Genetic analysis and targeted therapy for acute myeloid leukemia

Abstract

Several chromosomal abnormalities and gene mutations involved in the onset and recurrence of acute myeloid leukemia (AML) were discovered with the recent progress of genome analysis technology. The founding not only have clinical application as prognostic factors and minimal residual disease markers but also contribute to the novel molecular-targeted drug development. Many new drugs such as first-generation FLT3 inhibitor, IDH1/2 inhibitor, and BCL2 inhibitor have been developed in Europe and the United States. In addition, second-generation FLT3 inhibitors, gilteritinib and quizartinib, were developed in Japan, and the treatment outcome of AML has been improved. However, a large disparity in the drug availability remains between Europe and the United States and Japan. As a result, treatment guidelines in Europe and the United States cannot be applied to the practical use in Japan. In this paper, molecular-targeted drug treatment by gene diagnosis will be considered for AML in Japan, and the future paradigm shift of gene diagnosis and treatment of AML will be outlined.