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Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with…

By daniellenierenberg

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced positive preliminary findings from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 (stem cell factor receptor) monoclonal antibody, as a conditioning agent in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing hematopoietic (blood) cell transplantation.

Data from the first six patients who received a single dose of JSP191 prior to transplantation showed successful engraftment in all six patients. Complete donor myeloid chimerism (equal or greater than 95%) was observed in five of six evaluable patients at 28 days, and all three evaluable patients had total donor chimerism equal or greater than 95% observed at day 90. In addition, at 28 days, three of five evaluable patients showed complete eradication of measurable residual disease (MRD) as measured by next-generation sequencing. Two of the five evaluable patients showed substantial reductions in MRD. No treatment-related serious adverse events were reported.

The findings were presented by lead investigator Lori Muffly, M.D., M.S., Assistant Professor of Medicine (Blood and Bone Marrow Transplantation) at Stanford Medicine, as a late-breaking abstract at the 2021 Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

These early clinical results are the first to demonstrate that JSP191 administered in combination with a standard non-myeloablative regimen of low-dose radiation and fludarabine is well tolerated and can clear measurable residual disease in older adults with MDS or AML undergoing hematopoietic cell transplantation a patient population with historically few options, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. These patients could be cured by hematopoietic cell transplantation, but the standard-of-care myeloablative conditioning regimens used today are highly toxic and associated with high rates of morbidity and mortality particularly in older adults. Traditional lower intensity transplant conditioning regimens are better tolerated in older adults, but are associated with higher rates of relapse in MDS/AML patients with measurable residual disease. JSP191, a well-tolerated biologic conditioning agent that targets and depletes both normal hematopoietic stem cells and those that initiate MDS and AML, has the potential to be a curative option for these patients.

The open-label, multicenter Phase 1 study (JSP-CP-003) is evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine among patients age 65 to 74 years with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for full myeloablative conditioning. The primary outcome measure of the study is the safety and tolerability of JSP191 as a conditioning regimen up to one year following a donor cell transplant.

We designed JSP191 to be given as outpatient conditioning and to have both the efficacy and safety profile required for use in newborn patients and older patients for successful outcomes, said Wendy Pang, M.D., Ph.D. Executive Director, Research and Translational Medicine, of Jasper Therapeutics. We are enthusiastic about the reduction of measurable residual disease seen in these patients, especially given that it is associated with improved relapse-free survival. We are excited to continue our research in MDS/AML, with plans for an expanded study. We are evaluating JSP191, the only antibody of its kind, in two ongoing clinical studies and are encouraged by the positive clinical data seen to date.

About MDS and AML

Myelodysplastic syndromes (MDS) are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells.1 In about one in three patients, MDS can progress to acute myeloid leukemia (AML), a rapidly progressing cancer of the bone marrow cells.1 Both are diseases of the elderly with high mortality. Each year, about 5,000 patients with MDS and 8,000 people with AML in the G7 countries receive hematopoietic cell transplants. These transplants are curative but are underused due to the toxicity of the current high-intensity conditioning regimen, which includes the chemotherapy agents busulfan and fludarabine.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplantation. A Phase 1/2 dose-escalation and expansion trial is evaluating JSP191 as a sole conditioning agent to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplantation for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting showed that a single dose of JSP191 administered prior to stem cell transplantation in a 6-month-old infant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. A Phase 1 clinical study is evaluating JSP191 in combination with another low-intensity conditioning regimen in patients with MDS or AML undergoing hematopoietic cell transplantation. For more information about the design of these two ongoing clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

1 https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html

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Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of…

By daniellenierenberg

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced the results of a Phase 3 clinical study of omidubicel presented in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), or the TCT Meetings. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the Gamida Cell website.

The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. The comparator, a transplant with umbilical cord blood, has been historically shown to result in low incidence of graft versus host disease (GvHD) in relation to other graft sources, and in this study, omidubicel demonstrated a GvHD profile similar to the comparator. Moreover, previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study receiving their transplant more than 10 years ago. The data presented at this meeting indicate that omidubicel has the potential to be considered a new standard of care for patients who are in need of stem cell transplantation but do not have access to a matched donor.

Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings

Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The studys intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and a key milestone in a patients recovery from a bone marrow transplant. 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).

All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the 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 = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in December 2020.

Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, Ph.D., chief executive officer of Gamida Cell. Sharing the results of the Phase 3 study of omidubicel with the transplant community is a major moment for Gamida Cell, and we are forever grateful to the patients who participated in this study, their caregivers, and the work of the investigators and their teams.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter 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 anticipated regulatory submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the progress and expansion of Gamida Cells manufacturing capabilities and other commercialization efforts and clinical, scientific, regulatory and technical developments. 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 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, its Report on Form 6-K filed with the SEC on August 12, 2020, 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. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.

2 Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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World Symposium Orchard leads the crop of Hurler syndrome hopefuls – Vantage

By daniellenierenberg

Gene therapy companies have been under pressure lately, but Orchard Therapeutics got a lift yesterday from promising early data with its mucopolysaccharidosis type I candidateOTL-203.

The company is seeking to supersede the current standard of care, enzyme-replacement therapy or bone marrow transplant. But other gene therapy contenders are not too far behind, notablyRegenxbio, which in December started a proof-of-concept study of its rival project, RGX-111.

Good IDUA

Both projects seek to deliver the -l-iduronidase (IDUA) gene, which is mutated in MPS-I, leading to a deficiency of the IDUA enzyme. This enzyme usually breaks down glycosaminoglycans (GAGs), so in MPS-I patients these build up, causing tissue and organ damage. Symptoms of MSP-I, also known as Hurler syndrome, include cognitive impairment and skeletal deformity; if left untreated, patients rarely survive beyond the age of 10.

And both OTL-203 and RGX-111 are designed as one-time therapies, whereas the current enzyme replacement, Biomarin/Sanofis Aldurazyme, is given intravenously once a week.

However, the gene therapy candidates go about restoring IDUA enzyme activity in different ways. OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused.

RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot cross the blood-brain barrier.

Getting into the brain should not be a problem for OTL-203 either, Orchards head of medical affairs, Leslie Meltzer, told Evaluate Vantage. She explained that hematopoietic stem cells naturally cross the blood-brain barrier and, once in the CNS, differentiate into a microglial-like cell.

This claim appears to be supported by the latest data, which admittedly come in just a handful of subjects. The eight-patient phase I/II trial, presented at the World Symposium yesterday, found increases in the IDUA enzyme in patients blood and cerebrospinal fluid. There was also a decrease in GAGs in the CSF and urine.

Encouragingly, this activity appears to have translated into a clinical benefit: all eight patients showed stable cognitive scores and stable motor function versus baseline, as well as growth in the normal range for patients age.

Its a progressive disease, so youd expect these things to worsen over time, but the fact they continued to be stable is very promising, Ms Meltzer said.She admitted that the data were early, with only around a year of follow-up on most of the clinical endpoints.

Orchard plans to start a registrational study by the end of this year.Ms Meltzer would not give any details ondesign, saying this would be finalised after feedback from regulators.

Regenxbios proof-of-concept study of RGX-111 is due to complete in November, putting the project about a year behind OTL-203.

One candidate that will go no further is Sangamos SB-318. The company reported disappointing data with the in vivo zinc finger nuclease genome-editing project two years ago, and has since said it would focus on second-generation zinc finger projects.

Still, even two gene therapies might be too many for an ultra-rare disease like MPS-I, which affects just one in 100,000 people. Asked whether this market could support more than one gene therapy, Ms Meltzer said newborn screening recently implemented in countries including the UScould lead to a revision of that estimate.

