[Recent advances in molecular targeted therapy for acute myeloid leukemia]

Acute myeloid leukemia (AML) is a heterogeneous disease, and recent advances in sequencing technology have revealed that the accumulation of various genetic abnormalities is deeply involved in the pathogenesis of AML and its clonal evolutions during disease progression. Prognostic classifications and therapeutic strategies based on the cytogenetic and genetic abnormalities have been proposed, and several new therapeutic agents targeting the molecular abnormalities have been energetically investigated to eliminate AML cells. In the last few years, several new agents, including FLT3, IDH, BCL-2, and Hedgehog inhibitors, have been approved for the treatment of newly diagnosed AML, and their clinical applications have begun in the United States. Although there has been a delay of several years, the clinical development of these new agents is also underway in Japan, and it is expected that a new era of AML treatment will begin in the near future. It is necessary to establish novel risk-adopted treatment strategies incorporating these agents in monotherapies and/or effectively designed combination therapies.