Category Archives: Stem Cell Therapy

Stem cells are key building blocks for the human body. At the start of life, they divide over and over again to create a fully developed baby from an embryo. Many individuals now even turn to services that store and preserve umbilical cords should a person ever be in need.

Stem cells have the potential to develop into different types of cells in the body, serving as a repair system of sorts for damaged or lost cells. In recent decades, scientists have shown the miraculous ways of medicine through stem cell treatments.

So just how are doctors using stem cells to treat and help heal people battling various ailments? Heres a look at five studies published on StudyFinds that demonstrate the wondrous ways of stem cell treatments.

A heart condition called dilated cardiomyopathy, or DCM, weakens muscles of the ventricles, which causes heart failure and often death in children. Currently, the only cure is a heart transplant, which can take long periods of time to find an acceptable donor and increases the risk of rejection of the donor tissue. One study finds that stem cell therapy could help DCM patients survive longer while awaiting a transplant or potentially eliminate the need for a new heart entirely.

Cardiac stem cells called cardiosphere-derived cells (CDCs) have proven to be effective at treating certain heart conditions. The CDCs grow into tissue cells of the heart and can counter the effects of DCM. To test the safety of the CDC therapy, a team of scientists at Okayama University in Japan demonstrated the efficacy of CDCs in tissue damaged from DCM. For the study, DCM symptoms were induced in pigs, after which CDCs were administered in various doses for treatment. In a control group, some pigs were given a placebo.

Results showed thickening of the heart muscle in pigs who were given the stem cell treatment. This allows increased blood flowto the rest of the body, thereby effectively repairing the damaged tissue. Due to the dosage used in animal trials, researchers could estimate the proper dosage for human trials.

The first of these included 5 younger patients who were diagnosed with DCM. Injections of CDCs resulted inbetter heart function without any serious side effects. Thus, scientists believe this type of treatment could minimize the need for heart transplants and allow DCM patients to have normal lives.

READ MORE: Stem cell treatment shows promise as treatment for rare heart condition in children

Although their use is sometimes controversial, scientists often look at stem cells as a potential miracle cure for many conditions. One study finds stem cells from a babys umbilical cord may save the most at risk of dying from COVID-19. A treatment derived from non-altered versions of these stem cells significantly improves the survival rate among coronavirus patients already on a ventilator.

In a double-blind, controlled, randomized study, 40 adultpatients in intensive careand on a ventilator received the treatment intravenously. The infusions contained stem cells coming from the connective tissue of a human umbilical cord. Half of the patients received infusions not containing stem cells to serve as a control group.

Results reveal survival rates climbed by 2.5 times among patients receiving stem cells. Those with a pre-existing health problem, making them high-risk for COVID, saw their changes of beating coronavirus jump by 4.5 times. Moreover, the study says the stem cell infusions did not cause any life-threatening complications or allergic reactions.

READ MORE: Stem cells from a babys umbilical cord doubles survival chances among COVID patients

In the fight against heart disease, a new super-weapon is now even closer to deployment, and its capabilities are turning out to be beyond expectations. A study aimed at combating heart disease finds that stem cells are not only showing promise in treating heart failure, but in rats are actually reversing problems associated with old age.

The specific type of stem cells used in the study are cardiosphere-derived cells, or CDCs. While the latest research involving CDCs indicates possibilities that have previously been in the realm of science fiction, the scientists leading the charge urge restraint in face of the excitement.

Nevertheless, the latest results of stem cell infusions in rats are startling. Not only did rats that received the CDCs experience improved heart function, they also had lengthened heart cell telomeres. Moreover, the rats that received the treatment also had their exercise capacity increase by about 20 percent. They also regrew hair faster than rats that didnt receive the cells.

Still, the doctors and scientists working to push the frontier of medicine forward are very optimistic about the real possibilities of the therapy. Researchers of the study said they are also studying the use of stem cells in treating patients with Duchenne muscular dystrophy and patients with heart failure with preserved ejection fraction, a condition that affects more than 50 percent of all heart failure patients.