But, as in other rare disorders that have attracted several gene therapy players, a battle over a limited patient pool could be shaping up.

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Growth Factors of Hematopoietic Stem Cell Transplantation (HSCT) Market with Emerging Trends and Revenue Estimation By 2026 – AlgosOnline

By daniellenierenberg

Global Hematopoietic Stem Cell Transplantation (HSCT) Report offers market size, share, overview, segmentation by types, application, countries, key manufactures, cost analysis, industrial chain, sourcing strategy, downstream buyers, marketing strategy analysis, distributors/traders, factors affecting market, forecast and other important information for key insight.

The research report on Hematopoietic Stem Cell Transplantation (HSCT) market is an in-depth analysis of pivotal drivers, challenges, and growth prospects prevailing in the business space and their impact on the expansion graph over the ensuing years.

Request a sample Report of Hematopoietic Stem Cell Transplantation (HSCT) Market at:https://www.marketstudyreport.com/request-a-sample/3147041?utm_source=algosonline.com&utm_medium=Ram

According to the report, Hematopoietic Stem Cell Transplantation (HSCT) market is anticipated to record a y-o-y growth rate of XX% over the analysis duration (2020-2026) and is poised to amass substantial revenues by the end of study term.

Various disruptions are being observed on account of lockdowns imposed to control COVID-19 spread, leading to uncertainties. While all industry verticals are facing revenues troubles presently, some sectors will continue to fight these challenges even as economy emerges from pandemic blowback.

As a result, all the businesses are revising their budgets to formulate new profit trajectory for the forthcoming years. Our thorough analysis of this industry space will enable you to come up with contingency plans and prepare you to manage market qualms.

The research document scrutinizes different segmentations to offer comprehensive insights about the growth opportunities in the market.

Major points summarized in Hematopoietic Stem Cell Transplantation (HSCT) market report:

Elucidating Hematopoietic Stem Cell Transplantation (HSCT) market segmentations:

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Future Prospects: Future opportunities are estimated to emerge in the industry

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I Survived Cancer, and Then I Needed to Remember How to Live – The Atlantic

By daniellenierenberg

Ibram X. Kendi: What I learned from cancer

This is where I find myself, at the threshold between an old familiar state and an unknown future. Cancer no longer lives in my blood, but it lives on in other ways, dominating my identity, my relationships, my work, and my thoughts. Im done with chemo but I still have my port, which my doctors are waiting to remove until Im further out of the woods. Im left with the question of how to repatriate myself to the kingdom of the well, and whether I ever fully can. No treatment protocols or discharge instructions can guide this part of my trajectory. The way forward is going to have to be my own.

My first foray into this new selfhood is learning how to drive. As I get more comfortable behind the wheel, a hazy idea begins to crystallize into a grand plan. I need to leave the familiar, to trust that I can navigate the world alone. I need to become my own caregiver. It took me a while to say I was a cancer patient. Its time for me to figure out who I am now. By the time I finally pass my drivers test, the next step is obvious: Im going to embark on a solo cross-country road trip.

Over the next few weeks, I pack all of my belongings into boxes, put the boxes into storage, and sublet my apartment. I cant afford to buy my own car but my friend Gideon generously offers the use of his old Subaru. Between the extra income from renting out my apartment and the $4,000 in my savings account, I should be able to make do. I plan to camp and crash on couches as often as possible, staying in only the occasional motel room. I scour Craigslist for secondhand camping gear and buy a portable propane gas stove, a subzero sleeping bag, a foam bedroll, and a tent. I pack all this, along with a crate of books, a first-aid kit, a camera, and a sack of kibble for my scruffy terrier mutt, Oscar, into the car. Before leaving, I go in for a last checkup with my oncologist.

My road trip will take me 15,000 miles across 33 states. It will last 100 days, the maximum amount of time my medical team has agreed to until my next follow-up appointment. As I turn the keys in the ignition and drive away from New York City, I realize that this is a rite of passage that I hope will bridge the distance between no longer and not yet.

Either my GPS is a liar or I am an erratic driver, but I always seem to take nearly twice as long as it predicts to get to where Im going. Take a right turn inrecalculating its robotic voice says condescendingly when I miss yet another exit. My next destination, Columbus, Ohio, will entail my longest drive yet. The GPS predicts that, if I follow its barrage of orders exactly as told, I will arrive in nine hours and 21 minutes. Unlikely.

Since hitting the road, Im on no ones clock but my own.

Two weeks earlier, when I first left home, I was so tense that I regularly had to remind myself to breathe. Each minute behind the wheel presented new and overwhelming scenarios: Do I have the right of way? What does a blinking red light mean? Was that an Egyptian hieroglyph on the traffic sign? Lane changes and merging onto the freeway had proved especially stressfulan existential guessing game of will I live or will I not. But with each day, I am feeling more confident, and it has been at least 72 hours since another driver has honked at me in anger or bewilderment.

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Researchers curb local immune response in horses receiving stem cell injury therapy – Horsetalk

By daniellenierenberg

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures. Image by carolem41

Treating equine donor stem cells with a growth factor called TGF-2 may allow them to avoid tripping the immune response in recipients, according to new research.

The work carried out at North Carolina State University could simplify the stem cell treatment process for ligament and tendon injuries in horses, and may also have implications for human stem cell therapies.

Mesenchymal stem cell therapy is a promising avenue for treating musculoskeletal injuries, particularly tendon and ligament injuries, in horses.

Mesenchymal stem cells are adult stem cells found in bone marrow that act as repair directors, producing secretions that recruit healing-related paracrine factors to the site of injury.

Just as blood cells have types, depending upon which antigens are on the blood cells surface, mesenchymal stem cells have differing sets of major histocompatibility complex molecules, or MHCs, on their surfaces.

If the MHCs of donor and recipient arent a match, the donors stem cells cause an immune response. In organ transplants, MHCs are carefully matched to prevent rejection.

These treatments arent like a bone marrow transplant or an organ transplant, says Lauren Schnabel, associate professor of equine orthopedic surgery at the university and corresponding author of the study, reported in the journal Frontiers in Cell and Developmental Biology.

Since the mesenchymal stem cells are being used temporarily to treat localized injury, researchers once thought that they didnt need to be matched that they wouldnt cause an immune response. Unfortunately, that isnt the case.

Schnabel and Alix Berglund, a research scholar at the university and lead author of the paper, wanted to find a way to use mesenchymal stem cell therapy without the time, effort and additional cost of donor/recipient matching.

Since these cells dont have to be in the body as long as an organ does, hiding them from the immune system long enough for them to secrete their paracrine factors could be a way around donor/recipient matching, Berglund says. Downregulating expression of the MHC molecules could be one way to do this.

The researchers cultured stem cells and lymphocytes, or T cells, from eight horses, cross-pairing them in vitro so that the stem cells and lymphocytes had differing MHC haplotypes.

In one group, stem cells had been treated with transforming growth factor beta (TGF-2) prior to being added to the lymphocytes in the culture media; the other group was untreated. TGF-2 is a cell-signaling molecule produced by white blood cells that blocks immune responses.

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures.

We use mesenchymal stem cells to treat musculoskeletal injuries particularly tendon injuries in horses very effectively, Schnabel says.

And while you can extract the secretions from the stem cells, you get better results with the cells themselves. Stem cells arent just a reservoir of secretions, theyre a communications hub that tells other cells what they should be doing. So finding a way to utilize these cells without stimulating immune response gives us better treatment options.

