Acute myeloid leukemia (AML) remains a devastating disease in adults, with an overall survival rate of 30%, despite advances in supportive care and allogeneic stem cell transplantation. One of the rare success stories in AML was the development of differentiation therapy in the form of retinoic acid for the small subset (10%) of patients with acute promyelocytic leukemia (APL). While APL was once the subtype with the worst prognosis, it is now the best prognosis with an overall survival exceeding 85%.
Differentiation therapy does not exist for the remaining 90% of patients with AML, and the standard of care chemotherapy (cytarabine + an anthracycline) has not changed in more than thirty years.
My goal is to identify and develop differentiation therapy for the treatment of patients with acute myeloid leukemia.