Selectively Targeting Leukemia

Selectively Targeting Leukemia

The National Cancer Institute has awarded Ulrich Steidl, M.D., Ph.D., a five-year, $1.9 million grant to study pre-leukemic stem cells and how they are induced to form leukemia cells. The research could lead to better treatment options against acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), a preleukemic disease. Cure rates over the past 45 years for both conditions have been no better than 20 percent. Dr. Steidl’s group previously showed that PU.1, a transcription factor that regulates the expression of leukemia-related genes, is frequently inactivated in pre-leukemic stem cells and in patients with AML. Using genetic models and novel inhibitors of PU.1, Dr. Steidl hopes to uncover critical pathways that induce pre-leukemic stem cells to produce leukemia cells. These PU.1 inhibitors could potentially be developed into a drug, since they appear to selectively kill leukemia cells. Dr. Steidl is professor of cell biology and of medicine and is the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research. (1R01CA217092-01)