This is a promising pilot study, Berglund says. Our next steps will be to further explore the immune response in vivo, and to look at human cells in vitro, as this work has excellent potential to help humans with these injuries as well.

The research was supported by the National Institutes of Health and the Morris Animal Foundation. Research specialist Julie Long and statistician James Robertson, both with the university, also contributed to the work.

TGF-b2 Reduces the Cell-Mediated Immunogenicity of Equine MHC-Mismatched Bone Marrow-Derived Mesenchymal Stem Cells Without Altering Immunomodulatory PropertiesAlix K. Berglund, Julie M. Long, James B. Robertson, Lauren V. SchnabelCell Dev. Biol., 04 February 2021 https://doi.org/10.3389/fcell.2021.628382

The study, published under a Creative Commons License, can be read here.

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Cord Blood Banking: Myths And Facts You Should Be Aware Of, As Per Expert – NDTV Doctor

By daniellenierenberg

Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. Read here to know more.

A baby's umbilical cord contains blood-forming stem cells which can rebuild the immune system

As a parent to be, there are multiple things on your mind to consider and plan before the little one arrives. Between scan appointments and packing hospital bags, you certainly are on the lookout for the best for your baby. Every mother is always anxious to give birth to a healthy baby and assure that it has a healthy future. When you want to give it a healthy future, you need to think about life saving benefits. One of the most important decision which is made at birth for the baby and its family is preserving its cord blood from umbilical cord. The umbilical cord, which connects the baby and the mother in womb has life-saving benefits.

After the birth of the baby, the blood left in the umbilical cord has life-saving cells that can potentially treat over 80 medical conditions pertaining to blood disorders. Cord Blood Banking is the procedure of safely collecting blood from umbilical cord and placenta and preserving it in a sterile environment, thereby ensuring access to stem cells for one's lifetime.

The stem cells which is preserved can be used for blood related disorders. In simple words, stem cells can act like the body's own repair kit and help the body heal from life threatening diseases.

With several advancements, community cord-blood banking is a frontier in medical practice that will help secure not just your baby's future but also your extended family's health. Despite its benefits, there remains many common misconceptions about cord blood banking. Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon debunks the myths and facts about Cord Blood banking.

Cord blood can secure baby's future and also your extended family's healthPhoto Credit: iStock

Also read:Expert Reveals Common Skin Problems In Newborn Babies And How To Take Care Of Them

Fact: Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. The cord blood that is collected is blood that would normally be discarded after birth, so collection doesn't affect your baby's blood supply during pregnancy or delivery.

Fact: Cord Blood extracted from baby's umbilical cord is rich and can be easily available for the family and the child if preserved at birth. Incase of bone marrow or other source of stem cells, the donor has to be registered and should be a match to the family. Cord blood may be accessed more quickly than stem cells from an adult donor who may have registered for donation years ago. The donor must be located, consented, tested and harvested. The extraction of stem cells through Bone Marrow can be painful as well.

Also read:Newborn Care During Winter Season: Here's A Complete Guide For Parents

Fact: According to The Indian Council of Medical Research (ICMR) guidelines commercial banking of Cord Tissue is not permitted to be collected and preserved.

Fact: A baby's umbilical cord contains blood-forming stem cells which be transplanted and rebuild the immune system and bone marrow by saving a patient life with a certain life-threatening condition such as leukemia, lymphoma or thalassemia. Infusion of these cells can also treat patients with inherited genetic disorders, bone marrow failure or inherited immune deficiency.

Fact: It is difficult to find a matching donor of Indian origin of the times hence its important to bank baby's umbilical cord. Incase even if a match is found, it can also be incredibly costly to obtain donor stem cells. The cost to obtain a unit of stem cells can be anywhere between 15-20 lakhs and upwards.

Also read:What Is The Best Time To Give Water To Your Newborn?

(Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

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After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness – Here And Now

By daniellenierenberg

Every year, about 10,000 people in the U.S. need a stem cell transplant but cant find a donor.

The intense medical procedure, which can help those with leukemia, lymphoma, sickle cell anemia and other blood diseases, can save lives but securing a donor can be like finding a needle in a haystack.

Be The Match is a nonprofit, national registry where people can sign up to donate their stem cells. More than 35 million people around the world have volunteered yet only a small percentage of those donors are Americans, and even the registry admits most Americans dont know it exists.

The mother of a 12-year-old boy with leukemia has set out to change that.

Mandy Goldman is a hairdresser who lives with her husband and four children outside Boston. She remembers the devastating day five years ago when doctors told her the chemotherapy they gave her son Mateo Goldman, 9 years old at the time, didnt work.

They told us that our only option of curing Mateo was a bone marrow transplant, she says, a risky procedure that often involves a host of complications. But they had no other choice, she says.

The family got to work on the monumental task finding Mateo Goldman a close enough match.

Linda Matchan first reported the Goldman familys experience for The Boston Globe. In her research, she found very, very few people had any awareness of the need for bone marrow and stem cells donors. The awareness campaign around the subject is severely lacking compared to other campaigns like the importance of donating blood, she says.

For example, there's a little boy right now in North Carolina named Thor Forte, who's 10 and has sickle cell disease. And he has been waiting for literally half his life, five years, for a donor to be available, Matchan says. He's a tough match, but they finally did find somebody. And then when the time came for the procedure, the person backed out. So two years later, the boy is still waiting.

Fortunately, quickly after finding out Mateo Goldman didnt match with anyone in his family, he was paired with a donor on the registry from Germany. Mandy Goldman says Laura Stterlin of Frankfurt was ready to go and donate, ultimately saving her son.

Mateo Goldman wrote Stterlin, whose name he did not know at the time, a thank you note reading: Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family, he says.

And unlike usual Make-A-Wish requests, Mateo Goldman asked to meet Stterlin in person halfway across the world. The trip to Germany was planned for summer of 2020 but has since been canceled due to the pandemic.

In 2019 when she was reporting this story, Matchan had a trip planned to Germany. She ended up meeting Stterlin and hearing the story of how she became a donor. Stterlin said she was at a sporting event with her husband when she got hungry and went on the hunt for some grub.

Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family.

Germany has a robust public service campaign to get citizens to donate bone marrow, Matchan says. So it came to no surprise to Stterlin when she came across a kiosk to sign up.

Just three months later, she got a call and an email from the registry saying that there is somebody in the United States for whom she could be a match and was asked if she would donate, Matchan says. A couple of days later, she went into the hospital and did the donation.

Stterlins stem cells then crossed the Atlantic Ocean, making their way to America during a snowstorm.

The cells started working in Mateo Goldman right away but not without some difficulties, Mandy Goldman says. He battled total body stiffness from graft-versus-host disease, a complication of the transplant.

But, you know, Matteo's an amazing kid, she says, so through it all, he was smiling and making the best of it, even though he was suffering for a lot of the time.

Two years later, in July of 2020, the cancer came back. But since Mateo Goldmans first transplant, the science had evolved greatly.

So much so that his older brother, Leo Goldman, became a candidate to donate his cells for the second stem cell transplant.

I didn't realize how I could get my brother's cells, Mateo Goldman, now 12 years old, says. Once that sank in, I felt that it would connect me and my brother more.

Right before Christmas last year, the family got extraordinary news: Mateo Goldman had zero cancer in his bone marrow, Mandy Goldman says.

Now the mom of four is on a mission to raise awareness on stem cell donations and share the story of how it saved her sons life.

The amazing feeling Leo got from being able to be the person who saved his brother's life is something he's going to carry with him forever, she says. And even Laura [Stterlin], she gave him three and a half years of his life that we get to spend with him. I just really want to educate people about how empowering it is to do something so incredible for somebody else.

