Category Archives: Stem Cells

Stem cells are special human cells that are able to develop into many different cell types. This can range from muscle cells to brain cells. In some cases, they can also fix damaged tissues. Researchers believe that stem cell-based therapies may one day be used to treat serious illnesses such as paralysis and Alzheimer disease.

Stem cells are divided into 2 main forms. They are embryonic stem cells and adult stem cells.

Embryonic stem cells. The embryonic stem cells used in research today come from unused embryos. These result from an in vitro fertilization procedure. They are donated to science. These embryonic stem cells are pluripotent. This means that they can turn into more than one type of cell.

Adult stem cells. There are 2 types of adult stem cells.One type comes from fully developed tissues such as the brain, skin, and bone marrow. There are only small numbers of stem cells in these tissues. They are more likely to generate only certain types of cells. For example, a stem cell that comes from the liver will only make more liver cells.

The second type isinduced pluripotent stem cells. Theseare adult stem cells that have been changed in a lab to be more like embryonic stem cells. Scientists first reported that human stem cells could be changed in this way in 2006. Induced pluripotent stem cells don't seem to be different from embryonic stem cells, but scientists have not yet found one that can develop every kind of cell and tissue.

The only stem cells now used to treat disease are hematopoietic stem cells. These are the blood cell-forming adult stem cells found in bone marrow. Every type of blood cell in the bone marrow starts as a stem cell. Stem cells are immature cells that are able to make other blood cells that mature and function as needed.

These cells are used in procedures such as bone marrow transplants. These help people with cancer make new blood cells after their own hematopoietic stem cells have been killed by radiation therapy and chemotherapy. They may also be used to treat people with conditions such as Fanconi anemia. This is a blood disorder that causes the body's bone marrow to fail.

Stem cells may help your health in the future in many ways and through many new treatments. Researchers think that stem cells will be used to help create new tissue. For example, one day healthcare providers may be able to treat people with chronic heart disease. They can do this by growing healthy heart muscle cells in a lab and transplanting them into damaged hearts. Other treatments could target illnesses such as type 1 diabetes, spinal cord injuries, Alzheimer disease, and rheumatoid arthritis. New medicines could also be tested on cells made from pluripotent stem cells.

Stem cells need much more study before their use can be expanded. Scientists must first learn more about how embryonic stem cells develop. This will help them understand how to control the type of cells created from them. Another challenge is that the embryonic stem cells available today are likely to be rejected by the body. And some people find it morally troubling to use stem cells that come from embryos.

Scientists also face challenges when using adult pluripotent stem cells. These cells are hard to grow in a lab, so researchers are looking into ways to improve the process. These cells are also found in small amounts in the body. There is a greater chance that they could contain DNA problems.

Clinical trials that use stem cell therapies are currently being done in the U.S. If you are interested in trying this therapy to treat a certain condition, ask your healthcare provider how to find out about trials available in your area.

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What Are Stem Cells? - Lucile Packard Children's Hospital

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According to the report, the global stem cells market was valued at US$ 11.73 Bn in 2020 and is projected to expand at a CAGR of 10.4% from 2021 to 2028. Stem cells are defined as specialized cells of the human body that can develop into various different kinds of cells. Stem cells can form muscle cells, brain cells and all other cells in the body. Stem cells are used to treat various illnesses in the body.

North America was the largest market for stem cells in 2020. The region dominated the global market due to substantial investments in the field, impressive economic growth, increase in incidence of target chronic diseases, and technological progress. Moreover, technological advancements, increase in access to healthcare services, and entry of new manufacturers are the other factors likely to fuel the growth of the market in North America during the forecast period.

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Asia Pacific is projected to be a highly lucrative market for stem cells during the forecast period. The market in the region is anticipated to expand at a high CAGR during the forecast period. High per capita income has increased the consumption of diagnostic and therapy products in the region. Rapid expansion of the market in the region can be attributed to numerous government initiatives undertaken to improve the health care infrastructure. The market in Asia Pacific is estimated to expand rapidly compared to other regions due to shift in base of pharmaceutical companies and clinical research industries from developed to developing regions such as China and India. Moreover, changing lifestyles and increase in urbanization in these countries have led to a gradual escalation in the incidence of lifestyle-related diseases such as cancer, diabetes, and heart diseases.

