Archive for the ‘Bone Marrow Stem Cells’ Category

Since losing her cousin to leukemia during childhood, Heather Lynn made it her mission to ensure others battling blood cancers get a second chance at life. Earlier this year, the St. Louis native fulfilled that life purpose: saving a stranger by donating her stem cells.

Five years ago, Lynn became the director of global special events for DKMS, the worlds largest bone marrow and blood stem cell donor center, and registered as a potential donor with the hope that someday she could give a blood cancer patient what her cousin didnt have a second chance at life and more time with the patients family.

Amid this years coronaviral pandemic, Lynn received the life-changing call from a colleague that she was a match for a 58-year-old man battling acute myeloid leukemia. I screamed with joy, Lynn recalls. I was a match for someone with blood cancer and was about to be the first employee at DKMS to donate and ultimately save someones life. After the call, Lynn realized she would be giving more than stem cells: I was giving something much bigger: hope.

Despite the uncertainty surrounding COVID-19, Lynn felt a strong sense of purpose to help this man and donated her stem cells to save his life. I have seen how much someones life can change with a blood cancer diagnosis, Lynn says. The fear, the pain, the loss it can be devastating. I have spent the past five years working to elevate the message about donating and how easy it is to sign up and give back it simply requires swabbing the inside of each cheek for 60 seconds.

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St. Louis Native Heather Lynn Discusses Motivation for Donating Stem Cells - Ladue News

Lurbinectedin is being studied in a number of diseases, but in lung cancer it has a more favorable side effect profile compared with topotecan, said Apar Kishor Ganti, MD, University of Nebraska Medical Center.

Lurbinectedin is being studied in a number of diseases, but in lung cancer it has a more favorable side effect profile compared with topotecan, said Apar Kishor Ganti, MD, professor of internal medicine, Division of Oncology & Hematology, University of Nebraska Medical Center.

Are there other tumor types where lurbinectedin seems to hold promise?

So, lurbinectedin is being studied in other diseases like breast cancer, mesothelioma, chronic lymphocytic leukemia, among others. But the difference in these other conditions compared to small cell [lung cancer] is there are other treatment options that are reasonably effective in these other cancers, unlike in small cell, so that's where it becomes much more important in in this particular setting.

One other reason why lurbinectedin may be effective is, like I told you earlier, there is a group of cells that seem to be shielded from chemotherapy. We call them cancer stem cells. And there are some lab data that suggests that lurbinectedin may inhibit cancer stem cells, as well. Again, this is all preliminary data. And we don't necessarily know if that occurs in humans or not, but those are some of the hypothesized mechanisms of action.

What other advantages are there of lurbinectedin over topotecan?

One of the other advantages of lurbinectedin over topotecan is that topotecan has to be given 5 days in a row, whereas lurbinectedin is given just once every 3 weeks. And the side effect profile of lurbinectedin seems to be favorable. The main side effect of lurbinectedin is bone marrow suppression, anemia, leukopenia, neutropenia, [and] thrombocytopenia, but they seem to occur in about 5% to 10% of patients. And so, that's another possible advantage of lurbinectedin over for some of the other drugs that are available.

As far as small cell lung cancer itself is concerned, even though there is a lot of research going on in small cell, multiple different drugs have been triedtargeted therapies, immunotherapythere is some evidence to suggest that immunotherapy helps with chemotherapy in the frontline setting. But immunotherapy by itself in patients who have failed chemotherapy does not seem to be much more effective. People have tried targeted therapies, again, not one of them has shown to have any meaningful benefit for these patients. So that has been very disappointing.

There have been multiple drugs that have been studied. Unfortunately, none of them have had a significant benefit so far. So, it's a fairly difficult to treat disease. And like I mentioned earlier, even though it seems to respond quite well to initial chemotherapymost patients relapse and very few are cured even if they present with very early stage disease. And that's why it's a very challenging disease to treat.

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Dr Apar Kishor Ganti Outlines the Effectiveness of Lurbinectedin and Benefits Over Competition - AJMC.com Managed Markets Network

Summary

MSK researchers have uncoveredan important finding about the relationship between the microbiota and the immune system, showing for the first time that the concentration of different types of immune cells in the blood changes in relation to the presence of different bacterial strains in the gut.

In recent years, the microbiota the community of bacteria and other microorganisms that live on and in the human body has captured the attention of scientists and the public, in part because its become easier to study. It has been linked to many aspects of human health.

A multidisciplinary team from Memorial Sloan Kettering has shown for the first time that the gut microbiota directly shapes the makeup of the human immune system. Specifically, their research demonstrated that the concentration of different types of immune cells in the blood changed in relation to the presence of different bacterial strains in the gut. The results of their study, which used more than ten years of data collected from more than 2,000 patients, is being published November 25, 2020, in Nature.

The scientific community had already accepted the idea that the gut microbiota was important for the health of the human immune system, but the data they used to make that assumption came from animal studies, says Sloan Kettering Institute systems biologist Joao Xavier, co-senior author of the paper together with his former postdoc Jonas Schluter, who is now an assistant professor at NYU Langone Health. At MSK, we have a remarkable opportunity to follow how the composition of the microbiota changes in people being treated for blood cancers, Dr. Xavier adds.

(From left) Researchers Emily Fontana, Luigi Amoretti, Joao Xavier, Roberta Wright, and Jonas Schluter in the lab.

The data that were used in the study came from people receiving allogeneic stem cell and bone marrow transplants (BMTs). After strong chemotherapy or radiation therapy is used to destroy cancerous blood cells, the patients blood-forming system is replaced with stem cells from a donor. For the first few weeks until the donors blood cells including the white blood cells that make up the immune system have established themselves, the patients are extremely vulnerable to infections. To protect them during this time, patients are given antibiotics.

But many of these antibiotics have the unwanted side effect of destroying healthy microbiota that live in the gut, allowing dangerous strains to take over. When the patients immune system has reconstituted, the antibiotics are discontinued, and the gut microbiota slowly starts to grow back.

The parallel recoveries of the immune system and the microbiota, both of which are damaged and then restored, gives us a unique opportunity to analyze the associations between these two systems, Dr. Schluter says.

For more than ten years, members of MSKs BMT service have regularly collected and analyzed blood and fecal samples from patients throughout the BMT process. The bacterial DNA were processed by the staff at MSKs Lucille Castori Center for Microbes, Inflammation, and Cancer, which played a key role in creating the massive microbiota dataset. Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet, Dr. Xavier notes. The result of collecting them is that we have a unique dataset with thousands of datapoints that we can use to ask questions about the dynamics of this relationship.

This wider effort has been led by Marcel van den Brink, Head of the Division of Hematologic Malignancies, and a team of infectious disease specialists, BMT doctors, and scientists. For a fair number of patients, we collected daily samples so we could really see what was happening day to day, Dr. van den Brink says. The changes in the microbiota are rapid and dramatic, and there is almost no other setting in which you would be able to see them.

Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet.

Previous research using samples collected from this work has looked at how the gut microbiota affects patients health during the BMT process. A study published in February 2020 reported that having a greater diversity of species in the intestinal microbiota is associated with a lower risk of death after a BMT. It also found that having a lower diversity of microbiota before transplant resulted in a higher incidence of graft-versus-host disease, a potentially fatal complication in which the donor immune cells attack healthy tissue.

The databank that the MSK team created contains details about the types of microbes that live in the patients guts at various times. The computational team, including Drs. Schluter and Xavier, then used machine learning algorithms to mine electronic health records for meaningful data. The data from the health records included the types of immune cells present in the blood, information about the medications that patients were given, and the side effects patients experienced. This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota, Dr. van den Brink says.

Analyzing this much data was a huge undertaking. Dr. Schluter, who at the time was a postdoctoral fellow in Dr. Xaviers lab, developed new statistical techniques for this. Because experiments with people are often impossible, we are left with what we can observe, Dr. Schluter says. But because we have so many data collected over a period of time when the immune system of patients as well as the microbiome shift dramatically, we can start to see patterns. This gives us a good start toward understanding the forces that the microbiota exerts on the rebuilding of the immune system.

This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota.

The purpose of this study was not to say whether certain kinds of microbes are good or bad for the immune system, Dr. Xavier explains, adding that this will be a focus of future research. Its a complicated relationship. The subtypes of immune cells we would want to increase or decrease vary from day to day, depending on what else is going on in the body. Whats important is that now we have a way to study this complex ecosystem.

The researchers say they also plan to apply their data to studying the immune system in patients receiving other cancer treatments.

Originally posted here:
MSK Study Is the First to Link Microbiota to Dynamics of the Human Immune System - On Cancer - Memorial Sloan Kettering

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Leading players of Hematopoietic Stem Cell Transplantation (HSCT) including:

Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc

Global Hematopoietic Stem Cell Transplantation (HSCT) Segmentation:

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By Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT).

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Global Hematopoietic Stem Cell Transplantation (HSCT) Market Trends, Opportunities, Drivers, Challenges and Forecast to 2026 - The Market Feed

Severe infections like malaria cause short and long-term damage to precursor blood cells in mice, but some damage could be reversed, find researchers.

A team led by researchers from Imperial College London and The Francis Crick Institute have discovered that severe infections caused by malaria disrupt the processes that form blood cells in mice. This potentially causes long-term damage that could mean people who have recovered from severe infections are vulnerable to new infections or to developing blood cancers.

The team also discovered that the damage could be reduced or partially reversed in mice with a hormone treatment that regulates bone calcium coupled with an antioxidant. The research could lead to new ways of preventing long-term damage from severe infections including malaria, TB and COVID-19.

The research is published today in Nature Cell Biology.

First author Dr Myriam Haltalli, who completed the work while at the Department of Life Sciences at Imperial, said: We discovered that malaria infection reprograms the process of blood cell production in mice and significantly affects the function of precursor blood cells. These changes could cause long-term alterations, but we also found a way to significantly reduce the amount of damage and potentially rescue the healthy production of blood cells.

Blood is made up of several different cell types, that all originate as haematopoietic stem cells (HSCs) in the bone marrow. During severe infection, the production of all blood cells ramps up to help the body fight the infection, depleting the HSCs.

Now, the team has shown how infections also damage the bone marrow environment that is crucial for healthy HSC production and function. They discovered this using advanced microscopy technologies at Imperial and the Crick, RNA analyses led by the Gottgens group at Cambridge University, and mathematical modelling led by Professor Ken Duffy at Maynooth University.

The mice developed malaria naturally, following bites from mosquitoes carrying Plasmodium parasites, provided by Dr Andrew Blagborough at Cambridge University. The researchers subsequently observed the changes in the bone marrow environment and the effect on HSC function.