When she started talking to others to raise awareness, she was shocked to discover how fearful people were in committing to be a donor.

If people could see the trauma these patients go through her son had a drain placed in his stomach, total body radiation, chemotherapy that left him head-to-toe in a skin-burning rash she says then maybe they wouldnt be scared to dedicate a small action for someone whose only cure is through a stem cell transplant.

Once people are educated about how much of a difference it makes, she says, then I feel like they would do it.

Click here to learn more about the Be The Match Registry.

Tinku Rayproduced and edited this interview for broadcast.Serena McMahonadapted it for the web.

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Leukemia in children: Symptoms, causes, treatment, outlook, and more – Medical News Today

By daniellenierenberg

Leukemia is a type of cancer that affects the blood. The two most common types in children are acute lymphoblastic leukemia and acute myelogenous leukemia.

In a person with leukemia, blood cells are released into the bloodstream before they are fully formed, so there are fewer healthy blood cells in the body.

Below, we describe the types of childhood leukemia, the symptoms, and the treatments. We then look at when to contact a doctor, what questions to ask, and where to find support.

Childhood leukemia is the most common form of cancer in children. It affects up to 3,800 children under the age of 15 in the United States each year.

Leukemia occurs when bone marrow releases new blood cells into the bloodstream before they are fully mature.

These immature blood cells do not function as they should, and eventually, the number of immature cells overtakes the number of healthy ones.

Leukemia can affect red and white blood cells and platelets.

The bone marrow produces stem cells. A blood stem cell can become a myeloid stem cell or a lymphoid stem cell.

Lymphoid stem cells become white blood cells. Myeloid stem cells can become:

Leukemia is typically acute or chronic, and chronic types are rare in children. They can include chronic myeloid leukemia or chronic lymphocytic leukemia.

Most childhood leukemias are acute, meaning that they progress quickly and need treatment as soon as possible.

Acute lymphoblastic leukemia (ALL) is the most common type in children, accounting for 75% of childhood leukemia cases.

It affects cells called lymphocytes, a type of white blood cell.

In a person with ALL, the bone marrow releases a large number of underdeveloped white blood cells called blast cells. As the number of these increases, the number of red blood cells and platelets decreases.

There are two subtypes of ALL: B-cell and T-cell.

In most childhood cases of ALL, the cancer develops in the early forms of B-cells. The other type, T-cell ALL, typically affects older children.

Research from 2020 reports that the majority of people diagnosed with ALL are under 18 and typically between 2 and 10 years old.

The American Cancer Society report that children under 5 years old have the highest risk of developing ALL and that this risk slowly declines until a person reaches their mid-20s.

The outlook for ALL depends on the subtype, the persons age, and factors specific to each person.

Myeloid leukemias account for approximately 20% of childhood leukemia cases, and most myeloid leukemias are acute.

Acute myelogenous leukemia (AML) affects white blood cells other than the lymphocytes. It may also affect red blood cells and platelets.

AML can begin in:

Juvenile myelomonocytic leukemia (JMML) accounts for approximately 12% of leukemia cases in children.

This rare type is neither acute nor chronic. JMML begins in the myeloid cells, and it typically affects children younger than 2 years.

Symptoms can include:

The symptoms of leukemia may be nonspecific similar to those of other common childhood illnesses.

A doctor will ask how long the child has been experiencing the symptoms, which can include:

Children may experience specific symptoms depending on the type of blood cell that the leukemia is affecting.

A low number of red blood cells can cause:

A low number of healthy white blood cells can cause infections or a fever with no other sign of an infection.

A low platelet count can cause:

Various factors can increase a childs risk of leukemia, and most are not preventable.

The following genetic conditions can increase the risk of leukemia:

Also, having a sibling with leukemia may increase the risk of developing it.

These can include exposure to:

If a child has symptoms that might indicate leukemia, a doctor may perform or request:

A bone marrow aspiration involves using a syringe to take a liquid sample of bone marrow cells. The doctor may give the child a drug that allows them to sleep through this test.

During the diagnostic process, a person might ask:

The doctor may recommend a variety of treatments for childhood leukemia, and the best option depends on a range of factors specific to each person.

The treatment usually consists of two phases. The first aims to kill the leukemia cells in the childs bone marrow, and the second aims to prevent the cancer from coming back.

The child may need:

Before or during treatment, a person might ask the doctor:

Questions to ask after the treatment might include:

Children who have undergone leukemia treatments require follow-up care, as the treatments often cause late effects.

These can develop in anyone who has received treatment for cancer, and they may not arise for months or years after the treatment has ended.

Treatments that can cause late effects include:

These complications may affect:

The late effects that may come can also depend on the type of treatment and the form of leukemia.

Because many leukemia symptoms can also indicate other issues, it can be hard to know when to contact a doctor.

Overall, it is best to seek medical advice if a child shows symptoms or behaviors that are not normal for them.

If a child has received a leukemia diagnosis, the effects can extend to parents, other family members, caregivers, and friends.

A person can find support and additional resources from:

The following organizations based in the United Kingdom also provide support and guidance:

Childhood leukemia can affect mental health, as well as physical health.

Learn more about mental health resources here.

According to the American Cancer Society, most children with leukemia have no known risk factors. There is no way to prevent leukemia from developing.

Because there are very few lifestyle-related or environmental causes of childhood leukemia, it is very unlikely that a caregiver can do anything to help prevent the disease.

A childs outlook depends on the type of leukemia. It is important to keep in mind that current estimates do not take into account recent advances in technology and medicine.

For example, the most recent 5-year survival rate estimates reflect the experiences of children who received their diagnoses and treatments more than 5 years ago.

The American Cancer Society report that the 5-year survival rate for children with ALL is 90%. The same rate for children with AML is 6570%.

Childhood leukemia is typically acute, which means that it develops quickly. As a result, a person should contact a doctor if they notice any of the symptoms.

The most common type of childhood leukemia is ALL, representing 3 out of 4 leukemia cases in children.

Treatment may include a combination of chemotherapy, targeted drugs, immunotherapy, stem cell transplants, surgery, and radiation.

The prognosis depends on the type of leukemia and the childs age.

This diagnosis can affect mental as well as physical health, and the effects can extend to caregivers, family members, and friends. Many different resources are available for support.

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Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer – OncLive

By daniellenierenberg

Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 (COVID-19) tend to have favorable outcomes, especially if they are diagnosed in complete remission (CR) and further out from their cell infusion, according to Miguel-Angel Perales, MD, underscoring that care should not be delayed despite the ongoing pandemic.

Delayed therapy results in patients with relapse or progression of disease who did not receive the intended cellular therapy; [weve seen this happen] in 34% of cases, Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC), said during a presentation delivered at the 2021 AACR Virtual Meeting on COVID-19 and Cancer.1 Given that we can avoid the risk of nosocomial transmission, I think this clearly indicates that we should be careful about how we manage these patients and not try to delay their care.

In his talk, Perales highlighted registry data detailing the impact of the pandemic on cellular treatment in patients with cancer, outcomes of patients who were infected with the virus and received hematopoietic cell transplantation, and the impact of virus-related delays in care.

Data reported to the ASH Research Collaborative COVID-19 Registry for Hematology, a global reference tool available to the public, showed that as of January 15, 2021, a total of 813 malignant and non-malignant cases of COVID-19 were reported, with just over 500 cases reported in the United States alone.2

When looking at cellular therapies received prior to a diagnosis with the virus, 10 patients had received CAR T-cell therapies (6 recovered, 4 died), 46 patients had undergone allogeneic stem cell transplantation (34 recovered, 7 died, 5 had unknown outcome), and the majority, or 78 patients, had undergone autologous stem cell transplantation (67 recovered, 7 died, 4 had unknown outcome).

