Latest news with #GVHD
Time of India
3 days ago
- Health
- Time of India
Marengo CIMS Hospital completes 250+ Bone Marrow Transplants
Ahmedabad: Marengo CIMS Hospital has announced successful performance of more than 250 bone marrow transplants. According to the CIMS hospital, in the past two years, the department has successfully performed high-risk transplants, including haploidentical (half-match) transplants, matched unrelated donor (MUD) transplants, and allogeneic full-match transplants. It is also stated that the center has extensive expertise in pediatric bone marrow transplants, with procedures successfully carried out on children under 2 years of age. The hospital states that techniques such as T-cell engineering and ex vivo depletion are utilized to minimize the risk of complications like Graft-Versus-Host Disease (GVHD). Commenting on the development, Dr. R Shankaran, Unit Medical Director, Marengo CIMS Hospital, said, "Crossing the 250+ bone marrow transplant milestone is a proud moment for our entire team. It reflects our clinical expertise, state-of-the-art facilities, and commitment to delivering life-saving treatments that meet global standards of excellence." Dr. Raajiv Singhal, Founding Member, Managing Director & Group CEO, Marengo Asia Hospitals, said, 'I extend my heartfelt congratulations to the entire team of clinicians at Marengo CIMS Hospital for their relentless dedication and for always putting the patient first. Crossing the 250+ bone marrow transplant milestone at Marengo CIMS Hospital is a proud testament to our pursuit of clinical excellence and patient-centric approach.'
Yahoo
07-07-2025
- Health
- Yahoo
Palm Beach physician pioneering new advances in ALS and inflammatory disease treatment
A Palm Beach physician-scientist is leading efforts to create breakthrough treatments for Parkinson's, ALS, Alzheimer's and other diseases through a biotech company she established nearly a decade ago. Dr. Simrit Parmar, a Northwestern University-trained oncologist and research scientist, launched Cellenkos Inc., in 2016 to accelerate the clinical development of "T regulatory cell" therapies for the treatment of autoimmune diseases and inflammatory disorders. T regulatory cells, or Tregs (pronounced tee-regs), are natural immune system regulator cells that suppress the activity of other immune cells to prevent excessive inflammation and autoimmune reactions. Before launching Cellenkos, Parmar conducted extensive research on these cells and developed therapies based on them. While working as an oncologist/hematologist in the Department of Stem Cell Transplantation and Cellular Therapy at the University of Texas's MD Anderson Cancer in Houston in 2009, she developed treatments using T regulatory cells derived from umbilical cord blood to help prevent graft-versus-host disease (GVHD), a complication that can occur after an allogeneic stem cell or bone marrow transplant. Parmar told the Daily News it was 'very discouraging' to watch patients die from GVHD, which motivated her to focus her research on controlling the inflammation caused by donor cells, starting with the discovery of T regulatory cells. As she continued to conduct her research, she discovered that these cells had uses beyond preventing GVHD. "We found that the application of these cells is beyond just that one niche," she told the Daily News. "It can be applied to any autoimmune disease, to any inflammatory disorder." In 2016, the cell technology Parmar developed at MD Anderson was licensed to Cellenkos, which now supports her ongoing research through a sponsored research agreement. Since then, Parmar has focused on her work advancing targeted Treg cell therapies designed to ease inflammation and regulate immune function. Cellenkos is currently in clinical trials for various inflammatory and autoimmune diseases, including ALS, bone marrow failure, severe COVID-19 and myelofibrosis. Results have been positive so far, Parmar said, noting that ALS patients have seen a delay in disease progression and functional decline and an improvement in quality of life. "We have made some great strides in neurodegenerative disease," she said. "We just published a paper in the New England Journal of Medicine Evidence on ALS, where we showed not only disease halting, but slight improvement in resolving inflammation. That research has applications beyond just ALS. We are hoping as we are garnering more funding, we can go after Parkinson's, Alzheimer's and multiple sclerosis." Inflammation is the root cause of many diseases including cardiac, respiratory, neurological and hematological disorders, Parmar said, and it also drives many autoimmune diseases. Parmar's cell therapies restore immune balance by targeting harmful T cells — a type of white blood cell called lymphocytes — that can mistakenly attack healthy cells in the body, and retraining the