In a phase 2 multicenter trial of venetoclax administered as monotherapy in patients with relapsed/refractory AML an overall response rate of 19% was observed: interestingly, three of the five patients exhibiting a complete response have IDH-mutant AMLs [85]; 33% of AML patients with IDH1/2 mutations achieved a response to venetoclax monotherapy treatment (Table 2) [85]. The gene discussed is IDH1; the disease is acute myeloid leukemia.