Technological Advancements to Drive Market

Several companies are developing new approaches to culturing or utilizing stem cells for various applications. Stem cell technology is a rapidly developing field that combines the efforts of cell biologists, geneticists, and clinicians, and offers hope of effective treatment for various malignant and non-malignant diseases. The stem cell technology is progressing as a result of multidisciplinary effort, and advances in this technology have stimulated a rapid growth in the understanding of embryonic and postnatal neural development.

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Adult Stem Cells Segment to Dominate Global Market

In terms of product type, the global stem cells market has been classified into adult stem cells, human embryonic stem cells, and induced pluripotent stem cells. The adult stem cells segment accounted for leading share of the global market in 2020. The capability of adult stem cells to generate a large number of specialized cells lowers the risk of rejection and enables repair of damaged tissues.

Autologous Segment to Lead Market

Based on source, the global stem cells market has been bifurcated into autologous and allogenic. The autologous segment accounted for leading share of the global market in 2020. Autologous stem cells are used from ones own body to replace damaged bone marrow and hence it is safer and is commonly being practiced.

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Regenerative Medicines to be Highly Lucrative

In terms of application, the global stem cells market has been categorized into regenerative medicines (neurology, oncology, cardiology, and others) and drug discovery & development. The regenerative medicines segment accounted for major share of the global market in 2020, as regenerative medicine is a stem cell therapy and the medicines are made using stem cells in order to repair an injured tissue. Increase in the number of cardiac diseases and other health conditions drive the segment.

Therapeutics Companies Emerge as Major End-users

Based on end-user, the global stem cells market has been divided into therapeutics companies, cell & tissue banks, tools & reagents companies, and service companies. The therapeutics companies segment dominated the global stem cells market in 2020. The segment is driven by increase in usage of stem cells to treat various illnesses in the body. Therapeutic companies are increasing the utilization of stem cells for providing various therapies. However, the cell & tissue banks segment is projected to expand at a high CAGR during the forecast period. Increase in number of banks that carry out research on stem cells required for tissue & cell growth and elaborative use of stem cells to grow various cells & tissues can be attributed to the growth of the segment.

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Regional Analysis

In terms of region, the global stem cells market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global stem cells market in 2020, followed by Europe. Emerging markets in Asia Pacific hold immense growth potential due to increase in income levels in emerging markets such as India and China leading to a rise in healthcare spending.

Competition Landscape

The global stem cells market is fragmented in terms of number of players. Key players in the global market include STEMCELL Technologies, Inc., Astellas Pharma, Inc., Cellular Engineering Technologies, Inc., BioTime, Inc., Takara Bio, Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics, Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

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Stem Cells Market to Cross US$ 25.68 Bn by 2028, Increasing Demand for Stem Cells in Regenerative Medicines Accelerates Market Growth - BioSpace

MARSHALL, Ill. (WAWV/WTWO) A young Illinois woman welcomed the man that saved her life from across the world into her hometown.

Stem Cell Recipient 21-year-old Taylor Mason has had anything but a normal life, at 15-years-old she was diagnosed with Acute Myeloid Leukemia. After months of treatment and remission the Leukemia came back when she was 17.

They told me I needed a stem cell transplant so I did pre chemo, IVF fertility, and then I did a stem cell transplant, Mason said. All I was told is that it was from a donor in Europe.

Around the same time Stem Cell Donor Daniel Riedinger of Germany just happened to sign up to be a donor. After many tests he was notified he would be the best possible donor for Masons transplant, who he did not know at the time.

I was registered and I knew there was someone that needed my stem cells who would not survive without them, Riedinger said. It was not a question for me.

Mason had to wait two years in the registry and once she was well enough after her transplant she reached out to Reidinger.