body's own defense mechanisms. "We have a drug that heals inside out," she said. "We believe that we neutralize the bad player, which may not have initiated the inflammation, but definitely plays a key role in the propagation of widespread inflammation." Parmar, a mother of three who lives in Palm Beach's North End and travels regularly to Houston, where Cellenkos is based, said she remains committed to developing cell therapies targeting a broad range of autoimmune and inflammatory diseases. Cellenkos is pursuing funding for larger ALS research studies in the U.S., and it is also preparing for the next phase of clinical trials required for federal Food and Drug Administration approval. Parmar said she sees Palm Beach County's emergence as a neuroscience hub as a key benefit of her move, and she hopes it will lead to new research, development, and partnership opportunities. "We're incredibly excited about the momentum building in the Palm Beach and Jupiter area around neuroscience innovation," she said. "With new opportunities for R&D collaboration in the region, it presents an ideal environment for us to expand. We see great potential not only to develop a manufacturing facility for neurological compounds … but also to establish a dedicated treatment center that brings these breakthroughs directly to patients.' Jodie Wagner is a journalist at the Palm Beach Daily News, part of the USA TODAY Florida Network. You can reach her at jwagner@ This article originally appeared on Palm Beach Daily News: Palm Beach physician-scientist pioneering groundbreaking ALS therapy
Scoop
16-06-2025
- Health
- Scoop
Australasian-led Clinical Trial In Stem Cell Transplant For Blood Cancers Set To Change 40 Years Of Standard Practice
Leading Australian and New Zealand researchers have demonstrated that the use of a new, less toxic drug combination after stem cell transplants for leukaemia significantly improves patient outcomes post-transplant, reducing the risk of the life-threatening complication of Graft Versus Host Disease (GVHD). The BM12 CAST trial, a major clinical trial led by the Australasian Leukaemia & Lymphoma Group (ALLG) across New Zealand and Australian hospitals, will transform global blood stem cell transplant outcomes for people with high-risk blood cancers like leukaemia. 'This new treatment triples the chances of a patient being alive, healthy and free of GVHD three years after stem cell transplant,' said lead ALLG BM12 researcher, Professor David Curtis, Clinical Haematologist and senior bone marrow transplant physician at The Alfred and Director of Malignant Haematology Research at the Australian Centre for Blood Diseases, Monash University. Blood stem cell transplants are often lifesaving for leukaemia patients, but they come with a high risk of life-threatening complications, especially in the first 100 days after transplant. Common side effects include infections, organ damage, and the often-debilitating Graft versus Host Disease (GVHD), an irreversible lifelong complication. 'The BM12 trial showed the new treatment combination is simple, safe and more effective than current methods in preventing GVHD, which contributes to death or life-long illness in 20% of patients undergoing a blood stem cell transplant,' said Prof Curtis. Results of BM12 trial were published in the New England Journal of Medicine and presented at the European Hematology Association 2025 Meeting in Milan, Italy. These results are game-changing for stem cell transplant patients, with cyclosporin and cyclophosphamide offering a new standard of care for prevention of GVHD for patients with aggressive blood cancers undergoing transplant from a matched related blood stem cell donor. Researchers compared the standard drug combination used for the last 40 years with a new combination of cyclophosphamide and cyclosporin, for leukaemia patients. The new drug combination tripled the number of patients that were alive, cured of the blood cancer and not suffering from GVHD three years after transplant (49.1% vs 14.2% for the standard drug combination). The risk of serious side effects also dropped from 32.4% to 19.7%. The trial's success has immediate implications for clinical practice in the management of blood stem cell transplants. The simplicity and effectiveness of the new treatment, along with reduced toxicity and improved patient outcomes, will become the new standard of care in GVHD prevention for matched sibling transplants. The ALLG BM12 CAST trial's success is particularly important for patients such as Gladys Borgueta, who was diagnosed with Acute Myeloid Leukaemia (AML) and admitted to Auckland Hospital's Motutapu Ward in May 2021. 'Nothing prepares you for an unexpected diagnosis like acute myeloid leukaemia, everything else becomes a blur and your world is turned upside down. Nothing prepares you for the months, weeks in hospital and sometimes in isolation,' said Gladys. ALLG researcher, haematologist Dr Clinton Lewis and colleagues from Auckland Hospital, met with Gladys and suggested the ALLG BM12 trial. 