Within days of infection, blood vessels became leaky and there was a dramatic loss in bone-forming cells called osteoblasts. These changes appear strongly linked to the decline in the pool of HSCs during infection.

Lead author Professor Cristina Lo Celso, from the Department of Life Sciences at Imperial, said: We were surprised at the speed of the changes, which was completely unexpected. We may think of bone as an impenetrable fortress, but the bone marrow environment is incredibly dynamic and susceptible to damage.

Reducing the pool of HSCs can have several consequences. In the short-term, it appears to particularly affect the production of neutrophils white blood cells that form an essential part of the immune system. This can leave patients vulnerable to further infections, with potentially long-term consequences for the functioning of the immune system.

In the long term, the pool of HSCs may remain below normal levels, which can increase the chances of the patient developing blood cancers like leukaemia.

By injecting fluorescent molecules (magenta) that would normally remain in circulation and taking a series of images over time, intravital microscopy revealed that infected mice had very leaky vessels with the contents of the bone marrow blood vessels, lined by endothelial cells (green), escaping into the surrounding tissue. The red boxes highlight the areas compared in the analysis and the white lines mark the bone.

After determining in detail how severe infection affects the bone marrow environment and HSC function, the team tested a way to prevent the damage. Before infecting the mice, they treated them with a hormone that regulates bone calcium and an antioxidant to counter cellular oxidative stress, and then again after infection.

This process led to a tenfold increase in HSC function following infection compared to mice that received no treatment (around 20-40 per cent function compared to two percent function, respectively). Although this is not a complete recovery, the vast increase in function is a positive sign.

The team note that the requirement to start the hormone treatment before infection, combined with its expense and need to be refrigerated, make it unviable as a solution, especially in many parts of the world where severe infections like malaria and TB are prevalent.

However, they hope that proof that the impact of severe infection on HSC function can be significantly lessened will lead to the development of new treatments that can be widely administered.

Professor Lo Celso said: The long-term impacts of COVID-19 infection are just starting to be known. The impact on HSC function appears similar across multiple severe infections, suggesting our work on malaria could shed light on the possible long-term consequences of COVID-19, and how we might mitigate them.

Dr Haltalli concluded: Protecting HSC function while still developing strong immune responses is key for healthy ageing.

-

Manipulating niche composition limits damage to haematopoietic stem cells during Plasmodium infection by Myriam L.R. Haltalli et al. is published in Nature Cell Biology.

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Severe infections wreak havoc on mouse blood cell production | Imperial News - Imperial College London

MELVILLE, N.Y., Nov. 20, 2020 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, announced today that its amended joint plan of reorganization has become effective and it has emerged from Chapter 11 reorganization. Pursuant to the confirmed plan of reorganization, the Company has received $3,848,000 in financing. The confirmed plan of reorganization also provides for additional funding, subject to certain conditions, of $3,500,000 less the sum of the debtor-in-possession financing provided to the Company during the reorganization (approximately $1,227,000) and the costs incurred by the debtor-in-possession lender.

In connection with the reorganization, Lance Alstodt has been appointed the Companys President, Chief Executive Officer and Chairman of the Board. Mr. Alstodt said, This process has been a long and challenging journey for the Company. Im inspired by the great resolve and execution from our employees, professionals and investors. We are very pleased that all requirements have been met for us to emerge. Allowed creditor claims have been fully satisfied and, as importantly, our equity holders have retained their shares in this exciting new opportunity. We were able to preserve all of our intellectual property assets and look forward to initiating our Phase 2 clinical trial.

Based upon the Companys emergence from Chapter 11 reorganization, FINRA has removed the Q at the end of its trading symbol. Shareholders do not need to exchange their shares for new shares.

About BioRestorative Therapies, Inc.

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

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

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

Forward-Looking Statements

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

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Emerges from Chapter 11 Reorganization Other OTC:BRTX - GlobeNewswire

Databridgemarketresearch.com Present Global Stem Cell Banking Market Industry Trends and Forecast to 2027 new report to its research database. The report spread No of pages: 350 No of Figures: 60 No of Tables: 220 in it. This Global Stem Cell Banking Market report takes into consideration diverse segments of the market analysis that todays business ask for. The Global Stem Cell Banking Market report provides estimations of CAGR values, market drivers and market restraints about the industry which are helpful for the businesses in deciding upon numerous strategies. The base year for calculation in the report is taken as 2017 whereas the historic year is 2016 which will tell you how the Global Stem Cell Banking Market is going to perform in the forecast years by informing you what the market definition, classifications, applications, and engagements are. The report helps you to be there on the right track by making you focus on the data and realities of the industry.

The research studies of this Global Stem Cell Banking Market report helps to evaluate several important parameters that can be mentioned as investment in a rising market, success of a new product, and expansion of market share. Market estimations along with the statistical nuances included in this market report give an insightful view of the market. The market analysis serves present as well as future aspects of the market primarily depending upon factors on which the companies contribute in the market growth, crucial trends and segmentation analysis. This Global Stem Cell Banking Market research report also gives widespread study about different market segments and regions.

Global stem cell banking market is set to witness a substantial CAGR of 11.03% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. The increased market growth can be identified by the increasing procedures of hematopoietic stem cell transplantation (HSCT), emerging technologies for stem cell processing, storage and preservation. Increasing birth rates, awareness of stem cell therapies and higher treatment done viva stem cell technology.

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Competitive Analysis:

Global stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of inflammatory disease drug delivery market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in global inflammatory disease drug delivery market are: NSPERITE N.V, Caladrius, ViaCord, CBR Systems, Inc, SMART CELLS PLUS, LifeCell International, Global Cord Blood Corporation, Cryo-Cell International, Inc., StemCyte India Therapeutics Pvt. Ltd, Cordvida, ViaCord, Cryoviva India, Vita34 AG, CryoHoldco, PromoCell GmbH, Celgene Corporation, BIOTIME, Inc., BrainStorm Cell Therapeutics and others

Market Definition:Global Stem Cell Banking Market

Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Cord Stem Cell Banking MarketDevelopment and Acquisitions in 2019

In September 2019, a notable acquisition was witnessed between CBR and Natera. This merger will develop the new chances of growth in the cord stem blood banking by empowering the Nateras Evercord branch for storing and preserving cord blood. The advancement will focus upon research and development of the therapeutic outcomes, biogenetics experiment, and their commercialization among the global pharma and health sector.

Cord Stem Cell Banking MarketScope

Cord Stem Cell Banking Marketis segmented on the basis of countries into U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

All country based analysis of the cord stem cell banking marketis further analyzed based on maximum granularity into further segmentation. On the basis of storage type, the market is segmented into private banking, public banking. On the basis of product type, the market is bifurcated into cord blood, cord blood & cord tissue. On the basis of services type, the market is segmented into collection & transportation, processing, analysis, storage. On the basis of source, market is bifurcated into umbilical cord blood, bone marrow, peripheral blood stem, menstrual blood. On the basis of indication, the market is fragmented into cerebral palsy, thalassemia, leukemia, diabetes, autism.

Cord stem cell trading is nothing but the banking of the vinculum plasma cell enclosed in the placenta and umbilical muscle of an infant. This ligament plasma comprises the stem blocks which can be employed in the forthcoming time to tackle illnesses such as autoimmune diseases, leukemia, inherited metabolic disorders, and thalassemia and many others.

Market Drivers

Increasing rate of diseases such as cancers, skin diseases and othersPublic awareness associated to the therapeutic prospective of stem cellsGrowing number of hematopoietic stem cell transplantations (HSCTs)Increasing birth rate worldwide

Market Restraint

High operating cost for the therapy is one reason which hinders the marketIntense competition among the stem cell companiesSometimes the changes are made from government such as legal regulations

Key Pointers Covered in the Cord Stem CellBanking MarketIndustry Trends and Forecast to 2026

Market SizeMarket New Sales VolumesMarket Replacement Sales VolumesMarket Installed BaseMarket By BrandsMarket Procedure VolumesMarket Product Price AnalysisMarket Healthcare OutcomesMarket Cost of Care AnalysisMarket Regulatory Framework and ChangesMarket Prices and Reimbursement AnalysisMarket Shares in Different RegionsRecent Developments for Market CompetitorsMarket Upcoming ApplicationsMarket Innovators Study

Key Developments in the Market:

In August, 2019, Bayer bought BlueRock for USD 600 million to become the leader in stem cell therapies. Bayer is paying USD 600 million for getting full control of cell therapy developer BlueRock Therapeutics, promising new medical area to revive its drug development pipeline and evolving engineered cell therapies in the fields of immunology, cardiology and neurology, using a registered induced pluripotent stem cell (iPSC) platform.In August 2018, LifeCell acquired Fetomed Laboratories, a provider of clinical diagnostics services. The acquisition is for enhancement in mother & baby diagnostic services that strongly complements stem cell banking business. This acquisition was funded by the internal accruals which is aimed to be the Indias largest mother & baby preventive healthcare organization.

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Research objectives

To perceive the most influencing pivoting and hindering forces in Cord Stem Cell Banking Market and its footprint in the international market.Learn about the market policies that are being endorsed by ruling respective organizations.To gain a perceptive survey of the market and have an extensive interpretation of the Cord Stem Cell Banking Market and its materialistic landscape.To understand the structure of Cord Stem Cell Banking Market by identifying its various sub segments.Focuses on the key global Cord Stem Cell Banking Market players, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).To project the consumption of Cord Stem Cell Banking Market submarkets, with respect to key regions (along with their respective key countries).To strategically profile the key players and comprehensively analyze their growth strategiesTo analyze the Cord Stem Cell Banking Market with respect to individual growth trends, future prospects, and their contribution to the total market.

Customization of the Report:

All segmentation provided above in this report is represented at country levelAll products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

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Global Stem Cell Banking Market to Get Expansion admist COVID 19, Scope With Advanced Technologies Top Key Players and Forecast 2020-2027 - The...

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This past February, Easton Lindeman then age 2, son ofCorey and Samantha was diagnosed with high-risk neuroblastoma, a type of cancer that affects his adrenal glands, spinal column, and bone marrow.Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body. Treatment for Easton started right away. This was followed by hospitalizations for both treatments and illnesses. So far he has gone through 3 rounds of chemotherapy and IMIBG therapy at the University of Wisconsin. IMIBG Therapy is a form of radiation treatment. This was followed by more chemotherapy and surgery to try to remove as much of the tumor as possible.

The last time we talked with Easton's mom he was finishing up with his 6th round of high dose chemotherapy and stem cell transplant.

He has had countless blood and platelet transfusions, ECHO's, hearing tests, MRI's, CT scans, X-Rays, bone marrow biopsies, surgeries and sedations. PICC line placements and Port Access placement. Earlier this month, Easton took a trip to the Mayo Clinic for more treatment.