I thought about what ones says and what one does for the person that saves your life for awhile, Mason said. Simply just hugging them and meeting them and knowing they are the reason your here is everything.

Mason visited Riedinger in Germany last year however, this is Riedingers first time in Masons hometown. Friends and family of Mason gathered at the Marshall VFW to meet Riedinger.

I now have a new sister and a new family, Riedinger said. I cant explain its unbelievable.

Mason said Riedinger has become a huge part of her life.

Hes a big part of our lives now I mean every holiday we contact him or he contacts, Mason said. We are family I mean we are literally blood now.

Both Riedinger and Mason encourage those considering stem cell donation to sign up.

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Woman welcomes a man that saved her life from across the world to her hometown - MyWabashValley.com

An investigative stem cell-based therapy called PEC-Direct, designed to act as a replacement pancreas, has the potential to provide blood sugar control in patients with high-risk type 1 diabetes, suggests a clinical study presented Saturday, June 11at ENDO 2022, the Endocrine Societys annual meeting in Atlanta, Ga.

The study found multiple patients using the new treatment had clinically relevant increases in C-peptide, a substance made in the pancreas along with insulin. C-peptide and insulin are released from the pancreas at the same time and in about equal amounts, so measuring C-peptide can show how much insulin the body is making.

This research represents the first instance in multiple patients of clinically relevant increases in C-peptide, indicative of insulin production, with a stem cell-based therapy delivered in a device, according to Manasi Sinha Jaiman, M.D., M.P.H., Chief Medical Officer of ViaCyte, Inc., in San Diego, Calif., the company that makes PEC-Direct.

Patients with type 1 diabetes eventually lose the ability to produce their own insulin to control blood sugar levels. Patients must frequently check those levels with finger sticks, inject multiple insulin shots or carry around bulky devices. The injection of insulin also carries the risk of accidentally lowering blood sugar to dangerous levels.

The PEC-Direct device is designed to provide a long-term, stable source of insulin to regulate glucose levels. The device comprises a pouch containing stem-cell derived pancreatic cells which mature into insulin-producing cells once implanted into the body to regulate glucose levels. The open device membrane allows blood vessels to grow into the device to contact the cells. To prevent an immune reaction, patients take immunosuppressive drugs.

The treatment is meant for patients with high-risk type 1 diabetes, who may be especially vulnerable to acute complications due to factors such as recurrent severe low blood sugar, or frequent and extreme blood sugar fluctuations that are difficult to control.

The study included 10 adults with type 1 diabetes who had received their diagnosis at least 5 years prior to the start of the study and were not able to tell when their blood sugar went too low (called hypoglycemia unawareness). Initial data from one patient showed clinically relevant levels of stimulated C-peptide and corresponding improvements in blood glucose control within six months after implantation of PEC-Direct. Since then, increased C-peptide levels were seen in multiple patients, along with decreases in HbA1C (a blood test that measures average blood sugar levels over the past three months) by as much as 1.5%, and decreases in the amount of insulin patients needed to administer by as much as 70%.

The results suggest stem cell-based replacement therapy has the potential to provide blood glucose control and could one day eliminate the need for injecting or dosing insulin externally, Jaiman said. The study provides further proof-of-concept that continued optimization of PEC-Direct has promise as a functional cure for type 1 diabetes.

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Endocrinologists are at the core of solving the most pressing health problems of our time, from diabetes and obesity to infertility, bone health, and hormone-related cancers. The Endocrine Society is the worlds oldest and largest organization of scientists devoted to hormone research and physicians who care for people with hormone-related conditions.

