Researchers from Mass General Brigham Cancer Institute will present research discoveries and outcomes from clinical trials in hematology/oncology, including cancer and common blood disorders, at the 2025 American Society of Hematology (ASH) annual meeting, held December 6-9, in Orlando.
Presentations from Mass General Brigham investigators include a plenary session that shares a new treatment for acute myeloid leukemia, a disease that has not seen a new standard of care treatment in over 50 years; a new therapy for an autoimmune bleeding disorder; a first-in-human trial of a novel CAR-T cell therapy in patients with multiple myeloma; a deep dive into the prognostic indicators of overall survival in patients with T-cell lymphoma; and the first study to examine a bleeding disorder trigged by immune checkpoint inhibitors, a common cancer therapy; and many more.
Below are a few highlights from this year’s presentations. All times are Eastern Standard Time (EST).
Results from PARADIGM, a phase 2 multi-center study, which describes a promising new treatment for acute myeloid leukemia (link to full abstract)
When: Sunday, December 7, 2-4 p.m.
Who: Amir Fathi, MD, Director of Leukemia Program, Mass General Brigham Cancer Institute
What: Dr. Fathi will present the results of the PARADIGM study, a phase 2, multi-center prospective study of treatment for acute myeloid leukemia (AML), during the Plenary Scientific Session. For decades, the standard of care treatment of newly diagnosed AML has involved a combination chemotherapy, requiring a prolonged hospital stay and associated with many acute toxicities. No alternative chemotherapy regimen has been shown to be superior to this regimen, to date.
Dr. Fathi led the design and conduct of the PARADIGM study, which compared traditional induction chemotherapy to a novel combination therapy, known as Aza/Ven (azacitidine and venetoclax), a much better-tolerated regimen currently approved for older patients with AML. Excitingly, the results showed that patients treated with Aza/Ven had improved event-free survival and higher overall response rates to therapy. Also, notably, patients receiving Aza/Ven reported better quality of life, lower symptom burden and depression symptoms during treatment, as well as substantial reduction in hospital days and time in the ICU during the initial hospitalization. This potentially represents a momentous shift in the approach to AML, disrupting a standard of care that dates back over 50 years.
New antibody therapy shows promise for autoimmune platelet disorder (link to full abstract)
When: Tuesday, December 9, 7:45-8 a.m.
Who: Hanny Al-Samkari, MD, Mass General Brigham Cancer Institute
What: This late-breaking abstract shares results from VAYHIT2, a phase 3 clinical trial evaluating a novel monoclonal antibody therapy, ianalumab, in people with immune thrombocytopenia (ITP). ITP is an autoimmune disorder that causes chronic low platelet counts, leading to uncontrolled bleeding and fatigue. Currently, because patients with this disease must take medications long-term, and because the disease becomes more challenging to treat over time, researchers have been seeking a way to intervene earlier to improve outcomes and potentially modify the disease course. Ianalumab works by targeting the immune system and blocking the BAFF receptor, a key signal that helps B cells survive and produce harmful antibodies. By shutting down this pathway, the drug depletes autoreactive B cells and prevents the maturation of new ones, and thereby stopping the immune system from attacking platelets. The VAYHIT2 trial found that ianalumab was safe and effective, and may help modify the disease itself, instead of just controlling its symptoms.
First-in-human, phase 1 trial of a novel CAR-T cell therapy in multiple myeloma patients (link to full abstract)
When: Saturday, December 6, 3:15-3:30 p.m.
Who: Charlotte Graham, MD, PhD, Ben Puliafito, MD, Matt Frigault, MD & Marcela Maus, MD, PhD, Mass General Brigham Cancer Institute
What: This presentation details the results of a first-in-human, phase 1 clinical trial of a novel CAR-T cell therapy in patients with multiple myeloma that has not responded to treatment or has returned (known as relapsed or refractory multiple myeloma). This new therapy, known as TriPRIL CAR-T cell, targets two antigens (BCMA and TACI), was designed and developed at Mass General Brigham Cancer Institute, and has demonstrated encouraging results in patients. The study reported an overall response rate of 80 percent, including 60 percent complete responses, even among patients who had previously received BCMA CAR-T therapy. The median progression-free survival was 8.4 months, and one patient remains in complete remission 14 months after treatment. These findings position TriPRIL as one of the first ligand-based CAR-T therapies to show clinical activity in patients who have received many other treatments before.
Early time-to-relapse as a predictor of survival in mature T-cell/NK-cell lymphomas: Results from the PETAL consortium (link to full abstract)
When: Monday, December 8, 2:45 - 3 p.m.
Who: Mark Sorial, PharmD, BCOP & Salvia Jain, MD, Mass General Brigham Cancer Institute
What: Nodal mature T-cell lymphoma (nMTCL) is a rare, aggressive form of non-Hodgkin lymphoma, for which the risk of cancer returning post-initial treatment is high. Researchers will share findings from a study of nMTCL patients that examined the relationship between the time at which a person’s cancer recurs and their overall chance of surviving. This is designed to inform treatment decisions for a second-line therapy. Dr. Jain and her colleagues found that patients with nMTCL who relapse within 12 months of starting treatment face significantly worse overall survival than those who relapse later; thus, showing that length of time to relapse is a predictor of survival. This defines a new, high-risk subgroup of patients, for whom researchers aim to reshape treatment strategies to more effectively control disease in one of the most challenging lymphoma types.
Immune thrombocytopenia in patients treated with immune checkpoint inhibitors (link to full abstract)
When: Monday, December 8, 2:45 - 3 p.m.
Who: Rebecca Leaf Karp, MD Mass General Brigham Cancer Institute and David E. Leaf, MD, MMSc, FASN, Mass General Brigham
What: Immune checkpoint inhibitors (ICIs) have revolutionized cancer treatment, but they can trigger autoimmune side effects called immune-related adverse events. Among these, blood-related complications like ICI-associated immune thrombocytopenia (ICI-ITP)—a condition in which platelet counts drop dangerously low—are rare and poorly understood. Researchers analyzed data from 86,648 patients treated with ICIs at 29 hospitals across 11 U.S. states, identifying 214 cases of ICI-ITP. This study is the first to provide detailed, real-world data on ICI-ITP. While most patients respond to standard treatments, the condition signals higher mortality risk and requires rapid recognition and management. The findings could shape future guidelines for monitoring and treating patients on ICIs.
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