On Saturday, Easton had a very special treat. The pink firetruck, overseen by Dennis and Diana Brammow made a stop to see Easton.

Easton is 3 years old now and his mom said that he loves to pass out candy to the doctors in and nurse friends while he's in the hospital. Like most 3 year old, there isn't a lot that slows him down. At 3 he's a family man already. Tagging around after his big brother Hunter and cousin Eleanor and he's always thrilled to visit his Grandpa and Grandma Dough and Dawn Yenser.

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Easton Lindeman gets a visit from the Pink Fire Truck - News - vintontoday.com

NEW YORK, Nov. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today highlighted its intellectual property portfolio for apamistamab, a CD45 targeting antibody, and the Antibody Radiation Conjugate (ARC) comprised of apamistamab and the radioisotope iodine-131 used in the Company's lead Phase 3 candidate, Iomab-B, and its Iomab-ACT programs. Actinium owns issued or pending patents within the United States and globally covering composition of matter, formulation, methods of use, and methods of administration with potential coverage for 19 years or longer. Importantly, Actinium owns an issued patent in the US covering composition of matter, for which the Company expects validity until 2037.In addition, the Company owns a second issued US patent that further covers composition of matter, methods of use, and methods of administration for Iomab-B. The company has also received a notice of allowance in Europe for this second patent and expects it to be in force until 2036.

Iomab-B is currently being investigated in the ongoing pivotal Phase 3 SIERRA trial, which is over 75% enrolled, for targeted conditioning prior to potentially curative bone marrow transplant (BMT) for patients with relapsed or refractory Acute Myeloid Leukemia ("R/R AML"). In addition, Actinium is utilizing apamistamab with lower doses of iodine-131, known as Iomab-ACT, for targeted conditioning prior to gene therapy and adoptive cell therapy ("ACT"), namely CAR-T, including in its recently announced collaboration with Memorial Sloan Kettering Cancer Center that is supported by NIH STTR Fast Track grant funding.

"The continued protection of our lead asset Iomab-B, our Iomab-ACT program and apamistamab by a strong patent position is an important component of our development efforts, particularly as we approach the conclusion of our pivotal Phase 3 SIERRA trial for BMT conditioning in R/R AML The growth of BMT, ACT and Gene Therapy has highlighted the importance of conditioning and the need to move beyond non-targeted chemotherapy to increase the number of patients that could benefit from these potentially curative therapies.CD45 is an ideal target for conditioning applications given its unique expression on blood cancer cells and blood forming stem and immune cells and with no expression outside the hematopoietic or blood system," said Dr. Dale Ludwig, Actinium's Chief Scientific Officer. "Apamistamab is well characterized and its use in conditioning is supported by extensive clinical data across multiple clinical trials and indications. Our robust data shows that apamistamab has a favorable biodistribution profile that, together with our ARC technology, has significant advantages over other approaches such antibody drug conjugates that require payload internalization, making them impractical for targeting CD45.Further, our ARC approach allows us to use varying intensities of targeted radiation to achieve our desired conditioning outcome.With these important patents in place, and continued expansion of our patent portfolio in the US, EU and other select countries, we look forward to continuing to build out our targeted conditioning strategic business unit."

About Iomab-B

Iomab-B is Actinium's lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, apamistamab (formerly BC8), labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient's cancer and marrow cells. In a Phase 1/2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced enabled 100% of patients to proceed to transplant with all patients achieving transplant engraftment by day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, Hodgkin's disease, Non-Hodgkin lymphomas and multiple myeloma. Actinium obtained the worldwide, exclusive rights to apamistamab (BC8) and Iomab-B from the Fred Hutchinson Cancer Research Center. Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation.Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities.With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently.Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning.The SIERRA trial is over seventy-five percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings.More information on this Phase 3 clinical trial can be found at sierratrial.com. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1 study with a CD19 CAR T-cell Therapy and Phase 1/2 anti-HIV stem cell gene therapy with UC Davis. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform.This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes.Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Clayton Robertson Actinium Pharmaceuticals, Inc. [emailprotected]

Hans VitzthumLifeSci Advisors, LLC[emailprotected](617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com

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Actinium Highlights Foundational Patents Covering the Composition of Apamistamab Antibody and Iomab-B Antibody Radiation Conjugate for Targeted...

The arrival every October of a survey from the long-term follow-up care unit (LTFU) at the Fred Hutchinson Cancer Care Alliance in Seattle sets in motion a routine. I walk it back from the mailbox, putting it on the kitchen table where it sits unopened for four or five days. Then its moved to the dining room table where it sits for another few days. Then its taken downstairs, still unopened, where its put in a stack of other stuff for a week-plus. The routine continues when a follow-up to the follow-up comes in the mail.

Paraphrasing, the second LTFU letter reads: cmon, man. Take 15 minutes, fill it out, mail it back. Which I do. Could this be avoidance behavior, as per psychologists? Quoting Bill Murray, the casual philosopher, (Stripes, 1981), thats a fact, Jack!

Ive been in remission for 17 years after a stem cell transplant from the Hutch a day after my 51st birthday. The LTFU began following patients in 1990, citing improvement in the mortality of transplantation.

There are about 6,800 people enrolled currently in LTFU. In 2019, 4,764 former patients received the survey and about half of them returned it, said Molly McElroy, the communications director for the Hutch. She added how theres one transplant patient ambulatory and upright from 1971; 100-plus from before 1980.

I was diagnosed in 1998 with a friendly leukemia, but during an annual checkup a few years later, the diagnosis changed, and: the prognosis isnt as promising.

Conversations like that are ineradicable, along with related reminiscences of hallway walks with chemo poles and technicians who warn, youre going to feel a little prick. This is before they draw blood or remove bone marrow. (The first time I heard it, I thought it funny; after the umpteenth, not so much.) Treatment of mantle cell lymphoma required difficult, four-cocktail, hospitalized chemotherapy at St. Marys in Grand Junction. Soon thereafter, we left for the Hutch and transplant.

Hokey and contrived as it sounds, remission from cancer is indeed about some of the people you meet along the way.

Lee Bradley fruit farmer, wine maker, country marketer, located in the northern suburbs of Paonia calmed the hothouse of my mind after diagnosis. We talked about what was coming, sharing common stories about doctors, the Hutch, the determination of it all. Last month, Bradley celebrated 20 years of good health. (His familys market, which sits alongside the north fork of the Gunnison river, is a good place to grab an easy chair and enjoy a summers day under the cottonwoods.)

Early on at the Hutch, a physician who looked like she had just left a Grateful Dead concert with her long hair, granny-glasses and leather-fringed jacket, calmly explained options. Including a clinical trial for which she had written the protocol. As a result, Im still getting mail from the LTFU. Dr. Leona Holmberg, MD, PhD., continues to get it done as a physician, researcher and oncology professor at the U. of Washington.

We also met a nattily dressed doctor with great humor. He was placing into my chest a dual Hickman catheter which would expeditiously transport stem cells, chemo, nutrition, blood products and whatever else. (A terrific invention, but not a good look on a beach. It flops around a lot.) He flirted good-naturedly with Susan, who was maybe 10 inches taller. Are you the same guy whose name is on this? I cracked wise about him getting a commission; smiling, he replied that hed done all right. Dr. Robert O. Hickman, who was a founding member of the Hutch bone marrow transplant team, died last year at 92, his invention still widely used.

After transplant, my blood counts were stubbornly stuck at zero. The body language of the doctor-posse when they rolled through my room in the mornings wasnt encouraging. Then I caught an infection and spent 10 days in isolation. My nurse recognized despair. On a rainy Saturday afternoon, she sat on the bed and without saying a word, pulled me close. For less than a minute. She likely broke a slew of HR rules, but Corinne Vaniers humanity was the tonic. The next day, my counts started to move north. Ten years after the transplant, we had lunch in Seattle and continue to be in touch.

Julia Vega, the daughter of longtime friends and a newspaper publisher pal, was a year younger than our son. She had a pelvic cancer and enjoyed some initial remission. Her dad and I shared stories about the Hutch, the Pete Gross House (apartments) and Dr. Holmberg, also her physician.

Vega was a theater student at Arizona State U. when diagnosed and instead of dropping out and taking it easy, she forged through doing the chemo, going to classes, participating in productions, graduating. I enjoyed her company as a middle schooler in North Carolina, and again at a newspaper convention where she accompanied her father, fragile thin and wearing a wig. We have a photo together from that meeting. Whenever I hear one of her favorite songs, Bob Dylans Shooting Star, I recall her grace.

A story about Chris Mesaric was framed and placed along the walls of a Hutch examination clinic. My brother and I had flown to Seattle to nail down the second opinion and I happened on it while he was wrapping up a conversation with yet another oncologist. Mesaric had grown up here, MHS Class of 1987, and had worked as an airframe mechanic for Boeing in Seattle before returning to Montrose. The docs had given him little chance in 1992 of surviving aplastic anemia, a rare bone marrow disease. He was also told that there would be no children, either. Yet he and Robin are the parents of two lovely daughters.

Mesarics brave fight went on for 11 years, receiving a Hutch transplant in 2001. He was inspirational, although we never met. We left the Hutch after five months, arriving home the day before Thanksgiving, 2003; Mesaric, 35, passed three weeks later. I know his folks, Frank and Linda Mesaric, and whenever I see Frank around town, his big, welcoming voice is at once a comfort and a knowing.

There were good times, of course, during the procedures, before the transplant. Routines that got us away from the grimness. After morning bloodwork, we had a daily 16-block hike to the Barnes & Noble bookstore in downtown Seattle. One day, former Sen. Gary Hart was spotted in the shelves, browsing, and we chatted. There were plenty of windows to look into, galleries of all sorts, and all the bits and bobs and fish tossing from the Pike Place Market. Even a free monorail to ride. We enjoyed a couple of nights out with live music at Dimitrious Jazz Alley. William visited from Alaska and the three of us caught a Mariners/White Sox game at Safeco Field. Ryanns down-the-elevator, in-the-building interim school provided friendships and field trips. There was retail therapy. The downtown flagship Nordstroms store provided almost 400,000 square feet of distraction and attraction. (After we returned to Montrose, they had to lay off the lobbys pianist. Jes kidding.)

Theres a picture of the four of us on the refrigerator from Day One. Bald and bloated, Im unrecognizable. After Susan declared we can do this, upon turning that apartments front door key, thats when the recovery, like the new bone marrow, began to graft itself.

The piece of mail in October confirms it. Im thankful for those follow-up people who still have my address.