The Society has more than 18,000 members, including scientists, physicians, educators, nurses and students in 122 countries. To learn more about the Society and the field of endocrinology, visit our site atwww.endocrine.org. Follow us on Twitter at@TheEndoSocietyand@EndoMedia.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Stem-cell based therapy shows promise in treating high-risk type 1 diabetes - EurekAlert

Magdalena Zernicka-Goetz, Bren Professor of Biology and Biological Engineering and affiliated faculty member with Caltech'sTianqiao and Chrissy Chen Institute for Neuroscience, has been awarded the 2022 NOMIS Distinguished Scientist and Scholar Award. Established in 2016, the award is presented to "pioneering scientists and scholars who, through their innovative, groundbreaking research, have made a significant contribution to their respective fields and who inspire the world around them," according to the NOMIS Foundation.

Zernicka-Goetz's research addresses fundamental questions about how life begins, such as: What drives a fertilized egg to divide and grow until it becomes 40 trillion cells, and how do these cells know how to make a person? To address these questions, she has developed methods for tracking living embryos to determine how stem cells are first created, establish their fates, and work together to shape the body. She also pioneered methods to grow embryos beyond implantation, techniques that won the "People's Choice Scientific Breakthrough of the Year" in 2016 inSciencemagazine. Her team used these methods to createthe first complete embryo models from stem cells that develop like natural embryos.

In 2021, the team determined the molecular signals involved in how an embryo becomesasymmetrical and polarizedand how the embryo forms itshead-to-tail body axis.

Zernicka-Goetz received her PhD from Warsaw University and joined the Caltech faculty in 2019. Prior to Caltech, she was professor of mammalian development and stem cell biology at the University of Cambridge, England. She is a fellow of the British Academy of Medical Science, the Polish Academy of Sciences, and a recipient of anNIH Director's Pioneer Award and the 2022 Edwin G. Conklin Medal from the Society for Developmental Biology.

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Zernicka-Goetz Receives Honors from the NOMIS Foundation - Caltech

An Australian-first study has found collecting and storing cells from a babys umbilical cord and later transferring them to a sibling who has cerebral palsy has been found to be safe.

The clinical trial, led by theMurdoch Childrens Research Institute (MCRI)and published inCytotherapy, stated fully matched umbilical cord blood cells from one child could safely be infused into a sibling with cerebral palsy. The study was funded by the Cerebral Palsy Alliance Research Foundation and Cell Care cord blood bank.

Only one serious adverse reaction related to the study was reported, while sixof the 12 participants experienced minor adversereactions. All adverse reactions were treated effectively in hospital.

Murdoch Childrens researcherDr Kylie Cromptonsaid while there was no cure for cerebral palsy, cord blood cells have the potential to improve brain injury and gross motor function because of their ability to activate repair processes and regenerate some tissues in the human body.

Our study found infusing matched sibling cord blood cells into children with cerebral palsy is a relatively safe procedure however it should only be conducted in tertiary hospitals with facilities to treat the infrequent adverse reactions, she said.

The phase I clinical trial involved 12 participants, aged 1-16 years, from across Australia who received a siblings cord blood cells and was followed up for 12 months after the infusion.

Most of the participants, monitored for a year, showed typical developmental progress for children with cerebral palsy, Improvements in gross motor function were noted in three children, three months after the infusion. Any changes were less pronounced one year afterwards.

Dr Crompton said the greatest gross motor improvements were seen in younger children who had not yet reached 90 per cent of their predicted gross motor skill potential.

This suggests that intervention may be more effective in the first few years of life, she said. To find outifthis is the case, we and other researchers are now planning additional trials to understand these effects better.

Cerebral palsy is caused by damage to the developing brain while in utero, during birth or in the first years of life. The congenital disorder affects around two per 1000 live births across the world, making it the most common physical disability in childhood. An Australian child is born with cerebral palsy every 20 hours.

Murdoch ChildrensProfessor Dinah Reddihoughsaid the findings were a valuable stepping-stone towards establishing the safety and feasibility of using umbilical cord blood in the management of cerebral palsy. However, not all children with cerebral palsy will have fully matched siblings cells available.

Investigating a cell product that is available to the broader cerebral palsy community, rather than just those with matched siblings who have cord blood stored, is an important next step, she said.

Further research is also needed to investigate whether different forms of cerebral palsy are more amenable to change following cord blood cell infusion, what cell dose is required and if multiple doses would provide a better outcome.

