In the bone-marrow cancer myelodysplastic syndrome (MDS), 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 anemia in low-to-intermediate risk MDS patients without the need for blood transfusions.

The findings, published online on September 3 in Clinical Cancer Research, come from an international multicenter phase 2 clinical trial conducted at 14 centers in Italy, Spain and Germany. The study’s principal investigator was Amit K. Verma, MBBS, professor of medicine and of developmental and molecular biology at Einstein and attending physician in oncology at Montefiore Einstein Center for Cancer Care.

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, MD, associate professor of hematology at the University of Florence.