'I knew that the team around me would give me the best care possible. I had however no high expectations just because this was a trial, it could go either way,' Gladys said. Gladys was allocated to the new treatment arm of the BM12 clinical trial and received a stem cell transplant in January 2022. The new drug combination meant that her transplant was successful and she avoided serious side effects such as GVHD. Three years on, Gladys continues to do well after her transplant and is in remission from her leukaemia. 'Without research, I would not have, or for that matter any other patient, benefited from the treatment I got. I feel grateful and blessed to be given a second chance at life.' Auckland Hospital lead investigator, Dr Clinton Lewis, said, 'The results of this exciting study are already changing practice in New Zealand and will continue to improve the lives of cancer patients receiving allogeneic stem cell transplants.' 'Our options for treating Graft versus Host Disease in New Zealand remain very limited compared to other countries, so preventing this complication is incredibly important for our patients.' 'This study shows that we can help our patients live better lives, free of their cancer and GVHD, when we utilise this treatment approach. This sets an improved standard of care in New Zealand', said Dr Lewis. The BM12 CAST trial was funded by the Australian Government's Medical Research Future Fund, the Auckland Medical Research Fund and the Cancer Society of New Zealand.
Medscape
10-06-2025
- Health
- Medscape
S2 Episode 5: Posttransplant Monitoring in Myelofibrosis
This transcript has been edited for clarity. For more episodes, download the Medscape app or subscribe to the podcast on Apple Podcasts, Spotify, or your preferred podcast provider. Tania Jain, MBBS: Hello everyone. We're back in our myelofibrosis for Medscape InDiscussion podcast. This is season two, and this is episode five, where we'll talk about transplant outcomes in myelofibrosis and how to address relapse post-transplant. I'm Tania Jain, and today we have our very special guest, Dr Rachel Salit, who is an associate professor at Fred Hutchinson Cancer Center and the University of Washington School of Medicine. As a hematologist-oncologist, Dr Salit specializes in stem cell transplant, focusing on improving outcomes for patients with myeloproliferative neoplasms. Her research has included developing clinical trials to enhance and improve transplant success, prevent graft-vs-host disease (GVHD), and she also works on initiatives to support patients' return-to-work journeys post-transplant. Welcome, Dr Salit. It's an honor to have you. Rachel Salit, MD: Thank you for having me today. Jain: What we want to talk about today is pre-transplant, post-transplant, and how to think about transplant. I think we would all agree that transplant is presented and thought about differently, to some extent, by different clinicians and others who see patients in the clinic. What is your approach, and how do you go about telling patients about the role of transplant? Salit: The way I approach patients with myelofibrosis is from two different sides. One is the scoring system criteria — going over the Dynamic International Prognostic Scoring System (DIPSS) and the Mutation-Enhanced International Prognostic Scoring System (MIPSS-70). That would include what their blood counts look like, their molecular profile, cytogenetics, whether they have any peripheral blasts, and whether we think they're at imminent danger for transforming to leukemia or needing blood or platelet transfusions. The second side I look at with them is their life goals — what they are hoping for in terms of longevity vs quality of life. I think we've made great strides in transplant in the last 10 years in myelofibrosis since the approval of Janus kinase (JAK) inhibitors. I talk with them about the role of JAK inhibitors in the pretransplant setting — whether they've had one or not — depending on their symptoms and splenomegaly, and how that affects transplant timing. Ultimately, it's their choice. Patients often have strong opinions. There are patients with high-risk disease by scoring systems, but who are having a rather good quality of life and don't want to rush into treatment. Then there are patients with lower risk but who, because of younger age or life goals, want to proceed more expediently. I let the patient guide me. The third component that goes into it is what our donor search looks like. I often don't know that when I first meet the patient, so I always add the caveat: This is the conversation we're having now, but once we assess sibling matches or unrelated donors, risks and benefits