Purpose of review: The extensive genomic characterization of acute myeloid leukemia (AML) led to the identification of a vast number of potential therapeutic targets. We review relevant data that have led to recent approval of new targeted therapies in AML and discuss the most promising drugs currently in development in this disease.
Recent findings: New formulations of cytotoxic agents, namely CPX-351 and gemtuzumab ozogamicin, improve the outcome of defined subgroup of patients. Midostaurin added to intensive chemotherapy is approved in FLT3-mutated AML. More selective FLT3 inhibitors and the IDH inhibitors enasidenib and ivosidenib have shown significant single agent activity in the relapsed setting, and preliminary results of combination strategies are encouraging. The addition of the BCL2 inhibitor venetoclax appears to markedly improve the results of hypomethylating agents. The therapeutic armamentarium of AML now includes novel cytotoxic drugs, drugs targeting recurrent oncogenes, or functional vulnerabilities of leukemic cells. Further work is required to optimize their integration to the current framework of AML management, including allogeneic stem cell transplantation.
Keywords: Acute myeloid leukemia; BCL2 inhibitors; FLT3 inhibitors; Hypomethylating agents; IDH inhibitors; Targeted therapy.