Diagnosed with cerebral palsy, Charlotte (pictured, right)at age two couldnt grip a water bottle or pen, verbally communicate and was yet to take her first steps. After taking part in the trial, mum Laura West said it was like a light switch had been turned on.

However, Laura said Charlottes developmental progression had slowed over time since the infusion.

While pregnant with her youngest daughter, Emma (pictured with Charlotte, below, left), Laura decided to collect and store cells from her umbilical cord to have them later transferred to Charlotte. Laura accessedCell Carescord blood collection and storage program for siblings of a child with cerebral palsy. The free program ensures these stem cells are collected in a fully audited and government-approved manner so that they will be available for use in future clinical trials in Australia.

Laura said the changes in Charlotte, now aged seven, were evident after starting the cell infusion.

Charlotte was quite static in her development and progress up until the infusion and then we saw this noticeable difference, she said.

It was striking at first and then settled into a steady improvement. Charlotte was always fed through a tube but quickly learned to drink out of a bottle. We never thought she would be able to grip a pencil properly but then suddenly she was holding it correctly.

She went from being propped up with pillows to sitting, standing and running. It was huge to see her so independent and able to do these things by herself. The rate of change in Charlotte in the immediate aftermath was so pronounced but now her development has slowed to baby steps.

Were glad the researchers will continue the work to see if the intervention may be more effective in the first few years of life, which will offer hope to other families.

Professor Nadia Badawi,Cerebral Palsy AllianceChair of Cerebral Palsy Research at The University of Sydney, said this was a landmark study, which gave a new understanding of how umbilical cord blood cells might one day be used to improve the lives of children with the disorder.

We are indebted to the children and their families who participated in this research, and who have helped to pave the way for many future trials, she said.

At Cerebral Palsy Alliance, we are committed to carrying out research that empowers people with cerebral palsy and their families. From surveying our community, we know that people with cerebral palsy and their families want more research into potential therapies, and this study is an important step forward in demonstrating the safety of using cord blood cells in an Australian hospital setting.

Reference: Crompton K, Novak I, Fahey M, et al. Safety of sibling cord blood cell infusion for children with cerebral palsy. Cytotherapy. 2022;0(0). doi:10.1016/j.jcyt.2022.01.003

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Umbilical Cord Cells Can Be Donated to Siblings With Cerebral Palsy - Technology Networks

Bioengineering Professor Changqing Li is building a high-resolution CT imaging scanner that will allow scientists to study and understand how oxygen plays a role in cancer therapy and stem cells growing in deep tissue such as bone marrow, and possibly develop new advances to culture stem cells outside the body and therapeutics to control tumor growth.

Funded by grants from the National Institutes of Health (NIH), Li and fellow bioengineering Professor Joel Spencer are working with molecules that, when excited by the X-ray beam, emit light in the visible spectrum. By measuring the intensity of the light and how long it takes to be emitted, the researchers can detect how much oxygen is present in the tissue.

The project, called Bio-tissue Oxygenation Nanophosphor Enabled Sensing (BONES), would be a brand-new medical imaging technique with an unprecedented combination of chemical sensitivity and high-spatial resolution imaging through deep tissue.

The industry-academia partnership grant provides about $1.9 million under the Small Business Technology Transfer program. By partnering with Bay Area company Sigray Inc., the researchers use the power of a bright X-ray tube and a fancy X-ray optics focusing a superfine X-ray beam to peer through thick tissue.

Hypoxic (low oxygen) conditions affect many medical conditions such as cancer, chronic kidney disease and failed organ transplant, but the heterogeneous nature of hypoxia is not well understood, Li said.

For example, bone marrow is a particularly hypoxic tissue, and its low-oxygen environment enables bone marrow to maintain adult stem cells. But the same conditions are believed to harbor cancer cells, which is why bone is a common cancer metastasis site.

