As a heterogeneous disease associated with many high-risk molecular and cytogenetic features, the treatment landscape has also expanded to include novel molecular-targeted therapies for patients with mutations such as FLT3 and IDH1/2, and potentially for TP53. With increased awareness of AML has come the realization that disease-specific factors, and not patient factors such as age and performance status, maybe the critical determinants of outcome. This evidence concerns the gene IDH1 and acute myeloid leukemia.