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Physicians and scientists from Memorial Sloan Kettering Cancer Center (MSK) presented advances in transplantation and cell therapy at a joint conference sponsored by the American Society for Transplantation and Cell Therapy (ASTCT) and the International Center for Blood and Marrow Transplant Research.
Miguel Ángel Perales, MD, MSK bone marrow transplant expert, cell therapist, and director of the Adult Bone Marrow Transplant Service, presided over the meeting in his role as ASTCT President for 2023-2024. Alan Hanash, MD, bone marrow transplant expert and cell therapist; He also served as Program Chair of the Scientific Organization Committee. The event, known as the Tandem Meeting, was held in San Antonio from February 21-25, 2024.
Two presentations at the conference will focus on improving outcomes for patients with blood cancers, including the prevention and treatment of graft-versus-host disease (GVHD), a potentially serious side effect of allogeneic (donor-derived) stem cell transplants. New methods were highlighted. This type of transplant is used to treat certain blood cancers by replacing a patient’s cancer cells with donor stem cells, which grow and mature into healthy blood cells.
New treatment options for patients with chronic skin graft-versus-host disease
Chronic GVHD most commonly affects the skin, eyes, and mouth, but can also affect the lungs, joints, gastrointestinal tract, liver, reproductive organs, and other organs. Topical and oral steroids are the most common treatments for skin rashes associated with GVHD, but they can cause side effects and may not completely treat symptoms. Oral immunosuppressive drugs, such as ruxolitinib, ibrutinib, and vermosudil, are also used to treat chronic GVHD, but they are not effective in all patients.
Dermatologist Alina Markova, MD, presented results from a phase 2 trial investigating a new method of administering ruxolitinib to patients with cutaneous GVHD. The trial enrolled 24 participants, most of whom were being treated for acute leukemia or non-Hodgkin’s lymphoma and were experiencing persistent GVHD symptoms unresponsive to other treatments. Patients were asked to apply ruxolitinib cream on one side of their face or body and a non-medicated cream (placebo) on the other side. 3D whole-body photographs after 1 month showed that ruxolitinib cream significantly reduced skin rash compared to placebo. One patient experienced a mild headache possibly related to treatment. By performing genome sequencing, the researchers found differences in the expression levels of interleukin-12, a key protein that regulates the inflammatory immune response, among patients who responded to treatment. These promising results suggest that ruxolitinib cream is a safe and effective treatment option for patients with chronic cutaneous GVHD.
New delivery model improves outcomes after CD34-selected allogeneic stem cell transplantation
Recent studies have shown that using stem cells containing high levels of a protein called CD34 can help prevent cancer recurrence and GVHD in people receiving allogeneic transplants. Bone marrow transplant expert and cell therapist Michael Skold, M.D., gave an updated presentation on a phase 2 study evaluating how to most effectively use the drug antithymocyte globulin (ATG) in patients undergoing CD34-selective transplants. Shared the results of clinical trials. This study investigated whether this approach could improve immune reconstitution, which is essential for recovery in patients undergoing transplantation. Delayed immune reconstitution for more than 100 days after transplantation is associated with severe side effects and increased mortality.
ATG is used to prevent the body from rejecting new donor stem cells. However, giving too much can delay immune rebuilding. In this study, the researchers used a model-based approach to give each patient an individualized dose, rather than dosing based on the patient’s weight as is customary. The goal was to maximize immune reconstitution and minimize the risk of side effects and mortality.
The trial enrolled 59 pediatric and adult blood cancer patients undergoing their first allogeneic stem cell transplant. Participants received personalized doses of ATG, followed by treatment that included transplantation of CD34-selected donor cells. Of the 56 patients whose results were included in the analysis, researchers found that approximately 70% of patients who participated in this treatment regimen experienced immune reconstitution after 100 days. This result was considered positive for this patient group. At 18 months after treatment, recurrence-free and overall survival rates were 80% and 87%, respectively. The findings of this study showed that using a model-based approach to ATG administration appears to be beneficial for people undergoing this type of transplant.
Notable presentations and awards
Several other MSK researchers presented research on various topics in immunotherapy and stem cell transplantation. among them:
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Hepatopancreatobiliary surgeon Dr. Vinod Balachandran; We gave a plenary presentation on recent advances in mRNA vaccine-based immunotherapy. Learn more about these advances here.
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Bone marrow transplant expert Katherine Hsu, MD, gave a plenary presentation on natural killer cell alloreactivity during a session on selecting the ideal human leukocyte antigen mismatched donor.
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Infectious disease expert Tobias Hall, MD, gave a presentation on: The mycobiome, diverse communities of fungi in the gut, and their impact on outcomes after hematopoietic stem cell transplantation.
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Dr. Sandeep Raj, a bone marrow transplant expert, set out to study how systemic inflammation affects CAR T cell function, as predicted by a machine learning model he developed. The project was awarded the ASTCT New Researcher Award. The goal of this study is to identify patients at high risk of cancer recurrence after CAR T therapy and provide early intervention to improve clinical outcomes.