READ MORE: Study: Cardiac stem cell injections reverse effects of aging

A new biomaterial can help regenerate tissue in people dealing with chronic lower back pain and spinal issues. A recent study finds the secret to this breakthrough therapy is all in the hiPS. Not thosehips, but human induced pluripotent stem cells.

The study explains that a common cause of lower back pain is the degeneration of intervertebral discs (IVDs). These discs sit between the vertebrae in the spine and help give the spinal column its flexibility. Severe IVD degeneration eventually leads to spinal deformity without treatment. In this study, scientists used cartilage tissue derived from stem cells to build back lost IVDs in lab rats.

Study authors used induced pluripotent stem cells (iPSCs) during their experiments. Importantly, scientists are capable of turning iPSCs into chondrocytes cells that produce and maintain cartilage. Previous studies have successfully used this same method to treat cartilage defects in animals. In the new study, researchers created human iPSC-derived cartilaginous tissue (hiPS-Cart) that they implanted into rats with no NP cells in their intervertebral discs.

Findings reveal that the hiPS-Cart implanted in the rats was able to survive and be maintained. IVD and vertebral bone degeneration were prevented. The researchers also assessed the mechanics and found that hiPS-Cart was able to revert these properties to similar levels observed in the control rats.

READ MORE: Stem cell cure for lower back pain is all in the hiPS

Stem cells taken from deceased patients may also help in creating a cure for blindness. Retina cells from a corpse continue to survive after being transplanted into the eyes of monkeys, scientists say.

RPE dysfunction is a leading cause of blindness, including causing disorders likemacular degeneration, which affects around 200 million people worldwide. Now, for the first time, scientists have successfully produced retina cells in monkeys using human stem cells. Human cadaver donor-derived cells can be safely transplanted underneath the retina and replace host function, and therefore may be a promising source for rescuing visionin patients with retina diseases.

For the study, researchers transplanted stem cells from the eyes of donated bodies under the monkeys macula, the central part of the retina. Following surgery, the transplanted patches remained stable for at least three months without any serious side-effects. The RPE created by the human stem cells partially took over from the old retina cells. In addition, this could successfully support the eyes light receptorswithout causing retinal scarring.

These unique cells could serve as an unlimited resource of human RPE, whichmay restore sightfor millions of people around the world. The scientists caution that they will need to conduct more research to see how the procedure works with human transplant patients. Human trials are still a long way off.

READ MORE: Eye stem cells transplanted from corpses to live patients could cure blindness

For more information on each of these stem cell treatments, you can refer to the READ MORE links in between each section.

Go here to read the rest:
Stem Cell Magic: 5 Promising Treatments For Major Medical Conditions - Study Finds

The objective of this study is to synthesise the findings of clinical studies in order to derive evidence for use of themesenchymal stem cell (MSC) therapy in the treatment of neurodegenerative cerebellar ataxias. In order to find relevant studies for the systematic review, we searched through Medline (1985 to July 2020), PubMed and Clinical trial register. We included both single-arm and comparative studies in which MSCs were given as intervention in neurodegenerative ataxia patients at any time after the diagnosis. We used Joanna Briggs Institute (JBI) quality scale to evaluate themethodological qualities of theincluded studies. Our literature search obtained 81 publications. Three articles comprising atotal of 47 patients were included in the meta-analysis. None of them were randomised controlled trials (RCTs). Pooled analysis noted that there was adecrease in the Berg Balance Scale (BBS)/Scale for the Assessment and Rating of Ataxia (SARA) score from pre to post assessment; however, the difference was statistically not significant (standardised mean difference (SMD)0.20; 95% CI0.78 to 0.38). No significant side effects were reported in any of the studies. We did not observe any statistically significant difference in the pooled mean difference in the International Cooperative Ataxia Rating Scale (ICARS) score between pre and post assessment in patients with ataxia after receiving the stem cells (SMD 0.36, 95% CI0.08 to 0.81). Our systematic review and meta-analysis concluded that MSC cell therapy appeared safe but provided insufficient evidence to support the use of MSCs to treat patients with neurodegenerative cerebellar ataxia at present. No l RCTs was available in the literature to test efficacy; therefore, well-designed RCTs are needed to ascertain the effectiveness of MSCs in patients with neurodegenerative cerebellar ataxias.