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COLUMN: Surveys bring Thanksgiving | Opinion | montrosepress.com - Montrose Daily Press

Community Cord Blood Banking, a stem cell banking initiative introduced by LifeCell in 2017, has helped save the life of a seven-year-old girl from Nashik in Maharashtra who was suffering from aplastic anaemia, a rare and serious blood disorder.

In a major breakthrough, a team of senior doctors from LOTUS Institute of Haematology, Oncology and Bone Marrow Transplantation, recently conducted Indias first dual cord blood transplant through an unrelated donor using Community Stem Cell Banking. People familiar with the development said the transplant was challenging because no apt bone marrow donors were available and the cost of retrieval of matching units from public cord blood banks would have been high.

The girls parents, as members of LifeCell Community Bank, placed a request for two matching cord blood units after the childs sibling was found to be only a 50% (4/8) match. Two high-quality matches (7/8) were found in the registry, which fulfilled the requirement for umbilical cord blood transplantation. The parents could withdraw the matched units at no extra expense, which would have cost around 45 lakh per unit.

Mayur Abhaya, MD, LifeCell International said, The purpose of Community Cord Blood Banking is to ensure easy and rapid access to stem cells for every Indian without the hurdles of public and private banking models. While stem cells from the umbilical cord blood can be procured from global public banks, the probability of finding a match for a patient of Indian origin is less than 10% because of the low inventory of available units plus the big issue of donor dropouts.

Mr. Abhaya said, Luckily, since the family was a part of LifeCells community banking programme, they could gain quick, free access to the huge inventory of over 50,000 qualified and consented units available at LifeCell, which provides greater than 97% probability of finding a match.

In majority of blood-related disorders treatable by transplants, patients own stem cells are not suitable. Hence, the best donor is a close family member, usually a sibling. However, in this case, there was only a 50% match with the sibling, thus needing a match from an unrelated donor, LifeCell said in a statement. It said while a regular stem cell transplant requires a dose of 25 million cells per kg of the patients weight, for aplastic anaemia, the recommended minimum dose is 40 million cells per kg, which is not easy to find.

The community banking model made it possible and the child received a timely transplant with an encouraging prognosis. Just 18 days after the transplant, white blood cells were completely engrafted, and platelets and red blood cell production also increased drastically, the statement added.

Dr. Pritesh Junagade, director, LOTUS Hospital, expressed surprise that the retrieval process for two cord blood units was smooth and at no additional cost to the patient as compared to other banking models.

Tasneem Bohari, the childs father, said: It was two years ago that my daughter was diagnosed with aplastic anaemia. At the time of diagnosis, the doctors had suggested she would need a stem cell transplant eventually and it would be the best possible treatment in the future.

The family did research and made enquiries about which stem cell bank to opt for as they were expecting their second child. It was during this time that their friends and relatives suggested LifeCell to them. Their doctor also suggested that they could go ahead and preserve their babys stem cell with LifeCell.

At the time of preservation, we didnt have much idea about Community Stem Cell Banking and its benefits, but today we are happy to associate with LifeCell, who have helped my daughter lead a quality life through cord blood transplant, Mr. Bohari said.

Community Cord Blood Banking allows sharing of preserved umbilical cord stem cells from a common pool amongst the members of the community. The model offers greater and easier access to donor stem cells, unlimited retrievals at the cost of one enrolment, and a higher probability for finding a close match for potentially life-saving treatments.

This facility ensures complete protection to the child, siblings, parents, and maternal and paternal grandparents from more than 80 disorders treatable by stem cells.

See more here:
Community Cord Blood Banking saves life of child with aplastic anaemia - The Hindu

Stem cells found in the lungs can be redirected to the heart using a new inhaled antibody therapy to help boost healing following a heart attack.

Image credit: Jesse Orrico on Unsplash

Cardiovascular diseases are one of the leading causes of death globally. Medical advancements have lowered a persons risk of dying from an episode, but cell death and scar tissue that form afterward drastically increase the risk of eventual heart failure.

Stem cell therapy is a promising strategy used to help minimize long-term damage by regenerating new, healthy heart cells at damaged sites. However, their clinical application is hampered by poor cell engraftment, which comes with trying to grow new cells on a constantly moving organ.

Solutions to this have been proposed in the past, such as using durable patches to help the cells adhere to the heart surface, but a team of researchers from North Carolina State University propose that redirecting easy-to-access stem cells already found in the body could help spur repair in an injured heart.

Stem cells have become [] important for cardiomyocyte regeneration over the last two decades, wrote the team in their paper published in Advanced Science. In fact, as [part of] the bodys own repair mechanism, endogenous stem cells from the bone marrow are recruited into the heart. The issue, they point out, is that these stem cells lack the ability to properly target the heart, which hinders their ability to accumulate where they are needed.

In previous studies, the team demonstrated that heart attack biomarkers can be used to guide infused or endogenous stem cells to the site of cardiac injury. They also took advantage of cells called platelets, which help form blood clots and have an exceptional ability to target and accumulate in injured tissue.

By taking advantage of the injuryhoming ability of platelets, we can target stem cells to the [damaged] area, said the authors. [However], this strategy has a caveat: most of the bodys endogenous stem cells are produced in the bone marrow, and these cells are difficult to access.

The lung has recently been recognized as a site where both stem cells and navigator cells, such as platelets, are found in high concentrations. The team therefore sought to develop a strategy to link those two types of cells to provide an in-house treatment for repairing damaged cardiac tissue following a heart attack.

In this way, platelets can piggyback on stem cells found in the lungs called hematopoietic stem cells (HSCs) and bring them to the damaged site for repair, they said. To do this, they designed what is called a bispecific antibody, which is an artificial protein that can simultaneously bind to two different types of molecules. They are commonly used in treating cancers and different autoimmune diseases, but in the current study, the researchers used them to bind HSCs as well as platelets, to help the stem cells accumulate where they are needed.

An interesting and advantageous aspect of this strategy is the fact that the bispecific antibodies can be delivered through inhalation since their desired location is the lungs. Unlike previous strategies, this provides a safe, non-invansive, and convenient way of administration.

In the study, the team tested whether the inhaled antibodies could effectively link HSCs with platelets and redirect them to the injured heart, and bench-marked this route of administration against an intravenous infusion. It was found that the antibody therapy effectively conjoined HSCs and platelets, and the redirected stem cells helped promote repair and minimized inflammation at the site of injury, which led to increased cardiac function compared to controls. In addition, inhalation of the platelet-antibody therapy was found to be more efficient and specific than the intravenous route.

While there are limitations that still need to be overcome before this can move into clinical trials, this work demonstrates an innovative and easy approach to help boost heart healing and limit the long-term damage of heart attacks. The team is hopeful that this work can be used as a jumping point to help bring this type of treatment closer to clinical application.

Reference: Mengrui Liu, et al. Bispecific Antibody Inhalation Therapy for Redirecting Stem Cells from the Lungs to Repair Heart Injury. Advanced Science (2020). DOI: 10.1002/advs.202002127

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Leveraging the lung's biogenesis to repair the heart - Advanced Science News

During a Targeted Oncology Case Based Peer Perspective event, Usama Gergis, MD, MBA, professor of Oncology, director, Bone Marrow Transplant and Immune Cellular Therapy at Sidney Kimmel Cancer Center, at the Thomas Jefferson University Hospital, discussed the case of a 48-year-old male patient with acute graft-versus-host-disease (GVHD).

Targeted Oncology: For a patient such as this, what is the risk of developing acute (GVHD) following transplant?

GERGIS: The list of risk factors for GVHD is huge: donor HLA [human leukocyte antigen] disparities that are major/minor, sex matching, donor parity, donor age, blood typewhile its controversialdonor CMV seropositivity, gene polymorphism, and stemcell graft source. Obviously, peripheral mobilized stem cells have more T cells than bone marrow. Other factors include graft composition, the higher CD34 the higher lymphocytes, and conditioning intensities. There are a bunch of factors here, at least for our patient, who had a MAC. The fact that his donor is a multirisk, 50-year-old woman and a MUD [infers] similar higher risk.

How quickly do you determine whether a patient is steroid refractory or steroid dependent?

Basically, steroid refractoriness or resistance versus dependence versus intolerance [can be determined in] as early as 3 days. If there is progression of the grade of GVHD, this is considered steroid refractory. If by 4 weeks it does not go to grade 0, its considered refractory. However, I disagree with the 28-day [timeline]. I think its a long time to wait. Usually, I look at 2 weeks.

This has been the consensus among many of us. I think the trials REACH-1 [NCT02953678] and REACH-2 [NCT02435433] looked at 28 days; but if you go to the 2 papers, most responses took place in 7 to 14 days. Again, this is in the second-line setting.

Ive been doing this for 15 years. Ive been through most of this; so many lines [of therapy and] so many heartbreaks. I teach my fellows to look at any [research] paper in second-line GVHDthe overall response rate is 30%. Ive done it with MMF [mycophenolate mofetil], sirolimus [Rapamune], infliximab [Remicade], ATG [antithymocyte globulin], cyclophosphamide, Ontak [denileukin diftitox], and mesenchymal stem cells and overall response rate is 30%.

Which data support the use of ruxolitinib (Jakafi) in patients who are steroid refractory?

Based on a small trial of 49 patients, a phase 2 trial [INCB 18424-271; NCT02953678], ruxolitinib was approved as a second-line therapy for patients with acute steroid-refractory GVHD.1

Review the details of the REACH-2 trial.

The REACH-2 trial data were just published in 2020. Ruxolitinib was approved based on a small trial of 49 patients with steroid-refractory acute GVHD. They enrolled 70 but only 49 were available for efficacy. Obviously, it was a pilot phase 2 with no comparative arm, and the response rates across the board were [somewhere in the range of] 50% or so by day 28.

Then they were asked to run a phase 3 trial comparing ruxolitinib at 10 mg twice daily versus best available care. The best available care goes through the list that I just mentioned, including photopheresis, ATG, and others. After 4 weeks, patients who were not responders on the best available care were allowed to cross over to ruxolitinib.

By day 28, the responders on the ruxolitinib composed 60% of the cohort versus 40% on the control arm with a P value of less than .001. The durability of response at 8 weeks was 40% versus 20% [odds ratio, 2.38; 95% CI, 1.43 to 3.94; P < .001].

This tell us that ruxolitinib works in this group, that it does not work that great in one-third of patients who lose their response at 8 weeks, and that its just better than anything else [thats available]. A good thing about the ruxolitinib arm is that the response duration was much longer than best available treatment, as illustrated by the Kaplan-Meier curve for response duration.