might change. Jain: One of the things that challenges me is that there's a lot of prediction involved in the transplant decision, especially the first part you mentioned, about leukemia progression risk. There's no perfect metric to predict someone's transplant outcomes, whether for disease control, preventing relapse, or even nonrelapse mortality. How do you play the prediction game, without a crystal ball? Salit: I think it is one of the most challenging aspects of myelofibrosis. I empathize with patients in that we consider this a somewhat elective procedure that no one wants to need. It's tougher on them than it is for acute myeloid leukemia (AML) patients, where we say, 'This is your option, and you need to do it now.' For me, I try to look at the disease kinetics. When did it develop? How long has it been stable? If they have peripheral blasts in their blood, have they stayed at 1% for 2 years, or have they risen to 2% or 3%? Have their counts stayed stable? Is their spleen growing? Are their symptoms increasing? If patients have poor-risk factors but stable disease, I'm less aggressive than I am with patients who have more benign features but rapidly progressing disease. Sometimes in younger patients, the disease progresses faster than expected, especially if they have just one high-risk mutation. Jain: I always say that one of the most important things to follow is the trajectory of disease. When counts begin to drop or blasts start to appear, that makes you pause and think about transplant, or at least do a bone marrow biopsy to help make that decision. I want to shift gears. I know you've been involved in defining remission post-transplant, which is a huge unmet need. Many disease features don't resolve quickly after transplant, yet we consider the transplant successful. The forms and shapes that remission or recurrence can take post-transplant have evolved. Can you share how you got involved in unifying the concept of remission post-transplant and what insights you can offer for our audience? Salit: I got involved in this from two angles. Clinically, it's long been unsatisfying to tell patients post-transplant that they have low counts, persistent fibrosis, or splenomegaly when we discharge them from the acute transplant service at day 100. The providers we discharge them to — often general oncologists — aren't always sure what to say. Are you in remission? How long will it last? How do we test for it? The second part came from my work on the Medicare–Center for International Blood and Marrow Transplant Research (CIBMTR)) composite study, looking at whether transplant benefits patients over 55. We've been working on this for the past 7 years. When we met to define the response to transplant, we used the newly developed International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria for drug response in myelofibrosis, and we found that to be very complex for CIBMTR reporting. The people reviewing charts were not reporting consistently. I was approached by Wael Saber from CIBMTR to help develop a consistent remission definition so that centers reporting outcomes — remission, persistent disease, or relapse — could be compared. We want to draw conclusions about what regimens and donors are working best. We recognized that, unlike AML patients who go into transplant in CR1 (first complete remission), myelofibrosis patients go into transplant with active disease: persistent blasts, fibrosis, splenomegaly. We formed a committee of myelofibrosis transplant experts from the US and Europe. We wanted both CIBMTR and the European Society for Blood and Marrow Transplantation (EBMT) to have the same criteria so we could collaborate and combine data. The strongest takeaway was that the main thing transplant can do that drugs currently cannot is put patients into molecular remission. Most patients have driver mutations — JAK2, CALR, MPL — and we know the majority clear these mutations post-transplant. We recognized that patients with molecular remission may still have low counts or morphologic abnormalities. We classified these as subsets of molecular remission. We also agreed that persistent molecular abnormalities — especially if decreasing — shouldn't be considered relapse. Persistent disease can clear over time with rising CD3 chimerism and immune suppression taper. We reserved 'relapse' for patients who had previously cleared their mutation and then redeveloped it. That's a much more concerning scenario requiring intervention. Jain: That makes a lot of sense. One way I explain it to patients is that a transplant is an intervention with curative potential, but disease elimination takes time, even after donor hematopoiesis is established. It's not an immediate clinical remission — spleen and counts take time to improve. But once donor hematopoiesis is present, that can eventually trigger graft-vs-leukemia