Dr. Schiller on Addressing Unmet Needs in Myelofibrosis Treatment

Gary J. Schiller, MD, professor-in-residence, Department of Medicine, Hematology/Oncology, David Geffen School of Medicine, University of California, Los Angeles, discusses several areas of unmet need in myelofibrosis, and how to potentially address these challenges.

Some of the available therapeutic agents can reduce the adverse effects associated with myelofibrosis and other myeloproliferative diseases (MPNs), such as inflammatory symptoms and fatigue, Schiller states. However, an unmet need still exists, as these approved therapies are associated with bone marrow toxicity as well as decreased red blood cell (RBC) count and platelet production, Schiller adds.

Accordingly, research in this space should focus on developing drugs that target the disturbance to clonal hematopoiesis, but do not negatively affect this process in non-cancerous cells, according to Schiller. Efforts should also focus on ameliorating the substantial morbidity and mortality associated with allogenic stem cell transplantation, which makes it difficult to operationalize this therapy in myelofibrosis, Schiller adds. Notably, patients with myelodysplastic syndrome/MPN experience a great amount of unmet need due to very limited treatment options, Schiller concludes.