As our understanding of the molecular aberrations driving AML increases, a number of targeted therapeutics, such as protein kinase inhibitors (FLT3, PI3K, Akt, Erk or Pim inhibitors), inhibitors of DNA methylating- and acetylating enzymes, such as DNMT1, DNMT3, DOT1L and HDACs or BH3-mimetics against anti-apoptotic Bcl-2 proteins are being developed3,4. The gene discussed is FLT3; the disease is acute myeloid leukemia.