activity to eliminate residual disease. Do you know if there's a difference in how CIBMTR and EBMT capture relapse? Salit: I honestly don't know. But one of the goals of our project, with about six stakeholders from each registry, was to create a uniform definition. I think the recent New England Journal of Medicine paper by Gagelmann and colleagues spearheaded this molecular remission definition. CIBMTR and Dr Saber are very enthusiastic about this, and I think we can develop reporting forms that consistently capture remission, persistent disease, and relapse as three categories. We acknowledge there will be missing data. Not every center regularly tests molecular driver mutations. We've also discussed the role of chimerism. Fractionated chimerism is important, but most European centers don't assess it. They can do weekly molecular tests; we might test every 3 months at best. So, blood counts and chimerism will still play a role. I've piloted this approach with my patients, and they find it more satisfying. At day 100, I can say, 'You still have fibrosis, splenomegaly, and low platelets — but your JAK2 is negative. We're considering this remission.' That's much more reassuring for them than wondering why we even did the transplant. Jain: I totally agree. Bringing together the American and European efforts — making them globally unified — would be a huge advancement in research. Right now, it's hard to use or compare registry data across systems since it's not apples to apples. You mentioned you're already using this with your patients. There are times when spleen, fibrosis, or counts aren't fully normalized. Can you share your approach for relapse or 'impending relapse' when you see persistent disease features like cytopenias, transfusion needs, splenomegaly, dropping chimerism, or persistent molecular mutations? Salit: We check molecular driver mutations every 3 months — at months 3, 6, 9, and 12. If the 3-month result is negative but the patient still has low CD3 chimerism, needs transfusions, or has fibrosis, we start tapering immune suppression at day 100. We follow chimerism monthly during tapering. If it's not at 100% by 6 months, we may do a bone marrow biopsy to see if anything concerning is present. If the mutation's variable allele frequency is decreasing, that's reassuring. If it's back and chimerism is dropping — say it was 100% at day 100 and 90% at 6 months — we consider donor lymphocyte infusion (DLI), assuming they're off immune suppression and don't have GVHD. If they're still on immune suppression, we taper conservatively and wait a month before giving DLI. We only use therapy like hypomethylating agents and JAK inhibitors if blasts return in the peripheral blood or marrow, or if abnormal cytogenetics reappear. Jain: Those are tough situations — when blasts are visible, or disease features reappear. How often do you consider a second transplant in myelofibrosis? Salit: Not often. Thankfully, we're seeing only 5%-10% relapse rates. At our center, we're still doing relatively mild ablative transplants: Cytoxan/busulfan for those under 60, or decitabine/melphalan for those over 60. If a patient completely loses CD34 or CD33 chimerism, we consider a second transplant. There's concern that giving DLI in that setting could cause aplasia. If the disease burden is too high, we go straight to the second transplant. Jain: That makes sense. Well, this was phenomenal. Thinking about transplant is a nuanced process, and we loved hearing your thoughts. We look forward to reading about the remission definitions post-transplant and implementing them in the clinic. On behalf of our audience, thank you so much for your time. That concludes episode 5 on transplant outcomes in myelofibrosis. We'll see you in the next episode. Listen to additional seasons of this podcast. Primary Myelofibrosis Role of Hematopoietic Stem Cell Transplantation in Patients With Myeloproliferative Disease Decreasing Chronic Graft-Versus-Host Disease Rates in All Populations Diagnosis and Evaluation of Prognosis of Myelofibrosis: A British Society for Haematology Guideline Prognostic Value of Blasts in Peripheral Blood in Myelofibrosis in the Ruxolitinib Era Acute Myeloid Leukemia (AML) CIBMTR Myelofibrosis Medicare Study Proposals for Revised International Working Group-European LeukemiaNet Criteria for Anemia Response in Myelofibrosis Utility of Assessing CD3+ Cell Chimerism Within the First Months After Allogeneic Hematopoietic Stem-Cell Transplantation for Acute Myeloid Leukemia Clearance of Driver Mutations After Transplantation for Myelofibrosis Donor Lymphocyte Infusion and Molecular Monitoring for Relapsed Myelofibrosis After Hematopoietic Cell Transplantation Medscape © 2025 WebMD, LLC Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape. Cite this: Posttransplant Monitoring in Myelofibrosis - Medscape - Jun 10, 2025.