An earlier analysis of data collected from this registry showed that among the first 250 patients for whom data were collected, the overall mortality rate was 28% (95% CI, 23%-34%).3 However, in patients with moderate to severe COVID-19 infection, the mortality rate was even higher, at 42% (95% CI, 34%-50%). This is a condition that has significantly impacted our patients with hematologic malignancies, noted Perales.

Another registry, of the Center for International Blood & Marrow Transplant Research (CIBMTR), requires the inclusion of outcomes of patients who have undergone transplantation or received CAR T cells.4 As of January 15, 2021, data for 1258 patients from 195 centers were reported to the registry and showed that 50.08% of patients had undergone allogeneic transplantation and 44.66% had undergone autologous transplantation. Only a small percentage of patients received cell therapy, according to Perales.

The age of patients at the time of infection ranged from less than 20 years to older than 70 years, with the majority of patients between the ages of 60 years and 69 years. When looking at infections by region, 29.35% of cases were reported in the Midwest, 23.44% were reported in the Northeast, and 22.73% were reported in the South. The majority of cases occurred within the first 2 years of their infusion. A total of 614 casesalmost half of all patientshad their infection resolve, while 58 experienced improvement; 187 patients had died.

In a subsequent paper, investigators examined risk factors associated with death from COVID-19 in recipients of allogeneic transplantation based on data from the CIBTR registry.5 Results from the multivariate analysis showed that age greater than 50 years (P = .016), male gender (P = .006), and COVID-19 infection in less than 12 months following transplantation (P = .019) were all significantly associated with increased risk of death.

Interestingly, race and ethnicity were not significant in this series, noted Perales. Similarly, when we look at patients [who have undergone] autologous transplant, the only factor that we saw was the diagnosis of lymphoma versus myeloma. Other factors were not significant.

In another analysis, investigators examined outcomes of patients following transplant who were infected with the virus at MSKCC. Of the first 77 patients diagnosed between March 15, 2020 and May 7, 2020, 37 had undergone autologous transplant, 35 had undergone allogeneic transplant, and 5 had received CAR T-cell therapy.6

The disease distribution was as expected, according to Perales. Thirty-eight percent of patients had plasma cell disease, 23% had acute leukemia, 23% had aggressive non-Hodgkin lymphoma (NHL), 5% had Hodgkin lymphoma, 4% had chronic myeloid leukemia, 4% had myelodysplastic syndrome, and 3% had indolent NHL.

When you look at day [of infection] post infusion, you see there was a significant range, said Perales. In fact, the number of patients were diagnosed with COVID-19 several months or even years after their cell therapy. These are the demographics of 77 patients, but this is representative of the patients that we transplant at our center.

Notably, 44% of patients did not have any comorbidities. Investigators also examined the home medications that patients were receiving at the time of their COVID-19 diagnosis. Here, 10 patients were receiving steroids, 18 were receiving immunomodulatory agents, 4 were receiving anticoagulation agents, and 14 were receiving immunosuppressive drugs.

Almost half, or 48%, of patients had mild COVID-19 infection, so they were not admitted to the hospital. Twenty-six percent of patients had moderate infection, and thus, were admitted to the hospital, while 22% had severe infection and were either admitted to the intensive care unit or died.

In that group, the majority of them actually had active malignancy, unlike the other 2 groups where the majority actually were in remission, said Perales. Patients who required high levels of oxygen [were often those who] had active malignancy.

Results from a univariate analysis looking at the predictors of disease severity revealed significant associations between the presence of comorbidities and infiltrates on imaging at the time of diagnosis. Overall, however, we were able to see favorable outcomes with patients after COVID-19 infection, said Perales. Two-thirds of patients actually had a resolution. We did see 14 deaths, which represented 18% of patients. This was 41% of patients who were admitted, but particularly those with an active malignancy.

Among patients who were admitted to the hospital but had a malignancy that was in remission, the mortality rate was 21%. This was due, in part, to the fact that in many cases, patients or their family members decided to forego aggressive medical care.

Additional data revealed that COVID-19 was linked with a drop in lymphocyte populations across the board, added Perales. Notably, lymphopenia with COVID-19 was not found to impair long-term immune reconstitution in patients who had undergone bone marrow transplant.

When looking at survival in patients after infection with COVID-19, overall outcomes were found to be favorable.

Investigators also examined the risk of nosocomial infections in patients who had undergone transplantation or received cellular treatment in light of the pandemic. They looked at a series of 44 cases.

In March 2020, 2 healthcare workers were exposed at MSKCC and 3 patients had documented COVID-19 infection. One patient was receiving treatment in the inpatient setting, but the patient did have frequent visits from family members, according to Perales. So, its unclear when or how the exposure occurred, Perales said. The patient ended up dying.

Two additional patients may have been exposed in the donor room while they were collecting the stem cell from the autologous transplant, added Perales. One patient eventually died from the virus.

Again, its unclear whether these patients were infected in the center or in the community, as COVID-19 was very prevalent at the time, said Perales. Importantly, we have not seen any additional cases of potential or definite COVID-19 nosocomial infection since March 2020 at our center.

When examining the impact of the pandemic on treatment delays, in March 2020, investigators started to prospectively collect data from patients whose transplant or cellular therapy was delayed as a result of the impact of the virus on resources at the hospital, particularly the capability of using intensive care unit beds.1

Results showed that 85 patients delayed treatment; of those patients, 29 have not received their intended cellular treatment. Sixteen were supposed to receive autologous transplant, 12 were supposed to undergo allogeneic transplant, and 1 was supposed to receive CAR T-cell therapy.

Of the 56 patients who eventually proceeded to treatment, 62% received autologous transplant, 67% received allogeneic transplant, and 86% received CAR T-cell therapy. The biggest reason for not proceeding to treatment with autologous transplant and CAR T-cell therapy was because they were deferred due to good disease control. Other reasons included was because of a new comorbidity (12%) or they died from the virus. The most prominent reason for not proceeding to allogeneic transplant during the pandemic was progression of disease (42%).

We conclude that patients who are recipients of allogeneic transplant, and particularly those with acute leukemia, as much as possible should proceed to their indicated therapy and not be delayed, concluded Perales.

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Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer - OncLive

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh – News18

By daniellenierenberg

Image for representation purpose only.

A tech entrepreneur, who is determined to live for 180 years, claims his bizarre strategies will soon be as popular as cell phones. Dave Asprey firmly believes one of the keys to living longer is skipping breakfast. The American lifestyle guru has asserted that he can at least live to the year 2153 by using a variety of techniques, including a cold cryotherapy chamber sitting and intermittent-fasting. The 47-year-old millionaire devised the term 'Biohacking' to detail his methods of turning back the biological clock. In an effort to survive for as long, Dave has spent over $1,000,000 on hacks and techniques to improve the overall functioning of his body. He has parts of his bone marrow removed to re-inject his own stem cells back into his body.

Appearing on ITVs This Morning today, he joined host Holly Willoughby for a virtual conversation. Holly quizzed Dave why he wants to live so long, to which he replied that as a curious person, he feels there is a lot in the world that can be fixed and improved that he thinks he has not done yet. Dave confirmed that some of the things he wants to pioneer are expensive. However, there are other ways that are free of cost like fasting.