Failure-free survival [in this trial] was 5.0 months versus 1.0 month [HR, 0.46; 95% CI, 0.35-0.60].

Do you apply this therapy to any of your patients?

We have a patient [at my institution] who has steroid-refractory GVHD. Last Friday in our weekly meeting, we were discussing his [case]. He has lower GI [gastrointestinal] GVHDgrade IV, steroid-refractory, as bad as it getsand I said lets start him on ruxolitinib. One of my colleagues said it doesnt work well in the lower GI. I asked the organizers to pull [data] from the supplemental figures on the New England Journal of Medicine paper of REACH-2 [indicating that these patients did have a benefit with ruxolitinib].2

REFERENCES:

1. FDA approves ruxolitinib for acute graft-versus-host disease. FDA. May 24, 2019. Accessed October 10, 2020. https://bit.ly/2SMU7I8

2. Zeiser R, von Bubnoff N, Butler J, et al; REACH2 Trial Group. Ruxolitinib for glucocorticoid- refractory acute graft-versus-host disease. N Engl J Med. 2020;382(19):1800-1810. doi: 10.1056/NEJMoa1917635

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Ruxolitinib for Acute Graft-vs-Host Disease Improves Outcomes in the Second Line - Targeted Oncology

Egg cells harvested from Mauritian cynomolgus macaques (top left) were fertilized (top right) and injected with CRISPR gene editing materials to insert a genetic mutation that cured two men of HIV in the last decade. The growing embryos (developing in the bottom images), if carried to maturity by surrogates, will help researchers study the mutation as a potential treatment for HIV. Courtesy of Golos and Slukvin labs

A gene that cured a man of HIV a decade ago has been successfully added to developing monkey embryos in an effort to study more potential treatments for the disease.

Timothy Brown, known for years as the Berlin Patient, received a transplant of bone marrow stem cells in 2007 to treat leukemia. The cells came from a donor with a rare genetic mutation that left the surfaces of their white blood cells without a protein called CCR5. When Browns immune system was wiped out and replaced by the donated cells, his new immune systems cells carried the altered gene.

This mutation cuts a chunk out of the genome so that it loses a functional gene, CCR5, that is a co-receptor for HIV, says Ted Golos, a University of WisconsinMadison reproductive scientist and professor of comparative biosciences and obstetrics and gynecology. Without CCR5, the virus cant attach to and enter cells to make more HIV. So, in Timothy Browns case, his infection was eliminated.

In 2019, a second cancer patient Adam Castillejo, initially identified as the London patient was cleared of his HIV by a stem cell transplant conferring the same mutation.

Thats very exciting, and there have been some follow up studies. But its been complicated, to say the least, Golos says.

Between the two transplants came a more infamous application of the mutation, when in 2018 Chinese biophysicist He Jiankui announced he had used the DNA-editing tool CRISPR to write the mutation into the DNA of a pair of human embryos. His work drew criticism from scientists concerned with the ethics of altering genes that can be passed down to human offspring, and he was jailed by the Chinese government for fraud.

The promise of the CCR5 mutation remains, but not without further study. The mutation occurs naturally in fewer than 1 percent of people, suggesting that it may not be associated only with positive health outcomes. An animal model for research can help answer open questions.

Given interest in moving forward gene-editing technologies for correcting genetic diseases, preclinical studies of embryo editing in nonhuman primates are very critical, says stem cell researcher Igor Slukvin, a UWMadison professor of pathology and laboratory medicine.

Golos, Slukvin and colleagues at UWMadisons Wisconsin National Primate Research Center and schools of Veterinary Medicine and Medicine and Public Health employed CRISPR to edit the DNA in newly fertilized embryos of cynomolgus macaque monkeys. They published their work recently in the journal Scientific Reports.

Slukvins lab had already established a method for slicing the CCR5-producing gene out of the DNA in human pluripotent stem cells, which can be used to generate immune cells resistant to HIV.

We used that same targeting construct that we already knew worked in cells, and delivered it to one-cell fertilized embryos, says Jenna Kropp Schmidt, a Wisconsin National Primate Research Center scientist. The thought is that if you make the genetic edit in the early embryo that it should propagate through all the cells as the embryo grows.

Primate Center scientist Nick Strelchenko found that as much as one-third of the time the gene edits successfully deleted the sections of DNA in CRISPRs crosshairs base pairs in both of the two copies of the CCR5 gene on a chromosome and were carried on into new cells as the embryos grew.

The goal now is to transfer these embryos into surrogates to produce live offspring who carry the mutation, Schmidt says.

Cynomolgus macaques are native to Southeast Asia, but a group of the monkeys has lived in isolation on the Indian Ocean island of Mauritius for about 500 years. Because the entire Mauritian monkey line descends from a small handful of founders, they have just seven variations of the major histocompatibility complex, the group of genes that must be matched between donor and recipient for a successful bone marrow transplant. There are hundreds of MHC variations in humans.

With MHC-matched monkeys carrying the CCR5 mutation, the researchers would have a reliable way to study how successful the transplants are against the simian immunodeficiency virus, which works in monkeys just like HIV does in humans.

Anti-retroviral drugs have really positively changed the expectation for HIV infection, but in some patients, they may not be as effective. And theyre certainly not without long-term consequences, says Golos, whose work is funded by the National Institutes of Health. So, this is potentially an alternative approach, which also allows us to expand our understanding of the immune system and how it might protect people from HIV infection.

The animal model could lead to the development of gene-edited human hematopoietic stem cells the type that work in bone marrow to produce many kinds of blood cells that Slukvin and Golos say could be used as an off-the-shelf treatment for HIV infection.

This research was supported by grants from the National Institutes of Health (R24OD021322, P51OD011106, K99 HD099154-01, RR15459-01 and RR020141-01).

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Gene-edited monkey embryos give researchers new way to study HIV cure - University of Wisconsin-Madison

The global Hematopoietic Stem Cell Transplantation (HSCT) market is broadly analyzed in this report that sheds light on critical aspects such as the vendor landscape, competitive strategies, market dynamics, and regional analysis. The report helps readers to clearly understand the current and future status of the global Hematopoietic Stem Cell Transplantation (HSCT) market. The research study comes out as a compilation of useful guidelines for players to secure a position of strength in the global Hematopoietic Stem Cell Transplantation (HSCT) market. The authors of the report profile leading companies of the global Hematopoietic Stem Cell Transplantation (HSCT) market, such as Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc They provide details about important activities of leading players in the competitive landscape.

The report predicts the size of the global Hematopoietic Stem Cell Transplantation (HSCT) market in terms of value and volume for the forecast period 2019-2026. As per the analysis provided in the report, the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to rise at a CAGR of XX % between 2019 and 2026 to reach a valuation of US$ XX million/billion by the end of 2026. In 2018, the global Hematopoietic Stem Cell Transplantation (HSCT) market attained a valuation of US$_ million/billion. The market researchers deeply analyze the global Hematopoietic Stem Cell Transplantation (HSCT) industry landscape and the future prospects it is anticipated to create.

This publication includes key segmentations of the global Hematopoietic Stem Cell Transplantation (HSCT) market on the basis of product, application, and geography (country/region). Each segment included in the report is studied in relation to different factors such as consumption, market share, value, growth rate, and production.

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The comparative results provided in the report allow readers to understand the difference between players and how they are competing against each other. The research study gives a detailed view of current and future trends and opportunities of the global Hematopoietic Stem Cell Transplantation (HSCT) market. Market dynamics such as drivers and restraints are explained in the most detailed and easiest manner possible with the use of tables and graphs. Interested parties are expected to find important recommendations to improve their business in the global Hematopoietic Stem Cell Transplantation (HSCT) market.

Readers can understand the overall profitability margin and sales volume of various products studied in the report. The report also provides the forecasted as well as historical annual growth rate and market share of the products offered in the global Hematopoietic Stem Cell Transplantation (HSCT) market. The study on end-use application of products helps to understand the market growth of the products in terms of sales.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Product: , Allogeneic, Autologous

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

The report also focuses on the geographical analysis of the global Hematopoietic Stem Cell Transplantation (HSCT) market, where important regions and countries are studied in great detail.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Geography:

Methodology

Our analysts have created the report with the use of advanced primary and secondary research methodologies.

As part of primary research, they have conducted interviews with important industry leaders and focused on market understanding and competitive analysis by reviewing relevant documents, press releases, annual reports, and key products.

For secondary research, they have taken into account the statistical data from agencies, trade associations, and government websites, internet sources, technical writings, and recent trade information.

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Key questions answered in the report:

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Table Of Contents:

