Unlike FLT3 inhibitors currently in clinical trials which target the FLT3 kinase activity, the TOPK inhibitor targets the expression of FLT3 in FLT3-ITD AML cells and, therefore, may overcome the obstacles observed with the FLT3 kinase inhibitors, including the emergence of resistant clones that carry mutations in the kinase domain, or the negative feedback mechanisms resulting in the upregulation of FL ligand and FLT3 expression. The gene discussed is FLT3; the disease is acute myeloid leukemia.