Both Li and Spencer focus on biomedical imaging. Spencer had already developed a technique for visualizing stem cells in live, intact mice, but Lis idea for BONES would allow researchers to see even deeper inside to directly measure molecular oxygen.

Its not easy to measure oxygenation in deep tissue, Li said. Right now, we can use a needle to extract samples, but it can only study that one spot. The proposed novel technology, BONES, will let us see inside a whole area of tissue such as tumors and bone marrow.

Changes in the oxygenation levels of tumors can be indicative of responses to therapies, Spencer said. And in bone marrow, he explained, oxygenation is important for the tissues health.

That can also be in the context of treatment, such as with bone marrow transplants, he said. Youd want to look at the recovery of the bone marrow. Oxygenation changes during recovery, but right now, no one has a way to look deep into the center of the marrow.

Besides being colleagues in the Department of Bioengineering, both Li and Spencer are affiliated with the Health Sciences Research Institute. The work on BONES under this grant lasts through 2024. It builds on work Li has been doing since he joined the campus in 2012, including through a $2.5 million R01 grant from the NIH to develop a first-of-its kind X-ray luminescence tomography scanner that allows researchers to visualize how cancer progresses and monitor the effectiveness of novel drug-delivery systems in live animals without invasive surgeries or euthanizing the animals.

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Bioengineers Work on New Technology to Look Deep Inside Living Tissue and Tumors | Newsroom - University of California, Merced

image:The cell nuclei are in cyan gray, the stromal cells in red and green, and the blood vessels are stained in blue. view more

Credit: Stroma, Inflammation & Tissue Repair Unit Institut Pasteur

The intestine is responsible for absorbing nutrients while protecting the body from external aggression, a task performed by a complex intestinal barrier. Scientists from the Institut Pasteur demonstrated in a mouse model that a population of tissue-resident cells known as stromal cells is crucial for the development of a functional intestinal barrier in the first few weeks after birth. Absence of these cells induces a defect in postnatal growth and increases susceptibility to intestinal inflammatory diseases. These findings were published in the journal Cell Stem Cell on May 5, 2022.

The intestinal barrier allows assimilation of nutrients while ensuring a proper defense against potential pathogens. The first weeks after birth are critical in this process as the intestine undergoes important steps of maturation and becomes colonized by microorganisms. In a few weeks, intestinal stem cells become restricted to the crypts, while differentiated epithelial cells ensuring absorption and defense migrate to the villi, which are in contact with the digested food and the microbiota.

In this study, scientists from the Institut Pasteur used an animal model to identify a population of stromal cells that develops before weaning age and promotes the maturation of the intestinal barrier. The stromal cells identified by the scientists are in contact with epithelial cells and receive a signal via their growth factor receptor (PDGFRa). This signal induces a new "mature" stromal niche that promotes intestinal epithelial cell differentiation and balanced immunity.

If the signal is blocked, the intestine does not develop properly in the first weeks of life, inducing a delay in postnatal growth and perturbations of intestinal homeostasis. At the "young adult" stage, individuals with such an overreactive intestine will develop pathological responses to injury and increased susceptibility to intestinal inflammation.

"We identified a subpopulation of stromal cells in the first weeks after birth that is essential for the maturation of the postnatal intestine. By forming a specialized niche in the villi, these stromal cells provide local cues for the proper differentiation of epithelial and immune cells, which are key players in the intestinal barrier," explains Lucie Peduto, Head of the Stroma, Inflammation & Tissue Repair Unit (Institut Pasteur/Inserm) and lead author of the study.

Though the study was performed in an animal model, these stromal cells have also been identified in humans. This study could shed new light on the mechanisms underlying development of intestinal inflammatory diseases in childhood and young adulthood, paving the way for novel therapeutic approaches.

The research was funded by the European Research Council (ERC), Inserm, and the French Foundation for Medical Research.

PDGFR-induced stromal maturation is required to restrain postnatal intestinal epithelial stemness and promote defense mechanisms

5-May-2022

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Stromal cells, maestros of the intestine - EurekAlert

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, today announced completion of the rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant.