1 Market Overview of Hematopoietic Stem Cell Transplantation (HSCT)1.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview1.1.1 Hematopoietic Stem Cell Transplantation (HSCT) Product Scope1.1.2 Market Status and Outlook1.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Overview by Region 2015 VS 2020 VS 20261.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Region (2015-2026)1.4 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Region (2015-2020)1.5 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Forecast by Region (2021-2026)1.6 Key Regions, Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.2 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.3 Asia-Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.4 Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.5 Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026) 2 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview by Type2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type: 2015 VS 2020 VS 20262.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Type (2015-2020)2.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Type (2021-2026)2.4 Allogeneic2.5 Autologous 3 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview by Application3.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application: 2015 VS 2020 VS 20263.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Application (2015-2020)3.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Application (2021-2026)3.4 Peripheral Blood Stem Cells Transplant (PBSCT)3.5 Bone Marrow Transplant (BMT)3.6 Cord Blood Transplant (CBT) 4 Global Hematopoietic Stem Cell Transplantation (HSCT) Competition Analysis by Players4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Players (2015-2020)4.2 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Hematopoietic Stem Cell Transplantation (HSCT) as of 2019)4.3 Date of Key Manufacturers Enter into Hematopoietic Stem Cell Transplantation (HSCT) Market4.4 Global Top Players Hematopoietic Stem Cell Transplantation (HSCT) Headquarters and Area Served4.5 Key Players Hematopoietic Stem Cell Transplantation (HSCT) Product Solution and Service4.6 Competitive Status4.6.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Rate4.6.2 Mergers & Acquisitions, Expansion Plans 5 Company (Top Players) Profiles and Key Data5.1 Regen Biopharma Inc5.1.1 Regen Biopharma Inc Profile5.1.2 Regen Biopharma Inc Main Business5.1.3 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.1.4 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.1.5 Regen Biopharma Inc Recent Developments5.2 China Cord Blood Corp5.2.1 China Cord Blood Corp Profile5.2.2 China Cord Blood Corp Main Business5.2.3 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.2.4 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.2.5 China Cord Blood Corp Recent Developments5.3 CBR Systems Inc5.5.1 CBR Systems Inc Profile5.3.2 CBR Systems Inc Main Business5.3.3 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.3.4 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.3.5 Escape Therapeutics Inc Recent Developments5.4 Escape Therapeutics Inc5.4.1 Escape Therapeutics Inc Profile5.4.2 Escape Therapeutics Inc Main Business5.4.3 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.4.4 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.4.5 Escape Therapeutics Inc Recent Developments5.5 Cryo-Save AG5.5.1 Cryo-Save AG Profile5.5.2 Cryo-Save AG Main Business5.5.3 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.5.4 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.5.5 Cryo-Save AG Recent Developments5.6 Lonza Group Ltd5.6.1 Lonza Group Ltd Profile5.6.2 Lonza Group Ltd Main Business5.6.3 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.6.4 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.6.5 Lonza Group Ltd Recent Developments5.7 Pluristem Therapeutics Inc5.7.1 Pluristem Therapeutics Inc Profile5.7.2 Pluristem Therapeutics Inc Main Business5.7.3 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.7.4 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.7.5 Pluristem Therapeutics Inc Recent Developments5.8 ViaCord Inc5.8.1 ViaCord Inc Profile5.8.2 ViaCord Inc Main Business5.8.3 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.8.4 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.8.5 ViaCord Inc Recent Developments 6 North America6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country6.2 United States6.3 Canada 7 Europe7.1 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country7.2 Germany7.3 France7.4 U.K.7.5 Italy7.6 Russia7.7 Nordic7.8 Rest of Europe 8 Asia-Pacific8.1 Asia-Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Region8.2 China8.3 Japan8.4 South Korea8.5 Southeast Asia8.6 India8.7 Australia8.8 Rest of Asia-Pacific 9 Latin America9.1 Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country9.2 Mexico9.3 Brazil9.4 Rest of Latin America 10 Middle East & Africa10.1 Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country10.2 Turkey10.3 Saudi Arabia10.4 UAE10.5 Rest of Middle East & Africa 11 Hematopoietic Stem Cell Transplantation (HSCT) Market Dynamics11.1 Industry Trends11.2 Market Drivers11.3 Market Challenges11.4 Market Restraints 12 Research Finding /Conclusion 13 Methodology and Data Source 13.1 Methodology/Research Approach13.1.1 Research Programs/Design13.1.2 Market Size Estimation13.1.3 Market Breakdown and Data Triangulation13.2 Data Source13.2.1 Secondary Sources13.2.2 Primary Sources13.3 Disclaimer13.4 Author List

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Hematopoietic Stem Cell Transplantation (HSCT) Market by Product Type, End User and by Region-Trends and Forecast to 2026|China Cord Blood Corp, CBR...

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Boca Raton, FL, November 17 Every three minutes a person in the United States is diagnosed with a blood disease such as leukemia, immunodeficiencies and sickle cell. To deliver cutting-edge, lifesaving therapy services for stem cell transplantations and improve patient outcomes, the Gift of Life Marrow Registry has announced the newly opened the Gift of Life Center for Cell and Gene Therapy.

Located at Gift of Lifesheadquarters in Boca Raton, Fla., theCenter will provide transplant physicians, researchers, and cell and gene therapy developers with the donor cell products they need to help more patients than ever before. One of the Centers signature services will be a biobank of off-the-shelf, on-demand cell therapy products available for transplantation, as well as those for engineering and ethical research. The inventory will be comprised of products collected from super donors with high frequency genetic characteristics sourced from the Gift of Life Marrow Registry. These cells will be tested, processed and cryopreserved by the Centers cellular therapy laboratory.

Another specialty service is the Centers innovation program, which will collaborate with world-renowned South Florida research institutions to improve transplant outcomes, develop processes for cell expansion and much more.

While many advancements have been made in stem cell treatments for those with blood diseases, there are still critical barriers that can impeded success, said Gift of Life CEO and Founder Jay Feinberg.

The only registry in the world started and run by a transplant survivor, Gift of Life has pioneered key innovations in the stem cell transplantation and donation process. Last April, the organization become the first registry to operate an in-house stem cell collection center that has not only helped to bring stem cell donors to collection faster, but also greatly improves the experience for donors. Gift of life is also the first registry to use buccal cheek swabs at recruitment drives instead of blood draws and the first to do mobile registration, including the creation of a smart phone app to register individuals quickly and effectively.

Gift of Lifes overarching mission isto democratize celltherapy and ensure that every patient has an equal opportunity to receive the treatment that can save their lives, said Feinberg. Doing so requires innovation, passion and an entrepreneurial approach to the challenges at hand and we are excited to draw upon our 30 years of expertise as we embark on this next phase of our work.

About Gift of Life Marrow RegistryGift of Life Marrow Registry is a 501(c)(3) nonprofit organization headquartered in Boca Raton, Fla. The organization, established in 1991, is dedicated to saving lives by facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, and other blood-related diseases. To learn more about Gift of Life Marrow Registry, visitwww.giftoflife.org.

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gift of life marrow registry announces new center for cell and gene therapy - The Boca Raton Tribune

For the first time, researchers at the Center for Cell-Based Therapy (CTC) in Ribeiro Preto, Brazil, have identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency.

GATA2 deficiency is a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2), which regulates the expression of genes that play a role in developmental processes and cell renewal.

An article on the study is publishedin the journalBlood.

The non-hereditary mutation may have acted as a natural gene therapy which prevented the disease from damaging the process of blood cell renewal. This meant that the patient did not develop such typical clinical manifestations as bone marrow failure, hearing loss, and lymphedema.

The researchers say that the findings pave the way for the use of gene therapy and changes to the process of checking family medical history and medical records for families with the hereditary disorder.

Luiz Fernando Bazzo Catto, first author of the article, said: When a germline [inherited] mutation in GATA2 is detected, the patients family has to be investigated because there may be silent cases.

The discovery was made when two sons were receiving medical treatment at the blood centre of the hospital run by FMRP-USP, both of which, in post-mortem DNA sequencing, showed germline mutations and GATA2 deficiency diagnosis. The researchers used next generation sequencing to estimate the proportion of normal blood cells in the fathers bone marrow, preventing clinical manifestations of GATA2 deficiency, and of cells similar to his childrens showing that 93% of his leukocytes had the mutation that protects from the clinical manifestations of GATA2 deficiency.

Following the sequencing of the fathers T-lymphocytes, the researchers found that the mutation occurred early in their lives and in the development of hematopoietic stem cells, which have the potential to form blood.

They also measured the activity of the blood cells, to see if they could maintain the activity of inducing normal cell production for a long time, by measuring the telomeres of his peripheral blood leukocytes. Telomeres are repetitive sequences of non-coding DNA at the tip of chromosomes that protect them from damage. Each time cells divide, their telomeres become shorter. They eventually become so short that division is no longer possible, and the cells die or become senescent.

The telomeres analysed by the researchers were long, indicating that the cells can remain active for a long time.

The researchers hypothesised that the existence of the somatic mutation in the fathers blood cells, and its restoration of the blood cell renewal process, may have contributed to the non-manifestation of extra-haematological symptoms of GATA2 deficiency such as deafness, lymphedema, and thrombosis.

Professor Rodrigo Calado, a corresponding author of the article, said: A sort of natural gene therapy occurred in this patient. Its as if he embodied an experiment and a medium-term prospect of analogous gene therapy treatment in patients with GATA2 deficiency.

The findings help us understand better how stem cells can recover by repairing an initial genetic defect.

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Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency - Health Europa

A succinct analysis of market size, regional growth and revenue projections for the coming years is presented in GlobalStem Cells Marketreport. The study further sheds light on major issues and the new growth strategies implemented by manufacturers that are part ofcompetitive landscape of the studied market.Thereport offers key trends, investment opportunities and drivers in Global Stem Cells Marketwith the latest market intelligence by adopting primary and secondary research methods. It also includes strategies adopted in the context of acquisitions and mergers, and business footprint extensions.

In order to provide more exactmarket forecast, the report comprises a complete research study and analysis ofCOVID-19 impact on the Global Stem Cells market. It also considers the strategies that can be adopted to deal with the situation.

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Competitive Landscape and Stem Cells Market Share Analysis:

The competitive landscape of the Stem Cells market provides data about the players operating in the studied market. The report includes a detailed analysis and statistics onprice, revenue and market share of the playersfor the period 2020-2025. The major players covered are as follows:

Thermo Fisher Scientific Inc.

Cellular Engineering Technologies Inc

Qiagen N.V

Sigma Aldrich Corporation

Becton, Dickinson and Company

Miltenyi Biotec

International Stem Cell Corporation

Stem Cell Technologies Inc.

Pluristem Therapeutics Inc

Medtronic, Inc

Zimmer Holdings, Inc.

Bio Time Inc

Zimmer Holdings, Inc

Orthofix, Inc.

Osiris Therapeutics Inc

Others Prominent Players

Key segments covered:

By Product

Adult Stem Cells

Neural Stem Cells

Hematopoietic Stem Cells

Mesenchymal Stem Cells

Umbilical Cord Stem Cells

Epithelial Stem cells and Skin Stem Cells

Others

Human Embryonic Stem Cells

Induced Pluripotent Stem Cells

Others

By Application

Regenerative Medicine

Neurology Regenerative Medicine

Oncology Regenerative Medicine

Myocardial Infraction Regenerative Medicine

Diabetes Regenerative Medicine

Hematology & Immunology Regenerative Medicine

Orthopedics Regenerative Medicine

Other Regenerative Medicine

Drug Discovery and Development

Other Applications

By Technology

Cell Acquisition

Bone Marrow Harvest

Umbilical Blood Cord

Apheresis

Others

Cell Production

Therapeutic Cloning

In-vitro Fertilization

Cell Culture

Isolation

Cryopreservation

Expansion and Sub-Culture

By End-User

Biopharmaceutical

Biotechnology Industry

Research Institutes

By Treatment Type

Allogeneic Stem Cell Therapy

Auto logic Stem Cell Therapy

Syngeneic Stem Cell Therapy

By Banking Type

Public

Private

By Region:

North America (U.S. & Canada)

Europe (Germany, United Kingdom, France, Italy, Spain, Russia, and Rest of Europe)

Asia Pacific (China, India, Japan, South Korea, Indonesia, Taiwan, Australia, New Zealand, and Rest of Asia Pacific)

Latin America (Brazil, Mexico, and Rest of Latin America)

Middle East & Africa (GCC, North Africa, South Africa, and Rest of Middle East & Africa)

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Some Important Highlights from the Report include:

Market CAGR during the 2020-2025 forecast period.

Comprehensive analysis on factors that will speed up the growth of Stem Cells marketover the next five years.

Precise estimates about the market size of global Stem Cells market and itscontribution to the parent market.