Follow this link:
Mesenchymal Stem Cell Therapy in the Treatment of Neurodegenerative Cerebellar Ataxias: a Systematic Review and Meta-analysis - Newswise

Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant

Study highlights increases in omidubicel use in eligible patients were associated with higher proportions of patients undergoing allo-HCT and overall improved outcomes, with improvements being greater among racial minorities

Only 20% of Black cancer patients can find a matched unrelated donor through donor registries, limiting access to disease-altering allogeneic hematopoietic stem cell transplants

Omidubicel BLA submission on track to be completed in second quarter of 2022

BOSTON, April 25, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today announced the results of a new study demonstrating the potential impact of access to omidubicel on health disparities in allogeneic hematopoietic stem cell transplant in a poster presentation at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022.

The study, titled "Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant Access and Outcomes for Patients with Hematologic Malignancies in the US," leveraged a decision-tree model to project allo-HCT access and clinical outcomes in a hypothetical population of 10,000 allo-HCTeligible patients in the U.S. with hematologic malignancies without an available match-related donor. The study concluded that broad use of omidubicel will extend access for allo-HCT-eligible patients, decrease time to transplant and improve clinical outcomes, notably among racial and ethnic groups with worse status quo outcomes. Projected increases in one-year overall survival ranged (with 20% omidubicel use among allo-HCTeligible patients) from 2.5% for whites patients to 6.3% for Black patients. The study also concluded that higher levels of modeled omidubicel uptake were associated with greater improvements in clinical outcomes and greater reductions in racial disparities.

Story continues

Previous studies indicate that non-white patients have a lower likelihood of finding an appropriate match in the U.S. public donor registries, with Black patients have a 16-20% chance of finding an appropriate match. Given that an allogeneic stem cell transplant is intended as a curative option, if patients cannot find an appropriate match they will not have access to allogeneic stem cell transplant, a potentially curative treatment. The Phase 3 study of omidubicel demonstrated the ability of the therapy to be used as a donor source for racially and ethnically diverse patients with 40% of patients enrolled in the study being non-white.

"Today, minority groups comprise only about 30% of all allogeneic hematopoietic stem cell transplant transplants, indicating that lack of access to a matched donor is a significant barrier to treatment in the current landscape," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "This study is encouraging in that it projects that broad access to omidubicel has the potential to open up allo-HSCT as an effective treatment for more patients and address the barriers that have contributed to this alarming health disparity. These data are particularly encouraging as we continue to advance our rolling BLA submission to the FDA and move closer to bringing the therapy to more patients in need."

Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.

In addition to this poster, two oral presentations and four additional poster presentations on omidubicel and a poster presentation on GDA-201, the companys leading NK cell therapy program, will be shared during the conference. All poster presentations will be publicly available at http://www.ASTCT.org. Details below:

Title (Oral Presentation): Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation (Part of Best Abstract Award Session)

Presenting Author: Paul Szabolcs, M.D., Division of Blood and Marrow Transplantation and Cellular Therapy, UPMC Childrens Hospital of Pittsburgh, Pittsburg, PA

Session Title: Tandem Meetings Best Abstracts Session in the Scientific Track

Session Date / Time: Monday, April 25, 2022, 6:00 PM - 6:15 PM MT, SPCC, Ballroom D

Title (Oral Presentation): Allogeneic HSCT with Omidubicel demonstrates sustained clinical improvement versus standard myeloablative UCBT: Final results of a Phase III randomized multicenter study

Presenting Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute

Session Title: Oral Abstract - Session L - Consider the Source: Stem Cell Grafts and Donors

Session Date / Time: Tuesday, April 26, 2022, 3:15 PM 3:30 PM MT

Title: (Poster): Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) With Omidubicel: Long-Term Follow-Up From A Single Center (ASH Encore)

Lead Author: Chenyu Lin, M.D., Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, NC