The BLA submission marks an important milestone for both Gamida and the transplant community, as omidubicel has the potential to be the first approved advanced cell therapy product for allogeneic stem cell transplantation, said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. Completion of this BLA submission is a key inflection point in our mission to deliver a new treatment option for patients with blood cancers. We look forward to working closely with the FDA to bring this potentially important therapy to patients.

The FDA has 60 days to determine whether the BLA for omidubicel is acceptable for filing. The omidubicel BLA is supported by the statistically significant results from Gamida Cells pivotal Phase 3 study, the results of which were published in Blood, the official journal of the American Society of Hematology. For the studys primary endpoint, the median time to neutrophil engraftment in patients with hematologic malignancies undergoing allogeneic bone marrow transplant receiving omidubicel compared to standard umbilical cord blood (UCB), the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001).

In key secondary endpoints of this Phase 3 study: platelet engraftment was significantly accelerated [55 percent of patients randomized to omidubicel achieving platelet engraftment by day 42, compared to 35 percent for the comparator (p = 0.028)]; the rate of infection was significantly reduced [cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.03)]; and hospitalization in the first 100 days after transplant was significantly reduced [median number of days alive and out of hospital for patients randomized to omidubicel of 61 days, compared to 48 days for the comparator (p = 0.005)]. Omidubicel was generally well tolerated in the Phase 3 study.

The full Blood manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.

Market Opportunity

In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant.1 Unfortunately, it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source.2 Omidubicel, if approved, has the potential to improve outcomes for patients based on transplanter feedback and to potentially increase access for patients to get to transplant. Omidubicel, if approved, has the potential to treat approximately 2,000 2,500 patients each year in the U.S.

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types including stem cells and natural killer cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cells product candidates (including omidubicel), anticipated regulatory filings (including the timing of submission of the BLA for omidubicel to the FDA), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of Gamida Cells product candidates (including omidubicel), and Gamida Cells expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cells goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on March 24, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.

1CIBMTR 2019 allogeneic transplants in patients 12+ years with hematological malignancies.2Gamida Cell market research

Read more from the original source:
Gamida Cell Completes Rolling Biologics License Application Submission to the FDA for Omidubicel - Business Wire

As baby number fours screams rang out from the birthing suite, parents Laura and Paul West held their breath.

Not just for their newborn baby girl, Emma, but for their third child, Charlotte, who was desperately awaiting the arrival of her sister - a much-needed stem cell donor.

As the umbilical cord and placenta were whisked away, Laura whispered to her tiny, healthy baby that she was destined to save her big sisters life.

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Charlotte, who was living with cerebral palsy, couldnt walk, talk or eat by herself.

Doctors told the West family she would never know her own name.

But five years after Emmas birth, Charlotte is now running.

Emma is the gift that keeps on giving, Laura tells 7Life.

When the West Australian woman was 22 weeks pregnant with Charlotte, doctors discovered an abnormality.

She had diaphragmatic hernia - a hole in the diaphragm which allowed organs to travel upwards and develop in the chest region.

Charlotte also had a hole in her heart, a number of gut issues, and white matter on the brain, among other issues.

Specialists suggested Paul and Laura consider termination - Charlotte had a 10 per cent survival rate, at best.

At home, the couple tried to come to terms with the gravity of the situation.

They gently broke the news to their two children, Josh, then seven, and Isabella, then four.

Every day they would rub my belly and go, Good morning Charlotte. She was already part of our family, Laura says.

So when we told Josh, he said, Why dont we let Charlotte decide? If she wants to fight she will fight. What if it were me, mum?.

The family agreed with the little boy.

They chose against termination and let Charlotte make the decision for herself, praying every day for her safe and healthy arrival.

When the time came, the hospital room was filled with more than 30 medical staff and grief counsellors, prepared for the worst possible outcome.

But Charlotte chose life.

In her first few days, she was in and out of surgery - each day touch and go.

