Progress against Acute Myeloid Leukemia

Progress against Acute Myeloid Leukemia

Einstein researchers led by Ulrich Steidl, M.D., Ph.D., demonstrated the potential of a new class of drugs for treating acute myeloid leukemia (AML), the most common form of acute leukemia in adults. AML and other cancers are caused by mutated forms of the protein isocitrate dehydrogenase 1 (IDH1). The new drugs inactivated IDH1 in AML cells isolated from patients through a novel mechanism of action: They induced the malignant cells to differentiate, and then the cells died. These findings suggest that IDH1 inhibitors should be considered for preclinical and clinical development and highlight the value of precision medicine for targeting specific proteins. The findings were carried out in close collaboration with scientists at GlaxoSmithKline and published in the November issue of Nature Chemical Biology. Einstein collaborators on the paper included Amit Verma, M.B.B.S., and the paper’s first author, Ujunwa Cynthia Okoye-Okafor, Ph.D. Dr. Steidl is associate professor of cell biology and of medicine, and is the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research. Dr. Okoye-Okafor is a former graduate student in Dr. Steidl’s lab who defended her Ph.D. in 2015 and is currently completing her final year in the Medical Scientist Training Program. Dr. Verma is professor of medicine and of developmental & molecular biology.