Title (Poster): Total Costs of Care and Complication Rates Among Patients with Hematologic Malignancies Who Receive Allogeneic Hematopoietic Cell Transplants in the US

Lead Author: Richard Maziarz, M.D., Professor of Medicine, Medical Director Adult Blood and Marrow Stem Cell Transplant Program, Oregon Health and Science University, Portland, OR

Title (Poster): Hospitalization and Healthcare Resource Use of Omidubicel Vs Umbilical Cord Blood (UCB) for Hematological Malignancies in a Global Randomized Phase III Clinical Trial Setting

Lead Author: Navneet Majhail, M.D., Taussig Cancer Institute, Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH

Title (Poster): HRQOL following transplantation with omidubicel versus UCB in patients with hematologic malignancies: Results from a Phase III randomized , multicenter study

Lead Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute

Title (Poster): Transcriptional and Metabolic Profiling of Nicotinamide-Enhanced Natural Killer (NAM-NK) Cells (GDA-201)

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.

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

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 are able to enhance, 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. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.

About Gamida Cell

Gamida Cell is pioneering a proprietary NAM-enabled immunotherapy pipeline of diverse potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies 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 transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information on Gamida Cell, 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 GDA-201), anticipated regulatory filings and the potentially life-saving or curative therapeutic and commercial potential of omidubicel. 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220425005348/en/

Contacts

For investors: Courtney TurianoStern Investor Relations, Inc.courtney.turiano@sternir.com 1-212-362-1200

For media: Rhiannon JeselonisTen Bridge Communicationsrhiannon@tenbridgecommunications.com 1-978-417-1946

Read the rest here:
Gamida Cell Announces Results of New Health Economic and Outcome Study Reporting Improved Health Equity and Health Outcomes With Omidubicel at 2022...

Diabetes mellitus (DM) is a worldwide chronic epidemic disease of impaired glucose metabolism. Transplantation of mesenchymal stem cells (MSCs) is considered a promising emerging treatment strategy for diabetes. However, the harsh internal environment of DM patients can inhibit the treatment effects of transplanted MSCs. Fortunately, this adverse effect can be reversed by resveratrol (Res). Therefore, we investigated and summarized relevant studies on the combined treatment of diabetes with MSCs and resveratrol. This review presents the therapeutic effects of this combination therapy strategy on DM in glycemic control, anti-inflammatory, anti-oxidative stress and anti-fibrotic. Moreover, this review explained the mechanisms of MSCs and resveratrol in diabetes treatment from 3 aspects, including promoting cell survival and inhibiting apoptosis, inhibiting histiocyte fibrosis, and improving glucose metabolism. These findings help to understand in-depth mechanisms of the treatment of DM and help to propose a potential treatment strategy for DM and its complications.

See the original post here:
The combined therapy of mesenchymal stem cell transplantation and resveratrol for diabetes: Future applications and challenges - Newswise

Jasper Therapeutics

JSP191 is well tolerated with no treatment-related severe adverse events in 24 patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) in Ph1b dose expansion study

24 of 24 patients achieved successful engraftment with neutrophil recovery

20 of 24 patients determined to be free from morphologic relapse or disease progression at last follow up

REDWOOD CITY, Calif., April 26, 2022 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc., (NASDAQ: JSPR) a biotechnology company focused on hematopoietic stem cell therapies, today announced updated efficacy, safety and pharmacokinetic data from its ongoing multicenter Phase 1 clinical trial of JSP191, the companys first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing allogeneic hematopoietic (blood) cell transplantation.

Updated data from the multicenter study showed that conditioning with a single dose of JSP191 0.6 mg/kg prior to low dose radiation and fludarabine in preparation for transplantation was well tolerated and led to successful engraftment as evidenced by primary neutrophil recovery and full donor myeloid chimerism in twenty-four older patients (aged 62-79) with AML in complete response (CR) or MDS. Twenty patients were determined to be free from morphological relapse or disease progression at last follow up with four patients off study due to relapse or progression. Clearance of measurable residual disease (MRD) was observed in 12 of 20 evaluable patients at last follow up. One case of late-onset grade III-IV acute GI graft vs. host disease (GVHD) and one case of secondary graft failure were reported. No JSP191 related Significant Adverse Events, no cases of classical acute grade II-IV GVHD and no cases of transplant related mortality within 100 days 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 2022 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).

