Based upon promising preclinical data in leukemia and clinical testing in solid tumor patients with platelet-derived growth factor receptor (PDGFR) mutations (65, 83), a Phase 2 study of the third-generation FLT3/PDGFR inhibitor crenolanib in adults with AML is also in progress (NCT01522469) (33). The gene discussed is FLT3; the disease is acute myeloid leukemia.