at impact of these mutational load increase on other features like secondary cancer rate.

TARGETED ONCOLOGY: The understanding of gene mutations in myeloma is evolving. Can you discuss the current role of genomic testing?

Sumar: It has been shown in many studies that genetic testing at diagnosis can tell us which patients are high risk and which are low risk. Studies have also shown that patients who have loss of p53 or with deletion 17p will have bad outcomes.

There was a study published last year in the Journal of Clinical Oncology showing which patients with myeloma would have a lower risk. The study also shows that there are certain genomic features prolong survival time in patients.

We have different genomic tools that we can use to look at these different alterations and assess patient risk. Today, I think people are looking at these alterations from all different angles to plan stratification in upcoming clinical trials.

TARGETED ONCOLOGY: In your opinion, what change will we see in the myeloma treatment landscape in the next 5 years?

Sumar: There are a lot of studies looking at new treatment. Everyone is carefully watching out for data on new treatment options like chimeric antigen receptor T-cell therapy, bispecific antibodies, and monoclonal antibodies. It looks like these agents are providing benefit to patients, but they are at the very early stages of research.

Reference:Attal M, Lauwers-Cances V, Hulin C, et al. Lenalidomide, bortezomib, and dexamethasone with transplantation for myeloma. N Engl J Med. 2017; 376(14):1311-1320. doi: 10.1056/NEJMoa1611750

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Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma - Targeted Oncology

Straight from the Heart: Cooper’s Story – WBIR.com

In just one year, 4-year-old Cooper Stansbury has conquered six rounds of Chemotherapy, two stem cell transplants and 14 days of Proton Therapy.

KNOXVILLE, Tenn. In August, we introduced you to 4-year-old Cooper Stansbury.

Law enforcement from around the area came together to encourage him as he's battling cancer.

In November of 2019, Cooper was diagnosed with neuroblastoma.

"I'll never forget that day. That was the worst day of our lives," said Cooper's father, Sam Stansbury. "My wife collapsed. Obviously, she was falling apart and all I wanted to know is what do we do now, what do we do next."

For more than a year, Cooper has endured treatment after treatment.

Cooper has conquered six rounds of Chemotherapy, two stem cell transplants and 14 days of Proton Therapy.

"The Power of Play" helps Cooper stay positive and decreases anxiety.

"Sometimes they do have to go through hard procedures that might hurt or be scary," said East Tennessee Children's Hospital Child Life Specialist Anna Taylor. "So, I can help distract and help them understand why they are going through it and help them cope and get through the procedure."

Cooper's father said "he's tough as nails."

Just like the toy race cars on his track, nothing slows cooper down.

Cooper is now on cycle 4 of immunotherapy.

"To see him now after everything he's gone through," Cooper's father said. "We're very hopeful."

As Cooper continues to fight, his entire family is looking forward to better days and more play days at home.

"I know he is destined for greatness, without a doubt," he said.

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Straight from the Heart: Cooper's Story - WBIR.com

Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL – Cancer Network

The allogeneic off-the-shelf CD22-directed T-cell product, UCART22, showed early signs of activity and no evidence of unexpected toxicities at 2 dose levels for adult patients with relapsed/refractory CD22-positive B-cell acute lymphoblastic leukemia, according to the results of a study presented during the 2020 ASH Annual Meeting.1

In the phase 1 BALLI-01 (NCT04150497) dose-escalation and dose-expansion study, 2 patients at the 1 x 105 cells/kg dose achieved a complete remission (CR) with incomplete hematologic recovery on day 28. One of these patients attained a minimal residual disease (MRD)positive CR at day 42 followed by subsequent inotuzumab ozogamicin (Besponsa) and then transplant.

One patient at dose level 2, 1 x 106 cells/kg, experienced a significant bone marrow blast reduction at day 28, followed by disease progression.

No patients experienced dose-limiting toxicities (DLTs), immune effector cellassociated neurotoxicity syndrome (ICANS), graft-versus-host disease (GVHD), adverse effects (AE) of special interest (AESI), a UCART22-related AE that was grade 3 or higher, or a serious AE (SAE).

UCART22 showed no unexpected toxicities at the doses of 1 x 105 cells/kg and 1 x 106 cells/kg with fludarabine and cyclophosphamide lymphodepletion, lead study author Nitin Jain, MD, an assistant professor in the Department of Leukemia, The University of Texas MD Anderson Cancer Center, said in a virtual presentation during the meeting. Host immune recovery was observed early, and the addition of alemtuzumab [Lemtrada] to fludarabine and cyclophosphamide lymphodepletion is currently being explored with the goal to achieve deeper and more sustained T-cell depletion and to promote expansion and persistence of UCART22.

Standard treatment for adult patients with B-cell ALL includes multiagent chemotherapy with or without allogeneic stem cell transplant. However, 30% to 60% of patients with newly diagnosed B-cell ALL who achieve a CR will relapse, and the expected 5-year survival rate for those with relapsed/refractory disease is approximately 10%.

Previously, UCART19, when paired with lymphodepletion using fludarabine, cyclophosphamide, and alemtuzumab, was found to show efficacy in this patient population.2

CD22 is an FDA-approved therapeutic target in B-cell ALL. UCART22 is an immediately available, standardized, manufactured agent with the ability to re-dose, and its CAR expression redirects T cells to tumor antigens, Jain explained.