Dave also speculated that after applying his methods for longer life, people under 40 will be 'happy and highly functional' over a 100-years-old. He stated that he won't be the only one as his wife both is also racing to get to 180 years old. In comparison to others, Dave has set himself up for much less inflammation by controlling what he eats and how he sleeps, besides several other anti-ageing treatments. Explaining the benefits of reintroducing his own stem cells in his body, he said people heal when they are young. With age, the stem cells of the body get exhausted, so he opts for ways which gives him more stem cells.

Dave also follows cryotherapy, which is also known as cold therapy. It involves using low temperatures to treat a variety of tissue lesions. He is also taking cold showers for more than ten years. Dave uses another technique to live a long life known as intermittent fasting. This involves controlling the number of times that one eats meals to create periods of fasting over a certain period. According to Dave, it helps in disease prevention as fasting periods lets his body to 'repair itself' while not digesting food.

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh - News18

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Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and…

By daniellenierenberg

The reduced osteogenic differentiation of bone marrow mesenchymal stem cells (BMSCs) is the typical characteristics of pediatric aplastic anemia (AA) pathogenesis. Long non-coding RNA MEG3 is reported to promote osteogenic differentiation of BMSCs via inducing BMP4 expression.This study aims to investigate the mechanism of DNMT1/MEG3/BMP4 pathway in osteogenic differentiation of BMSCs in pediatric AA.BMSCs were isolated and purified from bone marrows of pediatric AA patients (n=5) and non-AA patients (n=5). The expression of DNMT1, MEG3, and BMP4 in isolated BMSCs was detected using quantitative real-time PCR and western blot analysis. Osteogenic differentiation was determined using Alizarin red staining. The methylation of MEG3 promoter and the interaction between DNMT1 and MEG3 promoter were detected using methylation-specific PCR and chromatin immunoprecipitation assay, respectively.Lowly expressed MEG3 and BMP4 and highly expressed DNMT1 were observed in BMSCs of pediatric AA patients. The overexpression of MEG3 promoted osteogenic differentiation of BMSCs. Luciferase reporter assay showed that MEG3 overexpression increased transcriptional activity of BMP4. The inhibitor of methylation, 5-azacytidine, suppressed DNMT1 expression and reduced methylation of MEG3 promoter. Overexpression of DNMT1 increased the binding between DNMT1 and MEG3 promoter. The simultaneous overexpression of DNMT1 and MEG3 restored the inhibition of osteogenic differentiation caused by DNMT1 overexpression alone.Our findings indicated that DNMT1 mediated the hypermethylation of MEG3 promoter in BMSCs, and DNMT1/MEG3/BMP4 pathway modulated osteogenic differentiation of BMSCs in pediatric AA.

PubMed

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Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and...

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Family ‘living worst nightmare’ as they desperately seek donor for tot with rare blood cancer – Teesside Live

By daniellenierenberg

The mum of a Stockton toddler says time is running out to find a match for her son who is battling a rare blood cancer.

Little Mason Arrowsmith, one, was diagnosed with Juvenile Myelomonocytic Leukaemia (JMML) on Christmas Eve last year.

The condition is a rare type of slowly developing blood cancer that occurs in young children.

Although it was discovered at an early stage, doctors say Mason needs a stem cell or bone marrow transplant for the best chance of survival.

In a desperate attempted to help, his mum Katie Jordan will donate her own bone marrow later this month, but a full match is urgently needed.

"The hospital has been absolutely amazing, but unfortunately after searching worldwide there is no match for Mason due to his bone marrow being so rare," Katie said.

"We need to get him to transplant as soon as, the hospital is looking at the end of February, using me as his first donor in the hope that this will help him until we find a better match.

"I would give my life for Mason but unfortunately, I can only donate my bone marrow three times in a lifetime and Im not a full match for him.

"Children with JMML live around 12 months after diagnosis, we need to find mason a better match. Twelve months is not long enough for us to have with our boy, we are living the worst possible nightmare."

As Mason does not have a close enough match within his family - the only potential cure is through a stem cell transplant from an unrelated donor.

The family has now launched a 'Masons Mission' to encourage people to support blood cancer charity Anthony Nolan in raising urgent funds to add more donors to the stem cells register.

There is currently a blacklog of around 25,000 potential donors due to the impact of the coronavirus pandemic and the charity needs to raise up to an extra 500,000 to add people to the register, from ordering more swab packs to analysing completed swabs in its laboratory.

Any one of the 25,000 people who have applied to join the Anthony Nolan could be a match for Mason or one of the 2,300 patients in the UK, who need a stem cell transplant from a donor each year.

Katie added: "We have set our target at 10,000 which does seem a lot but this would help to cover 250 registrations and kits and allow us to continue our search for my baby."

Henny Braund, Chief Executive of Anthony Nolan said: "Were doing all we can to find a stem cell donor to give Mason a second chance of life.

"A perfect storm of the coronavirus pandemic, and a surge of 40,000 incredible people who have been inspired to join the Anthony Nolan register in the last month by patients, like Mason means that were in urgent need.

"The best thing people can do is support Anthony Nolans work financially. By giving anything, together we can add all potential lifesavers to the register, and give patients like Mason hope."

Anthony Nolan recruits people aged 16-30 to the stem cell register as research has shown younger people are more likely to be chosen to donate.

They also carry out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

It costs 40 to recruit each potential donor to the register, so Anthony Nolan relies on financial support.

You can support Masons Mission here.

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Stem cells efficacy confirmed in treating ototoxic hearing loss – Korea Biomedical Review

By daniellenierenberg

Researchers at the Catholic University of Korea St. Marys Hospital have recently proved the efficacy of bone marrow-derived stem cells to treat ototoxicity hearing loss, the hospital said Thursday.

The team, led by Professor Park Kyoung-ho of the Department of Otolaryngology, conducted an experiment on animal models with ototoxic sensorineural hearing, or sudden hearing loss.

They utilized Catholic MASTER cells, bone marrow stem cells developed by the Catholic Institute of Cell Therapy, to compare the stem cell injection group with the controlled group.

The result showed that animals started to recover their hearing after three weeks. Five weeks later, they recovered normal hearing at 8000Hz, 16000Hz and 32000Hz frequency.

Ototoxic hearing loss is caused when a person ingests chemicals or certain medications that adversely affect the inner ear functions. Major symptoms related to the illness are dizziness, false hearing, and hearing loss, which permanently defects hearing functions. Elders with such symptoms should have medical consultations as they are a high-risk group, the hospital said.

We have proved the efficacy of our bone marrow stem cells in recovering hearing, said Professor Park, who doubles as the director of the Stem Cell Institute. Through the results, we expect to provide new treatment opportunities for patients with hearing loss.

The test results were published in the Korean Journal of Otorhinolaryngology-Head and Neck Surgery.

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Understanding bone marrow transplant: The guidelines and the protocols – The New Indian Express

By daniellenierenberg

The outbreak of the Covidpandemic has made many patients reluctantto undergotreatments. While their apprehension seems to overpower them, doctors need to ensure thatstrict guidelines and protocols which assure the best quality service are followed.

Among elective surgeries andtransplants, bone marrow transplant cases have increased substantially in the past few months. Adhering to guidelines for pre-transplant evaluation and the management of a common complication, graft versus host disease (GVHD)is essential.

With the diversity of practice and expertise, the following guidelines will provide a pivotal tool for learning about the rapidly updated therapy landscape in Hematopoietic stem cell transplantation (HSCT).

The guidelines intended to provide a systematic approach for transplantation and help streamline clinical practices and educate new generations of physicians-in-training. Additionally, guidelines can help to evaluate a potential transplant recipient anddetermine if the patient is an eligible candidate for the procedure.

