Anemia is the major cause of morbidity and mortality in people with low-risk (slowly developing) myelodysplastic syndromes (MDS). In 2020, the FDA approved a new drug (luspatercept) to treat anemia in these patients. However, for reasons that aren’t clear, not all patients respond to luspatercept and it’s currently not possible to determine who will respond.

In a study published online on May 27 in The Journal of Clinical Investigation, Amit Verma, M.B.B.S., Teresa Bowman, Ph.D., Srinivas Aluri, Ph.D., and colleagues identified a biomarker that can be used to predict clinical responsiveness to luspatercept and uncovered which pathways are directly target by the drug. The findings may allow clinicians to make informed treatment decisions based molecular features of the disease and reduce the exposure of vulnerable patients to unnecessary treatment.

Dr. Verma is associate director of translational science at the National Cancer Institute-designated Montefiore Einstein Comprehensive Cancer Center (MECCC), interim chair of the department of oncology, the Susan Resnick Fisher Academic Chair in Brain Cancer Research, and professor of oncology, of medicine and of developmental & molecular biology at Einstein. Dr. Bowman is the Renée E. and Robert A. Belfer Chair in Developmental Biology, professor and chair of developmental & molecular biology, professor of oncology and of medicine at Einstein, and a member of the Ruth L. and David S. Gottesman Institute for Stem Cell Research and Regenerative Medicine and the MECCC. Dr. Aluri is a research assistant professor of oncology and of medicine at Einstein.