In myelodysplastic syndrome (MDS), a cancer of the bone marrow, anemia occurs because the proliferation of abnormal blood cells leaves people with too few normal ones. An experimental cancer drug called galunisertib may offer a way to treat anemia in low- to intermediate-risk MDS patients without the need for blood transfusions.
The findings, published in September 2019 in Clinical Cancer Research, come from an international phase 2 clinical trial conducted at 14 centers in Italy, Spain, and Germany. The study’s principal investigator was Amit K. Verma, M.B.B.S., professor of medicine and of developmental and molecular biology at Einstein and director of the division of hemato-oncology at Montefiore.
Dr. Verma and colleagues developed galunisertib using tumor samples from patients at Montefiore. The drug inhibits a molecule called ALK5 that plays a role in activating defective MDS stem cells.
The research was co-led by Valeria Santini, M.D., associate professor of hematology at the University of Florence in Italy.