Yahoo
19-05-2025
- Business
- Yahoo
Graft Versus Host Disease (GVHD) Global Clinical Trials Review 2025: Unlocking Strategies from GVHD Clinical Trials Across G7 & E7 Regions
Dublin, May 19, 2025 (GLOBE NEWSWIRE) -- The "Graft Versus Host Disease (GVHD) - Global Clinical Trials Review, 2025" has been added to offering. The report offers a comprehensive analysis of the GVHD clinical trial landscape worldwide. Presenting top-line data, this report furnishes details about the number of trials and their average enrollment across major countries. It covers trials by region, country (G7 & E7), phase, status, endpoints, and sponsor types. This report also highlights key drugs involved in ongoing trials, curated from the Pharma Clinical Trials Database. This database consolidates information from over 80 global trial registries, conferences, journals, and news sources and is periodically updated through a dynamic process to ensure data accuracy and relevance. The insights provided in this report enhance strategic decision-making and help formulate effective strategies to gain a competitive edge. Scope Provides a snapshot of the global clinical trials landscape, offering top-level data by Region, Country (G7 & E7), Trial Status, Trial Phase, Sponsor Type, and End Point Status. Reviews leading companies in the sector and lists all relevant trials with titles, phases, and statuses. Details unaccomplished trials, including terminated, suspended, and withdrawn, along with reasons for uncompletion. Shows enrollment trends over the past five years and shares the latest news from the past three months. Specific sections might be modified based on data availability and relevance to the disease. Reasons to Buy Supports strategic business investment decisions. Identifies optimal locations for cost-effective and timely clinical trials. Provides a top-level analysis of the Global Clinical Trials Market, pinpointing key business opportunities. Clarifies trial count and enrollment trends globally, aiding market understanding. Compares completed with incomplete trials to assist in interpreting clinical trial success rates. Offers an assessment of trials at global, regional, and country levels. Sections may be customized based on data relevancy to the implicated disease. Key Topics Covered: Report Guidance Clinical Trials by Region Clinical Trials and Average Enrollment by Country Top Five Countries Contributing to Clinical Trials in Asia-Pacific Top Five Countries Contributing to Clinical Trials in Europe Top Countries Contributing to Clinical Trials in North America Top Five Countries Contributing to Clinical Trials in Middle East and Africa Top Five Countries Contributing to Clinical Trials in Central and South America Clinical Trials by G7 Countries Clinical Trials by Phase Clinical Trials by Trial Status Clinical Trials by End Point Status Subjects Recruited Over a Period of Time Clinical Trials by Sponsor Type Prominent Sponsors Top Companies Participating in Graft Versus Host Disease (GVHD) Therapeutics Clinical Trials Prominent Drugs Latest Clinical Trials News on Graft Versus Host Disease (GVHD) Clinical Trial Profile Snapshots Appendix A selection of companies mentioned in this report includes, but is not limited to: Sanofi Incyte Corp Novartis AG Johnson & Johnson Pfizer Inc Takeda Pharmaceutical Co Ltd Mallinckrodt Plc Roche Holding AG Bristol-Myers Squibb Co Bellicum Pharmaceuticals Inc For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