Story continues

We are excited about the progress of JSP191 as a targeted conditioning agent in patients with MDS or AML in CR undergoing hematopoietic stem cell transplant. These data show that JSP191 may be safely used on top of a standard conditioning regimen in older patients unable to tolerate myeloablative conditioning, said Ronald Martell, President and CEO of Jasper Therapeutics. We are looking forward to the start of a registration clinical study of JSP191 for transplant conditioning in MDS or AML in CR patients and the potential to bring safer and more effective conditioning to the growing population of older patients in need of blood stem cell transplant.

The Phase I trial is an open-label, multicenter study evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine in patients aged 60 or older with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for 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. Secondary endpoints include engraftment and donor chimerism, MRD clearance, non-relapse mortality, event-free survival, and overall survival.

For more information on the study, refer to Clinicaltrials.gov identifier NCT04429191

About MDS and AML

Myelodysplastic syndromes 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. In about one in three patients, MDS can progress to AML, a rapidly progressing cancer of the bone marrow cells. Both are diseases of the elderly with high mortality. Each year, about 29,000 patients are diagnosed with MDS and approximately 42,000 patients are diagnosed with AML in the G7 countries for which approximately 2,500 patients with MDS and approximately 8,000 people with AML receive hematopoietic stem cell transplants. These transplants are curative but are underused due to the toxicity of the current intensive conditioning agents that have many off-target toxicities, which includes the chemotherapy agents busulfan and melphalan.

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 company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. Clinical study of JSP191 as a novel, disease-modifying, therapeutic for patients with lower risk MDS is also planned to begin in 2022. In parallel, Jasper Therapeutics is advancing its preclinical mRNA hematopoietic stem cell platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the potential benefits of hematopoietic stem cells (HSC) engraftment following targeted JSP191 conditioning in the treatment of myelodysplastic syndromes, acute myeloid leukemia, or severe combined immunodeficiency and JSP191s potential generally. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper Therapeutics and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper Therapeutics. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper Therapeutics develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jasper Therapeutics product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper Therapeutics will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that final study data may not be consistent with preliminary study data; the risk that Jasper Therapeutics product candidates may not be beneficial to patients or successfully commercialized; the risk that Jasper Therapeutics has overestimated the size of the target patient population, their willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper Therapeutics business; the risk that third parties on which Jasper Therapeutics depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper Therapeutics business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper Therapeutics will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others and other risks and uncertainties indicated from time to time in Jasper Therapeutics public filings with the SEC. If any of these risks materialize or Jasper Therapeutics assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that Jasper Therapeutics does not presently know, or that Jasper Therapeutics currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. While Jasper Therapeutics may elect to update these forward-looking statements at some point in the future, Jasper Therapeutics specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper Therapeutics assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts:

John Mullaly (investors)

LifeSci Advisors

617-429-3548

jmullaly@lifesciadvisors.com

Jeet Mahal (investors)

Jasper Therapeutics

650-549-1403

jmahal@jaspertherapeutics.com

Follow this link:
Jasper Therapeutics Announces Updated Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Older Patients with...

A reduction in steroid-refractory chronic graft versus host disease (cGVHD) and prednisone use was observed when patients were treated with abatacept (Orencia) following stem cell transplant, according to a phase 2 trial (NCT01954979) presented at the 2022 Tandem Meeting.

Abatacept was associated with a 58% overall response rate in steroid-refractory cGVHD, said lead author Anita G. Koshy, MD, a clinical fellow in medicine at Beth Israel Deaconess Medical Center. There was a durable reduction in prednisone dosing over time, severe infections were uncommon, and the infusions were well tolerated. Correlative immune studies are ongoing to better understand the mechanism of action of abatacept and chronic GVHD.

