Researchers at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai have recently conducted a groundbreaking study that showcases the potential of a new combination therapy in improving outcomes for patients battling myelofibrosis, a rare and aggressive blood cancer.
Published in Nature Medicine, this phase 3 trial marks a significant milestone as the first randomized investigation into JAK inhibitor-based combination therapy in treatment-naive myelofibrosis patients. Led by Dr. John Mascarenhas, Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai, the study explores the efficacy and safety of pelabresib plus ruxolitinib in newly diagnosed myelofibrosis patients. The results indicate that the synergistic effect of these targeted therapies leads to deeper clinical responses, potentially altering the disease course and enhancing overall survival rates.
Dr. Mascarenhas expressed optimism about the study’s implications, stating, “We are hopeful that this study will mark the beginning of a new era in myelofibrosis treatment. By combining these therapies, we can achieve more profound and durable responses, offering a new approach to managing this challenging disease.”
According to the American Association for Cancer Research, an estimated 13,000 individuals in the United States are living with myelofibrosis. For patients who do not respond well to single-agent treatments, the prognosis typically remains poor, with survival rates declining within two to three years. The study’s findings suggest that combination therapy could prolong responses, improve disease control, and potentially extend survival outcomes for these patients.
The data from this trial hold the potential to pave the way for regulatory approval of combination therapy, setting a new standard of care for early myelofibrosis management. The comprehensive correlative analyses conducted as part of the study further underscore the therapeutic potential of this dual approach in altering disease progression, offering a ray of hope for patients facing a median survival of just five years.
Moving forward, the research team will continue to monitor patients for long-term survival outcomes. Additional insights from the study will be presented at the European Hematology Association meeting in June 2025 and at the American Society of Hematology conference in December, shedding further light on the therapy’s enduring benefits.
Notably, this groundbreaking study was carried out across 80 medical centers worldwide, underscoring its global significance in advancing the field of myelofibrosis research and treatment.
For more information, readers can refer to the study published in Nature Medicine by Raajit K. Rampal et al, titled “Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial” (DOI: 10.1038/s41591-025-03572-3).
This transformative research was made possible by The Mount Sinai Hospital, demonstrating their commitment to innovation and excellence in cancer care.