Moreover, through its mechanism of action, TRAC becomes disrupted using Transcription activator-like effector nucleases (Talen) technology to eliminate TCR from cell surface and reduce the risk of GVHD. CD52 is also disrupted with the use of Talen to eliminate sensitivity to lymphodepletion with alemtuzumab. Finally, there is a CD20 mimotope for rituximab (Rituxan) as a safety switch, Jain added.

UCART22 has also demonstrated in vivo antitumor activity in immune-compromised mice that were engrafted with CD22-positive Burkitt lymphoma cells in a dose-dependent manner.

In the dose-escalation/dose-expansion BALLI-01 study, investigators are enrolling up to 30 patients in a modified Toxicity Probability Interval design. There are 3 cohorts, which have 2 to 4 patients on each cohort: 1 x 105 cells/kg (dose level 1), 1 x 106 cells/kg (dose level 2), and 5 x 106 cells/kg. The focus of the dose-escalation phase of the trial was to determine the maximum-tolerated dose (MTD) and the recommended phase 2 dose (RP2D) before heading into the dose-expansion portion of the trial.

To be eligible for enrollment, patients must have been between 18 and 70 years old, have acceptable organ function, an ECOG performance status of 0 or 1, at least 90% of B-cell ALL blast CD22 expression, and had previously received at least 1 standard chemotherapy regimen and at least 1 salvage regimen.

End points of the trial included safety and tolerability, MTD/R2PD, investigator-assessed response, immune reconstitution, and UCART22 expansion and persistence.

The lymphodepletion regimens were comprised of fludarabine (at 30 mg/m2 x 4 days) plus cyclophosphamide (1 g/m2 x 3 days); the study has since been amended to include the regimen of fludarabine (at 30 mg/m2 x 3 days), cyclophosphamide (500 g/m2 x 3 days), and alemtuzumab (20 mg/day x 3 days) and is currently enrolling patients.

Following screening, lymphodepletion, and UCART22 infusion, patients underwent an observation period for DLTs with a primary disease evaluation at 28 days; additional efficacy evaluations occurred at 56 days and 84 days. Patients were followed for 2 years and continued to be assessed for long-term follow-up.

As of July 1, 2020, 7 patients were screened, of which 1 patient failed and 6 were therefore enrolled on the study. One patient discontinued therapy before receiving UCART22 due to hypoxia from pneumonitis that was linked with lymphodepletion. Five patients were treated with UCART22 at dose level 1 (n = 3) and dose level 2 (n = 2).

The median age of participants was 24 years (range, 22-52), 3 of the 5 patients were male, and 3 had an ECOG performance status of 0. The median number of prior therapies was 3 (range, 2-6), and there were a median 35% bone marrow blasts (range, 10%-78%) prior to lymphodepletion.

Three patients had complex karyotype and 2 had diploid cytogenetics. One patient each had the following molecular abnormalities: CRLF2, CRLF2 and JAK2, CDKN2A loss, KRAS and PTPN11, and IKZF1. Only 1 patient had undergone haploidentical transplant. Four patients previously received prior CD19- or CD22-directed therapy, including blinatumomab (Blincyto), inotuzumab ozogamicin (Besponsa), and CD19-directed CAR T-cell therapy. At study entry, 3 patients had refractory disease and 2 patients had relapsed disease.

Grade 3 or higher treatment-emergent AEs (TEAEs), which were unrelated to study treatment, included hypokalemia, anemia, increased bilirubin, and acute hypoxic respiratory failure. Also not related to UCART22, 3 patients experienced 4 treatment-emergent SAEs: porta-hepatis hematoma, sepsis, bleeding, and sepsis in the context of disease progression. No treatment discontinuations due to a treatment-related TEAE were reported.

The patient who achieved a CR followed by transplant was a 22-year-old male who had undergone 2 prior treatments for B-cell ALL and received UCART22 at a dose of 1 x 105 cells/kg. He did not experience CRS, ICANS, GVHD, nor a SAE, and all TEAEs were grade 1.

Jain also noted that host T-cell constitution was observed in all patients within the DLT observation period. UCART22 was also not detectable through flow cytometry or molecular analysis, the latter of which was at dose level 1 only.

References:

1. Jain N, Roboz GJ, Konopleva M, et al. Preliminary results of BALLI-O1: a phase I study of UCART22 (allogeneic engineered T cells expressing anti-CD22 chimeric antigen receptor) in adult patients with relapsed/refractory anti-CD22+ B-cell acute lymphoblastic leukemia (NCT04150497). Presented at: 2020 ASH Annual Meeting and Exposition; December 4-8, 2020; Virtual. Abstract 163.

2. Benjamin R, Graham C, Yallop D, et al. Preliminary data on safety, cellular kinetics and anti-leukemic activity of UCART19, an allogeneic anti-CD19 CAR T-cell product, in a pool of adult and pediatric patients with high-risk CD19+ relapsed/refractory b-cell acute lymphoblastic leukemia. Blood. 2018;132(suppl 1):896. doi:10.1182/blood-2018-99-111356.

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Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL - Cancer Network