When she was really sick those first few days, Josh turns to me and said Dont you wish you could just take her soul and place it in a healthy body so she can enjoy life like we do, Laura says.

He was seven. He just says the most profound things.

Charlotte was diagnosed with cerebral palsy and epilepsy and told she would need care for the rest of her life.

Her prognosis was unknown.

Laura and Paul began making modifications around the home for their little girl and were happy she was part of their family.

Josh and Isabella doted on their new sister and took on new roles in the family, helping care for Charlotte.

There were more surgeries, therapy and ICU visits, and Charlotte battled every new challenge with a grin.

With a wheelchair, oxygen tank and a feeding tube, Charlotte became Lauras best friend and the pair never left each others side.

Then when Charlotte was two, Laura fell pregnant again.

As she waited with Charlotte for an ultrasound appointment early in her pregnancy, Laura caught a woman staring at her daughter from across the room.

I was pretty used to that and just thought, Here we go again, Laura says.

She asked me what was wrong with Charlotte and I started to tell her.

The woman stopped Laura mid-sentence and revealed she, too, had a daughter with cerebral palsy.

She then pointed to her pregnant bump - and revealed she had conceived in the hopes of using her unborn childs stem cells to help her eldest.

Cells of the newborns umbilical cord would be infused into her eldest child, hoping to help increase movement and brain development.

She spoke about a medical trial in Melbourne, she quickly grabbed a scrap bit of paper and wrote all the details on it and handed it to me, Laura says.

Back at home, Laura dug through her bag to find the crumpled piece of paper.

I thought, What was the harm? so I gave the number a call, she says.

The trial was purely focused on the safety of the procedure around sibling stem cell infusions, and Laura was told they had no evidence that stem cells could benefit Charlotte at that stage.

But, what did I have to lose, Laura says.

As she began her own research into stem cell trials, she discovered children around the world were benefiting from the infusion - with dramatic changes in development and increased independence.

For the Wests, the trial sparked hope for their daughters future. So they signed up.

Laura and Paul were told Charlotte had just a 25 per cent chance of matching with her new sibling.

And they would need to wait for the arrival of their latest bundle to test their compatibility.

When Emma was welcomed into the world, the family was overjoyed - not just by baby number four but the countless possibilities for Charlotte.

Emmas placenta and umbilical cord were quickly sent for testing.

When we finally got the call we couldnt believe it - they were a match, Laura says.

Then aged three-and-a-half, Charlotte and Laura flew to Melbourne for the two-hour procedure, which went flawlessly.

From what they (doctors) had told us, we had no expectations, Laura says.

Just two days after the infusion, Charlotte reached for a bottle and began making a sucking motion with her mouth.

I know it seems like such a tiny moment but she had never done that before, she is tube fed, the mum says.

A few weeks later, she was rolling - then crawling, standing and finally walking.

With no previous motor skills, Charlotte was speeding past every milestone she had missed in her short three years of life.

We were told she would have no chance of ever talking or walking, she will never know you, she will never form bonds or relationships with people, she will never know her family, Laura says.

Now she knows her family, she loves us dearly, she can hold a pencil and draw.

Every day Charlotte, now eight, continues to improve.

Josh and Isabella share a special bond with Charlotte, but Emma and her sisters connection is on a different level.

Emma grabs Charlottes hand and takes her to the trampoline and just encourages her, Laura says.

Laura has connected with the woman from the doctors waiting room and the pair share their journeys with stem cell research.

Laura calls the meeting her turning point, saying that without having encountered the kind stranger, Charlotte wouldnt be where she is today.

Doctors always give you the grim odds of everything, the mum says.

But looking at Charlotte now, she is doing everything she was told she wouldnt.

Laura is sharing her familys story to raise awareness of the importance of research into stem cell therapies, cord blood and tissue storage, and initiatives such as Cell Cares Sibling Cord Blood Collection program.

Visit http://www.cellcare.com.au for more information.

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Stem cell treatment for Cerebral Palsy: Aussie mum tells how baby was born to save her sister - 7NEWS