Types and selection of transplantation:

Selection of the type of transplantation for a patient depends on factors such as the type of malignancy, availability of a suitable donor, age of the recipient, the ability to collect a tumor-free autograft, the stage, the malignancy's susceptibility to the GVM effect, and status of disease -- bone marrow involvement, the bulk of disease, chemosensitivity to conventional chemotherapy. This method is particularly applicable for Autologous or Allogeneic Transplantation where one can have a sibling donor or a matched unrelated donor. In the case of a matched unrelated donor, ensure that the collection is adequate and stem cells are available well in time especially if they are imported from countries in Europe.

A haploidentical transplant is another type of transplant that uses healthy, blood-forming cells from a half-matched donor to replace the unhealthy ones. The ideal donor in this case is a family member.

That said, for bone marrow transplant blood products are the backbone and it is important to ensure to have adequate supply before you begin with the transplant.

What are the guidelines and protocols that can be adopted in current times?

Some measures for consideration are: Minimize face-to-face visits including monitoring and consider shifting to telehealth where feasible. Some adaptive community measures like the hospital in the home services, community practices for blood collection, imaging, and support services. For radiation oncology treatment, consider reducing fractions when supported by evidence Consider alternative and less resource-intensive treatment regimes. Minimize unnecessary visitors to cancer centers, for instance, limiting to only patients and their essential caregivers based on frailty and language needs Screen for possible symptoms of COVID-19 and triage patients for admission. If necessary, the admission has to be directed to oncology/hematology departments rather than emergency departments. Immunocompromised patients are likely to have atypical presentations of COVID-19 For suspected checkpoint inhibitor-related pneumonitis prioritizes COVID-19 testing for an early decision regarding corticosteroid therapy.

These are some guidelines that you should heed during a bone marrow transplant. While it is imperative to be updated about the guidelines, timely intervention can reduce the other possible complications during the process.

(The author is the Director, Medical Oncology and Hemato Oncology, atFortis Cancer Institute, Bangalore)

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Man who wants to live for 180 years spends Rs 18 lakh to re-inject his own stem cells – Times Now

By daniellenierenberg

Dave Asprey  |  Photo Credit: Twitter

A man who is determined to live until he is 180 years old says that his bizarre methods will soon be as popular as mobile phones. American millionaire tech entrepreneur Dave Asprey, 47, believes he will live to the year 2153 'at least' by using techniques such as sitting in a cold cryotherapy chamber and intermittent-fasting.

Dave coined the term 'Biohacking' to describe his methods of turning back the biological clock.

He got parts of his bone marrow removed to have the stem cells injected back into his body for $25,000 (Rs 18 lakh).

He speculated that people who are under 40 years of age will be "happy and highly functional" at 100 after applying his methods.

When This Morning's Holly Willoughby asked him why he wants to live so long, he replied, "I'm curious, I think there's a lot of things we can fix and improve in the world and I don't feel like I'm at all done yet."

Dave believes that he won't be the only one to be live for so long.

"The things I am working to pioneer, some of them are expensive, some of them are free like fasting. This will be like cell phones, everyone has cell phones - everyone will have anti-ageing. Change can happen rapidly in society," he said. "There will be many people who are under 40 right now who [will be] walking around under their own power, perfectly happy, highly functional, who are more than 100 years old."

Dave has spent an estimated $1 million on techniques and hacks to try and improve his body's overall functioning.

He added, "I set myself up to have much less inflammation than most people do, by controlling what I eat and how I sleep and a lot of other anti-ageing treatments."

He explained why he re-introduced his stem cells in his body, saying, "When we're young, we have a ton of stem cells and we heal like young people. As we age our stem cells get exhausted, so I do things like intermittent fasting which give me more stem cells and then I take my own stem cells and move them around the body so I heal and move like a young person."

Dave also believes in the benefits of cryotherapy, also known as cold therapy, which is the use of low temperatures in medical therapy to treat a variety of tissue lesions. He has been having cold showers for over ten years.

Another technique that Dave uses to live a long life is intermittent fasting, which involves restricting times that you eat meals to create periods of fasting over a certain period.

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World Cancer Day 2021: DKMS Announces The Milestone Of Giving 90,000 Blood Cancer Patients Worldwide A Second Chance At Life – PR Newswire India

By daniellenierenberg

- DKMS-BMST continues to urge Indians to step up to be a potential lifesaver!

BENGALURU, India, Feb. 4, 2021 /PRNewswire/ -- February 4th is marked as World Cancer day every year. This day is observed to spread awareness about the disease and its increasing burden. In line with the theme for this year "Create a futurewithout cancer. The time to act isnow", DKMS BMST Foundation India, a non-profit organization with a mission to provide a second chance at life to blood cancer and blood disorder patients in India makes an appeal to people to come forward and register as potential blood stem cell donors.

On World Cancer Day 2021, DKMS is also celebrating a milestone of providing more than 90,000 blood cancer patients across 57 countries with a second chance at life, since it was founded almost 30 years ago in 1991. DKMS is an international non-profit organization that helps provide patients with lifesaving blood stem cell transplants. DKMS has presence in India, Germany, USA, Poland, UK, Chile, and South Africa.

Patrick Paul, CEO, DKMS BMST Foundation India, says, "DKMS is proud to be the world's leading donor center, accounting for nearly 30% of the total donor pool. While, this is a global milestone, when it comes to India, the fact is that the Indian donors are highly underrepresented in the global database. This is why it becomes difficult for doctors to find a matching blood stem cell donor for Indian patients. While DKMS has registered over 10.5 million donors and has provided over 90,000 patients with a second chance at life globally, it is critical to highlight that only over 43,000 Indian donors are part of this donor pool."

In India, every year, over one lakh people are diagnosed with a form of blood cancer and it remains one of the leading causes of cancer-related deaths among children. Most people are unaware that a life-threatening disease like blood cancer can be treated and in most of the cases, a stem cell transplant is the patient's only chance for survival. For instance, 15-year-old Maheer from Gujarat, India, is one of the blood cancer survivors who had received a lifesaving blood stem cell donation in 2012. He was able to find his matching blood stem cell donor, Dr. Sita, who hails from Germany. Today, he leads a normal, healthy and happy life. He is in grade 9 and loves to travel, read and swim.

Today, more than 37 million potential unrelated donors are listed worldwide with stem cell donor centers and registries, of which only 0.03% are Indians. Currently, in India, the biggest challenge is the lack of awareness about blood stem cell transplant and the importance of registering as a potential blood stem cell donor. The entire procedure is safe and secure. Once the blood stem cells are collected from a donor, they are infused into the patient through a transplant process which then moves through the bloodstream and settles in the bone marrow. These new blood stem cells begin to increase in numbers and produce red blood cells, white blood cells, and platelets, resulting in the replacement of the patient's diseased cells and that's how a blood cancer patient gets a second chance at life. This situation can only be improved by recruiting many more potential stem cell donors from India.

This World Cancer Day, one can take a pledge to become a potential lifesaver. Registration takes only 5 minutes. If one between 18 and 50 years and in good health, the first step to register as a blood stem cell donor by ordering the home swab kit at http://www.dkms-bmst.org/register.

SOURCE DKMS BMST Foundation India

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Hematopoietic Stem Cell Transplantation (HSCT) Market Incredible Possibilities, Growth With Industry Study, Detailed Analysis And Forecast To 2027 …

By daniellenierenberg

Up Market Research (UMR) recently published a report entitled, the Hematopoietic Stem Cell Transplantation (HSCT) Market, describing the crucial aspects of the market by conducting an in-depth analysis of the current trend, emerging threats, and future market assessment. This report takes into account the adverse impact of the COVID-19 pandemic on the market for the period of 2020-2020 and provides a detailed information about how the market will perform during the forecast period, 2020-2027. Our research team presents the report in a simplistic manner supported by fact and actual figures that will assist clients to arrive an informed decision about their investment plans and business strategies.

