A breakthrough therapy for polycythemia vera, a potentially fatal blood cancer, has emerged from a groundbreaking multi-center clinical trial led by the Icahn School of Medicine at Mount Sinai.
During the phase 2 study, a novel drug called rusfertide effectively curbed the excessive production of red blood cells, the hallmark of polycythemia vera, over a 28-week treatment period. The findings indicate that rusfertide could offer a promising alternative to therapeutic phlebotomy, a conventional treatment method that poses significant challenges for many patients. The results of this pioneering research were unveiled today (Feb. 21) in The New England Journal of Medicine.
Dr. Marina Kremyanskaya, MD, Ph.D., Associate Professor of Medicine (Hematology and Medical Oncology) at Icahn Mount Sinai and the study's lead author, hails rusfertide's distinctive mechanism of action, which targets iron availability to regulate blood cell production. With her extensive expertise in myeloid blood cancers and as the Medical Director for inpatient oncology at The Mount Sinai Hospital, Dr. Kremyanskaya underscores the potential of rusfertide as a transformative therapeutic intervention for polycythemia vera.
Acknowledging the collaborative efforts of her Mount Sinai colleagues, Dr. Ronald Hoffman, MD, and Dr. Yelena Ginzburg, MD, Dr. Kremyanskaya emphasizes the seamless transition from laboratory insights to clinical application. Dr. Hoffman, a distinguished figure in myeloproliferative neoplasms research and Professor of Medicine (Hematology and Medical Oncology) at Icahn Mount Sinai, served as a senior author on the publication. Dr. Ginzburg, an authority in iron metabolism and Professor of Medicine (Hematology and Medical Oncology) at Icahn Mount Sinai, provided pivotal preclinical evidence supporting the investigation.
Polycythemia vera, characterized by abnormal blood cell overproduction in the bone marrow, affects a limited number of individuals, with 1–3 new cases per 100,000 people diagnosed annually in the United States. This condition, also known as erythrocytosis, poses serious health risks including blood thickening and heightened susceptibility to heart attacks, strokes, and blood clots.
Existing treatment modalities for polycythemia vera encompass aspirin therapy, medications like hydroxyurea, interferon, and ruxolitinib to reduce red blood cell counts, and the conventional approach of phlebotomy. However, frequent phlebotomies present challenges for patients, necessitating extended physician visits and exacerbating iron deficiency symptoms common among polycythemia vera patients. Moreover, studies reveal that many patients treated with current methods often maintain hematocrit levels exceeding the clinically safe threshold of 45%, elevating their risk of complications.
The advent of rusfertide signals a transformative stride forward in the management of polycythemia vera, offering newfound hope for patients burdened by the constraints of existing treatment options. As further clinical investigations unfold, rusfertide holds the promise of reshaping the therapeutic landscape for this rare blood cancer, offering a beacon of optimism for patients and physicians alike.
Source: The Mount Sinai Hospital