Precise forecasts for future developments in the Stem Cells industry and shifts in consumer behavior.

The growth of the Stem Cells Market across the Americas, APAC, Europe and MEA.

A detailed analysis of the industry competition and quantitative data on various vendors.

Comprehensive information on variables that will impede the growth of Stem Cells companies.

The Following are the Key Features of Global Stem Cells Market Report:

Market Overview, Industry Development, Market Maturity, PESTLE Analysis, Value Chain Analysis

Growth Drivers and Barriers, Market Trends & Market Opportunities

Porters Five Forces Analysis & Trade Analysis

Market Forecast Analysis for 2020-2025

Market Segments by Geographies and Countries

Market Segment Trend and Forecast

Market Analysis and Recommendations

Price Analysis

Key Market Driving Factors

Stem Cells Market Company Analysis: Company Market Share & Market Positioning, Company Profiling, Recent Industry Developments etc.

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Stem Cells Market Detailed Analysis by On-going Trends, Prominent Size, Share, Sales and Forecast to 2025 - PRnews Leader

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BioRestorative Therapies Inc (OTCMKTS: BRTXQ) (BRTX) is soaring up the charts after it was revealed at 12.04 pm Wednesday afternoon the Company was emerging from bankruptcy. BRTXQ came to the attention of many penny stock speculators after the Company partnered on a new bankruptcy reorganization plan with one of its creditors Auctus Capital in which it would emerge from bankruptcy with the commons intact, ready to begin phase 2 trials and get BioRestorative back on a national stock exchange.

BioRestorative Therapies has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs. BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. It is the Companys intend that the product be used for the non-surgical treatment of painful lumbosacral disc disorders.

BioRestorative Therapies Inc (OTCMKTS: BRTX) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders: Disc/Spine Program (brtxDISC): Its lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders.

The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. The Company has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

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

BioRestorative owns a valuable intelectual property portfolio including unique international Stem Cell patents as well as 8 patents issued, in the United States and other countries, for the Companys brown fat technology related to BioRestoratives metabolic program (ThermoStem Program).

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BioRestorative Therapies is making a powerful move up the charts after it was it was revealed at 12.04 pm Wednesday afternoon the Company was emerging from bankruptcy. This comes after the Company successfully entered into a reorganization plan with one of its creditors Auctus Capital after its March Bankruptcy filing in which the Company would emerge from bankruptcy with the commons intact, ready to begin their phase 2 trials and get BioRestorative back on a national stock exchange. BioRestorative Therapies has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs. BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. It is the Companys intend that the product be used for the non-surgical treatment of painful lumbosacral disc disorders. We will be updating on BioRestorative when more details emerge so make sure you are subscribed to Microcapdaily so you know whats going on with BioRestorative.

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

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BRTX-100; the Story of BioRestorative Therapies Inc (OTCMKTS: BRTX) - MicroCap Daily

NEW YORK, NY / ACCESSWIRE / November 18, 2020 / Pomerantz LLP announces that a class action lawsuit has been filed against Mesoblast Limited ("Mesoblast" or the "Company") (NASDAQ:MESO) and certain of its officers. The class action, filed in United States District Court for the Southern District of New York, and docketed under 20-cv-09111, is on behalf of a class consisting of all persons other than Defendants who purchased or otherwise, acquired Mesoblast securities between April 16, 2019 and October 1, 2020, inclusive (the "Class Period"). Plaintiff pursues claims against the Defendants under the Securities Exchange Act of 1934 (the "Exchange Act").

If you are a shareholder who purchased Mesoblast securities during the class period, you have until December 7, 2020, to ask the Court to appoint you as Lead Plaintiff for the class. A copy of the Complaint can be obtained at http://www.pomerantzlaw.com. To discuss this action, contact Robert S. Willoughby at newaction@pomlaw.com or 888.476.6529 (or 888.4-POMLAW), toll-free, Ext. 7980. Those who inquire by e-mail are encouraged to include their mailing address, telephone number, and the number of shares purchased.

[Click here for information about joining the class action]

Mesoblast develops allogeneic cellular medicines using its proprietary mesenchymal lineage cell therapy platform. Its lead product candidate, RYONCIL (remestemcel-L), is an investigational therapy comprising mesenchymal stem cells derived from bone marrow. In February 2018, the Company announced that remestemcel-L met its primary endpoint in a Phase 3 trial to treat children with steroid refractory ("SR") acute graft versus host disease ("aGVHD").

In early 2020, Mesoblast completed its rolling submission of its Biologics License Application ("BLA") with the U.S. Food and Drug Administration ("FDA") to secure marketing authorization to commercialize remestemcel-L for children with steroid refractory aGVHD.

Story continues

The complaint alleges that throughout the Class Period, Defendants made materially false and misleading statements regarding the Company's business, operational, and compliance policies. Specifically, Defendants made false and/or misleading statements and/or failed to disclose that: (i) comparative analyses between Mesoblast's Phase 3 trial and three historical studies did not support the effectiveness of remestemcel-L for steroid refractory aGVHD because of design differences between the four studies; (ii) as a result, the FDA was reasonably likely to require further clinical studies; (iii) as a result, the commercialization of remestemcel-L in the U.S. was likely to be delayed; and (iv) as a result of the foregoing, Defendants' positive statements about the Company's business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

On August 11, 2020, the FDA released briefing materials for its Oncologic Drugs Advisory Committee ("ODAC") meeting to be held on August 13, 2020. Therein, the FDA stated that Mesoblast provided post hoc analyses of other studies "to further establish the appropriateness of 45% as the null Day-28 ORR" for its primary endpoint. The briefing materials stated that, because of design differences between these historical studies and Mesoblast's submitted study, "it is unclear that these study results are relevant to the proposed indication."

On this news, the Company's American Depositary Share ("ADS") price fell $6.09 per share, or approximately 35%, to close at $11.33 per share on August 11, 2020, on unusually heavy trading volume.

On October 1, 2020, Mesoblast disclosed that it had received a Complete Response Letter ("CRL") from the FDA regarding its marketing application for remestemcel-L for treatment of SR-aGVHD in pediatric patients. According to the CRL, the FDA recommended that the Company "conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD." The CRL also "identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the product's biologic activity."

On this news, the Company's ADS price fell $6.56 per share, or over 35%, to close at $12.03 per share on October 2, 2020, on unusually heavy trading volume.

The Pomerantz Firm, with offices in New York, Chicago, Los Angeles, and Paris is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, the Pomerantz Firm pioneered the field of securities class actions. Today, more than 80 years later, the Pomerantz Firm continues in the tradition he established, fighting for the rights of the victims of securities fraud, breaches of fiduciary duty, and corporate misconduct. The Firm has recovered numerous multimillion-dollar damages awards on behalf of class members. See http://www.pomerantzlaw.com.

SOURCE: Pomerantz LLP

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SHAREHOLDER ALERT: Pomerantz Law Firm Reminds Shareholders with Losses on their Investment in Mesoblast Limited of Class Action Law Suit and Upcoming...

TEL AVIV, Israel, Nov. 18, 2020 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical Company focused on oncology, today announced that the Company's lead drug candidate, the CXCR4-inhibitor Motixafortide, will be evaluated in an investigator-initiated clinical trial in patients suffering from acute respiratory distress syndrome (ARDS) secondary to COVID-19 and other respiratory viral infections.

The open-label, single-arm, Phase 1b study will be conducted at the Wolfson Medical Center in Holon, Israel, with Dr. Yasmin Maor, Head of the Infectious Disease Unit, as lead investigator.

"Severe COVID-19 cases, where patients are hospitalized with ARDS and require ventilation, have generated renewed interest in the underlying pathology of acute respiratory stress disorder," noted Dr. Maor. "Substantial data is emerging regarding the involvement of neutrophils, neutrophil extracellular traps (NETs), monocytes and macrophages in the development of ARDS secondary to COVID-19 and other viral infections; as well as the key involvement of CXCR4 as a mediator of those cells in the inflamed pulmonary tissue. Based on the scientific data indicating the importance of blocking the CXCR4/CXCL12 axis during ARDS, Motixafortide could be of potential benefit for such patients. COVID-19 case counts are again surging in many parts of the world and addressing ARDS has become a top global health priority."

The primary endpoint of the study is to assess the safety of Motixafortide in patients with ARDS secondary to COVID-19 and other respiratory viral infections. Respiratory parameters and inflammatory biomarkers will be assessed as exploratory endpoints. Up to 25 patients will be enrolled, with a preliminary analysis planned after ten patients have completed the initial treatment period. Based on the preliminary evaluation, a decision to continue or not will be conducted by Dr. Maor, together with the Company.

"We believe there is strong scientific rationale for exploring Motixafortide in ARDS, and we are grateful to Dr. Maor for initiating this study," stated Philip Serlin, Chief Executive Officer of BioLineRx. "We have compiled a significant body of data demonstrating Motixafortide's utility as a best-in-class CXCR4 inhibitor and we are eager to evaluate its effectiveness in blunting the cytokine storm that is associated with poor COVID-19 infection outcomes. We look forward to results from the preliminary analysis in the first half of next year.

"In parallel, we remain on track to announce full data from our Phase 2a COMBAT/KEYNOTE-202 study in pancreatic cancer, as well as interim results from our Phase 2b BLAST study in AML, by the end of this year," Mr. Serlin concluded.

About BioLineRx

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

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform currently being evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, and for which positive data in respect of the study's primary endpoint was recently announced from an interim analysis, resulting in early cessation of recruitment. Motixafortide is also being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD, as well as a Phase 2b study in consolidation AML.

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

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook, Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the coronavirus outbreak; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 12, 2020. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

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

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

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BioLineRx Announces Initiation of Phase 1b Clinical Trial in Patients with Acute Respiratory Distress Syndrome (ARDS) Secondary to COVID-19 and Other...

WINNIPEG -- A baby boy in Winnipeg is in need of a bone marrow transplant to survive, but he has yet to find a donor.

Three-month-old Boston has a rare disease called hemophagocytic lymphohistiocytosis HLH, a rare auto-inflammatory condition with his immune system.

His mother Simone Jannetta, who is a nurse at Grace Hospital, said they need someone who is of mixed race to donate stem cells.

Thats the only way to cure this, she said.

In the meantime, hes just receiving chemotherapy and steroids to help keep him well until then."

Jannetta said the reason they are having difficulty finding a match is because they need someone half Filipino and half Caucasian, and there are not many mixed-race donors currently in the Canadian and worldwide stem cell registries.

A TOUGH ROAD FOR FAMILY DEALING WITH HEALTH ISSUES

This is not the first time the family has dealt with a child facing health issues over the last few years.

