In light of the heightened sensitivity we observed for a non-MLL-rearranged FLT3-ITD leukemia cell line to DOT1L inhibition and the coinciding reductions in cell viability and FLT3 expression, small molecules such as pinometostat may prove effective in treating the 30–40% of AML bearing FLT3-ITD mutations. This evidence concerns the gene FLT3 and acute myeloid leukemia.