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Latest News
Explore the forefront of MDS research, treatments, and community happenings to stay informed on the ever-evolving landscape of MDS care.
June 12, 2020
Molecular International Prognostic Scoring System Developed for Myelodysplastic Syndromes
The MDS Foundation introduces the IPSS-Molecular, a groundbreaking prognostic scoring system for Myelodysplastic Syndromes (MDS). Developed through global collaboration, it integrates genetic data to offer personalized risk assessment, revolutionizing treatment strategies. The IPSS-M is set to become the international standard, aiding clinicians with a web-based calculator for tailored patient care.
June 12, 2020
Geron Reports Four Imetelstat Data Presentations at Virtual Edition of the European Hematology Association (EHA) Annual Congress
Geron Corporation presented four sets of data on imetelstat, its telomerase inhibitor, at the Virtual Edition of the European Hematology Association (EHA) Annual Congress.
First, from the IMerge Phase 2 trial, they reported encouraging transfusion independence rates and potential disease-modifying effects, suggesting imetelstat's efficacy in treating lower risk myelodysplastic syndromes (MDS).
Second, data from the IMbark Phase 2 trial indicated improved overall survival with imetelstat treatment in patients with myelofibrosis (MF), along with other clinical benefits such as fibrosis improvement and symptom response.
These findings provide support for Geron's ongoing Phase 3 trials of imetelstat. In summary, imetelstat shows promise in treating hematologic malignancies, potentially altering disease progression, and improving patient outcomes.
June 3, 2020
Aprea Therapeutics Completes Full Enrollment of Phase 3 Clinical Trial in TP53 Mutant Myelodysplastic Syndromes (MDS)
Aprea Therapeutics announced the completion of patient enrollment for its Phase 3 clinical trial evaluating eprenetapopt combined with azacitidine for front-line treatment of TP53 mutant myelodysplastic syndromes (MDS). Topline results are expected by the end of 2020, with regulatory submissions planned for 2021 in the US and EU. The trial aims to compare the combination therapy to azacitidine alone, involving 154 patients with a primary endpoint of complete remission rate. Eprenetapopt is designed to reactivate the mutant p53 tumor suppressor protein, which is commonly mutated in various cancers, including MDS.
February 11, 2020
Astex Pharmaceuticals announces U.S. Food and Drug Administration (FDA) acceptance for review of and NDA for the combination oral hypomethylating agent cedazuridine and decitabine (ASTX727 or oral C-DEC), for the treatment of MDS and CMML
Astex Pharmaceuticals announced that the U.S. FDA has accepted its New Drug Application (NDA) for Priority Review for the combination oral hypomethylating agent cedazuridine and decitabine (ASTX727 or oral C-DEC), intended for the treatment of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). This application is supported by data from the Phase 3 ASCERTAIN study, which demonstrated the equivalence of oral C-DEC to intravenous decitabine over a 5-day treatment cycle. The FDA's Priority Review designation could accelerate the approval process to six months. If approved, oral C-DEC would become the first oral hypomethylating agent for these conditions in the U.S.
January 9, 2020
New England Journal of Medicine Publishes Results of Pivotal Phase 3 Reblozyl® (luspatercept-aamt) MEDALIST Trial
Bristol-Myers Squibb and Acceleron Pharma announced the publication of results from the pivotal phase 3 MEDALIST trial in the New England Journal of Medicine. The study evaluated Reblozyl® (luspatercept-aamt) for treating anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts who require red blood cell transfusions and are unresponsive to erythropoiesis-stimulating agents. The MEDALIST trial showed that Reblozyl significantly improved red blood cell transfusion independence and met key secondary endpoints, with manageable safety profiles. The U.S. FDA is reviewing Reblozyl for this MDS indication, with a decision expected by April 4, 2020.
December 16, 2019
Agios Receives FDA Breakthrough Therapy Designation for TIBSOVO® (ivosidenib) for the Treatment of Adult Patients with Relapsed or Refractory Myelodysplastic Syndrome with an IDH1 Mutation
Agios Pharmaceuticals announced that the FDA has granted Breakthrough Therapy designation to TIBSOVO® (ivosidenib) for treating adult patients with relapsed or refractory myelodysplastic syndrome (MDS) with an IDH1 mutation. This designation, which aims to expedite the development and review of drugs showing substantial improvement over existing therapies, was based on promising results from a Phase 1 study. The study showed that TIBSOVO® was well-tolerated and effective in achieving durable remissions and transfusion independence. Agios plans to gather additional data to support regulatory filing and expand the study to include more patients.
August 8, 2019
Geron Starts Phase 3 Clinical Trial in Lower Risk Myelodysplastic Syndromes
Geron Corporation has initiated patient screening and enrollment for the Phase 3 portion of IMerge, a clinical trial assessing imetelstat, a telomerase inhibitor, in lower risk myelodysplastic syndromes (MDS). This phase aims to confirm the positive outcomes observed in the Phase 2 segment, evaluating imetelstat's potential as a treatment option for patients with lower risk MDS who are relapsed after or refractory to erythroid stimulating agents (ESAs). The trial will enroll approximately 170 patients globally and is expected to deliver top-line results by mid-year 2022. Imetelstat has shown promise in providing transfusion independence and disease-modifying activity in previous studies. Geron remains optimistic about the potential of imetelstat in addressing the unmet needs of patients with lower risk MDS.
April 26, 2019
Celgene & Acceleron announced the submission of a Marketing-Authorization Application to the EMA for luspatercept
Celgene Corporation and Acceleron Pharma Inc. have submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept, an investigational therapy, for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia and beta-thalassemia-associated anemia who require red blood cell transfusions. The submission follows the Biologics Licensing Application submission to the U.S. FDA earlier this month. Luspatercept, a first-in-class erythroid maturation agent, demonstrated promising safety and efficacy results in the pivotal phase 3 studies MEDALIST and BELIEVE. The companies aim to address the anemia associated with these serious diseases and reduce the burden of transfusions. Both companies are optimistic about luspatercept's potential as a therapeutic option and await regulatory review and approval.