When Jannettas daughter and Bostons older sister Beatrix was seven-months-old she presented to the emergency department with a fever and low blood counts. After a bone marrow biopsy, they learned she had a rare condition called autoimmune neutropenia.

So her immune system is not well either, shes very susceptible to infection too, Jannetta said.

Weve had a lot of back and forth with the hospital through herits been a tough road for us.

Anyone in Canada who wants to register to see if they are a match for Boston can go to the Canadian Blood Services website and look up the stem cell registry.

Boston also has his own link where you can register. The Canadian Blood Services will then mail you a kit with a swab, which you can send back once completed.

Its that simple, Jannetta said.

Youre put on the registry and Boston can then match with somebody.

For anyone who is thinking about registering to become a stem cell donor, Jannetta wants them to know they could save somebodys life.

Its not hard, theres no obligation follow through even if you do register, she said.

Theres just such a small representation of ethnically-diverse people on the registry and I just feel like everybody deserves a chance.

- With files from CTVs Nicole Dube.

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Three-month-old Winnipeg boy in need of bone marrow transplant to survive - CTV News Winnipeg

Voters in California have approved Proposition 14, which will pump billions of dollars into the state's stem cell research program. The Associated Press called the vote on Thursday, with 51 percent of ballots for and 49 percent against.

The result will allow the state to borrow $5.5 billion from investors for its stem cell agency, the California Institute for Regenerative Medicine (CIRM). The moneywhich taxpayers will repay with interest over the next 30 yearswill enable the institute to stay open, expand its research programs, and build new facilities.

Some $1.5 billion of the money will be spent researching conditions affecting the brain and central nervous system, such as Alzheimer's, Parkinson's, epilepsy, and stroke.

Unlike specialized cellssuch as blood cells or bone cellsstem cells do not have a specific job. Think of them as the raw materials of our bodies. When they divide, they can either renew and make new stem cells, or turn into specialized cells.

Despite making headlines for years, stem cell research is still in its early stages, with some treatments that have appeared to have worked in animals now going into clinical trials. These include treatments for macular degeneration, a common cause of blindness, as well as stroke, Lou Gehrig's disease, and spinal cord injuries.

It is hoped growing stem cells into specialized cells could also one day be used to replace damaged tissue and organs, for instance by helping the pancreas produce insulin in people with diabetes.

Currently, stem bone marrow transplantation is the most common form of stem cell therapy, used to treat blood cancer patients. Stem cell therapy has also been used for grafts of corneal stem cells, as well as skin grafts for burns victims.

As well as creating treatments, stem cell research can also help scientists understand diseases. Observing the cells in a lab as they turn into specialized cells, for instance, can provide clues on how we develop certain conditions.

There are a number of stem cells: embryonic stem cells, adult stem cells, adult stem cells tweaked to behave like embryonic stem cells, and stem cells found in the amniotic fluid and the umbilical cord of babies.

The controversy around stem cell research largely lies in the use of embryonic stem cells. These are taken from human embryos in their early stages of development. Opponents have likened this to abortion, although others disagree with this stance.

Embryonic stem cells used in research come from donations from IVF clinics, where an egg is fertilized with a sperm but not implanted into a patient because it is not needed. Embryonic stem cells are preferred over adult stem cells, as it may not be possible to specialize the latter and they are more likely to have abnormalities. But research suggests that it may be possible to turn adult stem cells into a wider range of specialized cells than previously thought, which may make them more useful.

In 2001, the Bush administration banned federal funding for stem cell research. This lead real estate developer Robert N. Klein II to initiate and help fund Proposition 71 in California. The aim was to enshrine the right to carry out stem cell research in the state's constitution, and establish CIRM. Klein was motivated by his son's experience with Type 1 diabetes, and his mother's Alzheimer's diagnosis. In 2004, Californians voted in favor of the proposition.

The institute has performed 64 clinical trials, and published over 3,000 scientific articles on the subject. But 16 years after Proposition 71 passed, CIRM started to run out of funds, and stopped accepting applications for new projects last year. This prompted the Californians for Stem Cell Research, Treatments and Cures political action committee (PAC) to lead the campaign for Proposition 14. Klein was among its supporters, as well as California governor Gavin Newsom, LA mayor Eric Garcetti, and the Michael J. Fox Foundation established by the Back to the Future actor and Parkison's disease patient who is its namesake.

On November 1, the foundation urged people via Twitter to vote in favor of Prop 14 to fund research on neurological disease. "Without this proposition vital research may come to a halt, delaying new treatments for people with," it said.

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As California Passes Prop 14, What Is Stem Cell Research and Why Is It Controversial? - Newsweek

Paula Grisanti, Opinion contributor Published 6:20 a.m. ET Nov. 9, 2020

This year, Nov.17 has been designated World Cord Blood Day, an annual event to raise awareness for the life-saving benefits of cord blood transplants while educating parents, health professionals and the general public about the need to preserve these precious cells.

Cord blood transplants are being used to treat more than 80 different diseases and conditions including blood cancers like leukemia and lymphoma, neuroblastoma (the most common cancer in infants), bone-marrow failure disorders, inherited blood disorders and rare immune system disorders. They are also showing new promise for conditions that have never had treatment options, like autism and brain injury.

The first cord blood stem cell transplant, an international effort between physicians in the U.S. and Europe, was performed in France in 1988. Stem cells collected from a newborns umbilical cord blood were used to save the life of her brother, a 5-year-old with Fanconi Anemia. Since then, there have been more than 40,000 cord blood transplants performed worldwide.

Now standard of care for cancers of the blood and a host of other life-threatening diseases, blood forming stem cells for transplantation can be collected from bone marrow, circulating bloodor a newborn babys umbilical cord blood. Some experts believe cord blood contains nearly 10 times the number of stem cells found in bone marrow.

Because umbilical cord stem cells are less mature than adult bone marrow stem cells, they are also less likely to be rejected and can be used when there isnt a perfect match.

Between these threeoptions, the easiest collection by far is from umbilical cord and placental tissue after a baby is born and the umbilical cord has been cut, at no risk to mother or child, in a process that typically takes 5 to 10 minutes. The cells are then frozen in liquid nitrogen and can be stored indefinitely in private or public cord blood banks.

To store your babys cord blood for use by your child and your family only, you make arrangements with a private cord blood bank ahead of delivery to collect and store the cells; the cost to you includes a collection fee of $1,500 to $2,000 and an annual storage fee of $100 to $125.

If you cant afford or dont wish to save your babys cord blood stem cells, you can donate them to a public cord blood bank at no cost to you or your family.

Its the equivalent of registering these potentially life-saving cells with the national bone marrow registry; they will be available to the families of other children who need to find a bone marrow match after a devastating diagnosis. Without information and education, however, 95% of all cord blood is discarded as medical waste.

Right now, there is no public cord banking option in Kentucky, although public cord blood banking is highly recommended by both the American Academy of Pediatrics (AAP) and the American Medical Association (AMA). There are fewer than 25 public or hybrid cord blood banks in the U.S., many limited to a specific geographic area. None of them include Kentucky.

The chances of finding a bone marrow match in your family are only about 25%, making the bone marrow and umbilical cord blood registries a lifeline in desperate situations. Odds are worse for African Americans and other ethnic minorities who are underrepresented on the registry and ethnicity matters in a bone marrow transplant.

Donating cord blood cells to a public bank adds to the library of cells that may save someones life and increases the chance of a match for all of us. Who benefits most? Children, patients with rare human leukocyte antigen (HLA) types and ethnic minorities.

We need to do two things: Make public cord blood banking an option in the commonwealth of Kentucky, and then encourage conversations between health care providers and expectant parents about preserving these life-saving cells.

There are 28 states with legislation that ask or mandates physicians to talk to expectant parents about cord blood banking. Kentucky is not one of them, but most of our surrounding states have such legislation in place.

Through a long-standing relationship between the National Stem Cell Foundationand world-renowned cord blood expert Dr. Joanne Kurtzberg, we have a path forward for training hospitals and collecting cells for storage at the Carolinas Cord Blood Bank (CCBB), one of the largest public cord blood banks in the world. Dr. Kurtzberg directs both the Pediatric Blood and Marrow Transplant (PBMT) program at Duke University and the CCBB.

She performed the worlds first unrelated cord blood transplant in 1993, paving the way for this now routine source of donor cells for children who need a bone marrow transplant and dont have a matched donor. She established the CCBB in 1998.

Paula Grisanti is CEO of the National Stem Cell Foundation.(Photo: provided)

While weve initiated discussions between Louisville hospital systems and the CCBB, we need to begin the process of education for parents, nursing and medical school students, residents, midwives, practicing OB-GYNs and the general public.

What a waste to discard these life-saving cells the future of current and developing therapies for disabling and life-threatening diseases depends on our ability to make sure that doesnt happen.

Dr. Paula Grisanti is CEO and a founding member of the National Stem Cell Foundation, headquartered in Louisville, Kentucky. She holds a D.M.D. and MBA from the University of Louisville and has been actively involved in new venture start-ups for most of her career.

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It's time for Kentucky to talk to expectant parents about benefits of cord blood banking - Courier Journal

Troy McKinley donated stem cells to help a stranger with blood cancer.

Think about your birthday wish this year? Did it involve saving someone's life?

A Reynoldsburg man's wish did.

Thirty-five-year-olf Troy McKinely wanted to make sure his birthday wish made a difference in someone else's life.

I wanted to do something big if possible. I've never donated blood before I don't even like needles, he said.

Two years ago, he decided he wanted to make his birthday more about gifts, and instead give the gift of life.

I thought it would be great to save a life so what can I do to help, he said

He found DKMS, the world's largest bone marrow and blood stem cell donor center.

The company sent him a swab kit and he waited to see if he would be a match. Two and a half years later, he was notified that his stem cells matched a patient who was diagnosed with blood cancer.

It was kind of like 'wow this is big. I don't know this person. I don't know anything about him or her.' It's kind of amazing feeling that it could be better for somebody else, he said.

McKinley said it only took a few hours to give the needed stem cells that doctors would later implant to the unknown patient.

I'm hoping that this gentleman I helped is feeling better for it and helped him in some way. Maybe it didn't give him everything back but he has some more time and we all want more time in the world so hopefully, it helped him, he said.

Time, we can all use more of it, but how many of us take the time to think about how we can give others more days on this earth.

It was a birthday wish McKinley says he'd do again knowing his kindness gave a stranger something more valuable than anything.

I think it's amazing to save someone's life. It's an incredible experience, he said.

About DKMS

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Reynoldsburg man makes unusual birthday wish: 'I wanted to save someone's life' - 10TV