Investigators defined steroid-refractory cGVHD as persistent signs and symptoms of cGVHD despite the use of 0.5 mg/kg daily prednisone for at least 4 weeks.

Thirty-nine patients who had undergone HCT a median of 43 months prior to the study received abatacept and 36 were evaluable for overall response rate (ORR). Eighteen patients had moderate cGVHD at baseline and 21 had severe cGVHD. Patients received a median of 5 (range, 1-11) previous treatments for cGVHD.

Investigators administered 10 mg/kg abatacept every 2 weeks for 3 doses. One month after administration of the third dose, patients received 3 additional doses every 4 weeks. One month later, patients received the sixth dose of abatacept.

Koshy and her colleagues assessed patients for response 1 month after administration of the sixth dose and observed an ORR of 58%. All responses were partial.

Patients who completed 6 doses of abatacept and continued to demonstrate response were eligible to receive up to 12 additional doses.

At baseline, skin (84%) was the most common site of cGVHD, followed by joints (82%), eyes (72%), lung (56%), mouth (44%), liver (23%), and gastrointestinal (GI) tract (15%).

The most improved organ sites included the skin in 22% of patients, the mouth in 22% of patients, the eyes in 25% of patients, lung in 36% of patients, and joints in 22% of patients, Koshy said. Progression of disease occurred in 33% of patients and included organs such as the skin, mouth, eyes, liver, lung, and joints.

Rate of cGVHD declined to 8% in patients with GI involvement.

Koshy added that abatacept led to a durable reduction in prednisone use. At baseline, the median daily dose of prednisone 20 mg per day. There was a 27.5% reduction in the prednisone dose to 14.5 mg by the 1 month follow up time point, she said.

At the 4-month follow-up, prednisone dose further declined to 10 mg per day.

Eligible patients had received allogeneic bone marrow or stem cell transplantation with myeloablative or reduced intensity conditioning regimens. They had to be at least 100 days removed from transplantation or donor lymphocyte infusion to enroll.

Patients were not allowed to change immunosuppressive medications for at least 4 weeks prior to enrollment and had to be on a stable immunosuppressive regimen for 2 weeks prior to enrollment.

The median patient age was 62 years (range, 25-77) and most patients were female (53.8%). The majority of patients had an ECOG performance score of 1 (69.2%) or 2 (28.2%). The most common sites of primary disease were acute myeloid leukemia (46.2%), myelodysplastic syndrome (20.5%), and acute lymphoblastic leukemia (12.8%).

The majority of patients (89.7%) received peripheral blood stem cell transplantation. The remainder underwent bone marrow transplant.

For conditioning intensity, 61.5% of patients received myeloablative conditioning while 35.9% received nonmyeloablative.

Just over half (56.4%) of patients received matched unrelated human leukocyte antigens (HLA) and 38.4% received matched related HLA. One (2.6%) patient each received mismatched related and mismatched unrelated HLA.

The most common grade 3/4 adverse event possibly related to abatacept treatment was neutropenia in 4 patients. Investigators observed serious grade 3/4 lung infections in 3 patients. One patient experienced grade 4 hemolysis, respiratory failure, and hepatic failure, and eventually died of concurrent HSV hepatitis while on treatment.

Immune correlative studies are ongoing to help us better understand the mechanism by which abatacept may work in the treatment of cGVHD, Koshy said in conclusion. Thus far, T cell polarization assessments by flow cytometry have shown no difference in [interleukin] 10 and interferon gamma expression by CD4-positive T cells before and after treatment in our clinical responders. However, cytokine profiling of patient plasma and single cell RNA sequencing are currently underway to help us to better understand changes to the immune microenvironment potentially caused by abatacept.

Koshy AG, Kim HT, Stroopinksy D, et al. Phase II clinical trial of abatacept for steroid-refractory chronic graft versus host disease. Presented at: the 2022 Transplantation & Cellular Therapy Meetings; Salt Lake City, UT; April 23-26, 2022. Abstract 32.

See more here:
Reduction in GVHD and Prednisone Use With Abatacept Observsed After Stem Cell Transplant - Cancer Network