AI-10-49 is of particular interest for treatment of (inv16) AML as it inhibits RUNX1 binding to CSF1R, RUNX3 and CEBPa promoter targets and also strongly increases mice survival in CBFβ-MYH11 murine leukemia cell models by reducing leukemia burden. The gene discussed is RUNX1; the disease is acute myeloid leukemia.