Request Free Exclusive Sample on Hematopoietic Stem Cell Transplantation (HSCT) Market Report @ https://www.upmarketresearch.com/home/requested_sample/30593

The report provides a holistic view of the market offering key insights of the market behavior over last four years and key assessment of the future market performance. It includes a systematic analysis of historical data for the period, 2015-2020 and draws upon assessment of the market performance for the forecast period, 2020-2027 by observing 2020 as the base year. With the reference to the available data, it provides vital insights on key factors such as drivers, restrains, trends, challenges, and opportunities of the Hematopoietic Stem Cell Transplantation (HSCT) market growth.

This report provides a comprehensive outlook on the key segments and sub-segmentations that includes the complete information about the product types, applications, end-users and regions. It offers latest information regarding the growth rate, volume, and size of the market in respect to each segment and also explains the market performance of these segments in the respective five regions. Moreover, it discusses a wide range of the emerging market scope and potential drawbacks present in the segments.

Hematopoietic Stem Cell Transplantation (HSCT) Market Report Includes:

The Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report is segmented into:

By Types:

AllogeneicAutologous

By Applications:

Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

By Regions:

For More Information on This Report Visit: https://www.upmarketresearch.com/home/enquiry_before_buying/30593

The report covers the competitive landscape of various major global players, their current market positions, and key business strategies adopted to mark their major footprint in the market. This includes information about the product launch, expansion of the production facilities or plants, adoption of new technologies, latest merger & acquisition, partnership, and collaboration of the key players. It furthers provides concrete information about the existing market scope for the new entrants and the current competitive levels and scenario for the emerging players in the global market.

The Hematopoietic Stem Cell Transplantation (HSCT) Market Report Covers the Following Companies:

Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord I

Up Market Research (UMR) also offers customized report for a particular product, application, and region as per the requirement of clients and provides additional companies profiles as per the clients request.

Regarding the methodology, the report is prepared by relying on primary and secondary sources including interviews of the company executives & representatives and accessing official documents, websites, and press release of the companies related to the Hematopoietic Stem Cell Transplantation (HSCT) market. It also includes comments and suggestions from the experts in the market especially the representatives from government and public organizations as well as international NGOs. The report prepared by Up Market Research (UMR) is known for its data accuracy and precise style, which relies on genuine information and reliable data source. Moreover, customized report can be available as per the clients wishes or specific needs. It takes into account of various research tools and methods including predictive analysis, Porters 5 force analysis, SWOT analysis, and real-time analytics.

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This report includes the estimation of market size in terms of value (USD) and volume (K MT), with applying top-down and bottom-up approaches to estimate and validate the overall scope of the market. The report is presented with a group of graphical representations, tables, and figures that shows a clear picture of the developments of the products and its market performance over the last few years. With this precise report, it can be easily understood the growth potential, revenue growth, product range, and pricing factors related to the Hematopoietic Stem Cell Transplantation (HSCT) market. This report includes information on the latest government policies, norms, and regulations that can affect the dynamics of the market.

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Hematopoietic Stem Cell Transplantation (HSCT) Market Incredible Possibilities, Growth With Industry Study, Detailed Analysis And Forecast To 2027 ...

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Novel Bone Marrow ‘Ingredient’ To Help Arthritic Horses The Horse – TheHorse.com

By daniellenierenberg

Regenerative therapies such as stem cells and platelet-rich plasma already play an important role in managing osteoarthritis (OA). Nonetheless, veterinarians have found that response to even these therapies is less than ideal in many cases, prompting researchers to continuously seek novel therapies for this all-too-common musculoskeletal disorder. One of the newest to be unveiled is called bone marrow mononuclear cell (BMNC) therapy. One researcher who presented at the 2020 American Association of Equine Practitioners Convention, held virtually, reported that the equine industry is in critical need for therapies that resolve joint inflammation but preserve tissue healing, and BMNC appears a promising candidate.

Much more than stem cells classically sought for cartilage healing, bone marrow is rich in macrophage progenitor cells, explained James B. Everett, DVM, MS, previously of the Virginia-Maryland College of Veterinary Medicine, who now works at the Equine Surgical Center at ThorSport Farm,in Murfreesboro, Tennessee. Macrophages are a type of white blood cell that play a role in tissue repair and cartilage integrity, and produce the anti-inflammatory mediators, including interleukin-10 (IL-10).

Everett said macrophages in the synovial (joint) membrane are essential for joint health, clearing aggressors, secreting key molecules required for optimal joint function, and forming a shield that protects tissues undergoing repair, similar to a wound scab. However, when the amount of tissue damage overwhelms these housekeeping functions, macrophages stimulate inflammation as a means of recruiting more cells, especially more macrophages, to cope with increased demands for repair.

If this response is efficiently accomplished, macrophages then produce, among other things, high concentrations of IL-10 and resolve the inflammatory process, returning the joint to a healthy state, he said.

Everett emphasized that not all inflammation is bad. This acute inflammation is essential to establish a resolving response, and anti-inflammatory therapies can negatively interfere.

As presented by Everetts colleague Bruno Menarim, DVM, PhD, in a separate session, studies show that BMNCs promote the endogenous resolution of experimentally induced inflammation. To see if these promising features translated to naturally occurring inflammation in live horses, Everetts research team studied 19 horses, dividing them into three treatment groups:

The selected horses were diagnosed with OA in a single joint, and the team injected those joints once with the saline, triamcinolone, or BMNCs. The BMNCs were autologous, meaning veterinarians collected them from each patients own bone marrow aspirate. They processed the aspirate in-house, and the isolated mononuclear cells, composed predominantly of macrophages, were ready to inject into the affected joint within three hours of aspiration.

We found that while objectively assessed lameness (via Lameness Locator) decreased in all three groups, it was only significant in the BMNC-treated horses, said Everett. Further, the treatment was well-tolerated with no adverse events appreciated in this study.

He said that using BMNCs can help reduce the need for chronic use of non-steroidal anti-inflammatory drugs and corticosteroids, which produces potentially harmful consequences. Further, BMNCs preserve the production of molecules such as interleukins and cytokines that are essential for restoring joint homeostasis. Corticosteroids often inhibit these molecules.

The researchers noted that these results support a larger clinical trial using BMNCs in clinical cases of equine OA.

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‘Whatever it takes’: Stem cell drive underway to find bone marrow match for girl on Alta. First Nation – CTV Edmonton

By daniellenierenberg

EDMONTON -- A little girl with leukemia in northern Alberta is in desperate need of a bone marrow transplant.

Friends and family of 10-year-old Ameilia Powder have set up a stem cell drive to find a match.

"Asking for help is probably one of the most difficult things to do when you're in this situation and really opening your story up to everyone is really hard. but at the end of the day Ameilia needs a bone marrow match," Ameilia's grandmother Jaime Harpe said. "I'll do whatever it takes to get her that match."

Ameilia was diagnosed in March 2020 and went through five months of treatment at the Stollery Children's Hospital before returning home to Fort McKay First Nation in August.

The cancer returned late last month and this time, a bone marrow transplant is the only option to save her life.

"All people have to do is go blood.ca/stemcells and they can register online, and a kit gets sent to you in the mail," organizer Amanda Main said. "You just swab your cheek, pop it back in and you get entered in the data base, that's all you have to do."

Potential matches need to be between the ages of 17 and 35.

A GoFundMe page has already raised more than $11,000.

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