Advances in targeted treatment of acute myeloid leukemia

Abstract

Chemotherapy is still the main treatment of acute myeloid leukemia (AML) for the moment. The successful use of imatinib in chronic myeloid leukemia has accelerated the study of targeted therapy. With the development of next generation sequencing, researchers have a complete understanding of AML gene mutation profile. Based on that, scientists have tried to develop small molecular targeted drugs. There is no new drug for AML approved for several decades, until the United States Food and Drug Administration (FDA) approved four drugs for AML in 2017. So 2017 can be considered as the year of AML. These new drugs have been recommended in the National Comprehensive Cancer Network (NCCN) guidelines (2018) and have become the first line treatment of AML. In addition to the four approved new drugs, many drugs are in clinical trials or in preclinical stage. When more new drugs are approved in the future, the treatment of AML will truly enter the era of targeted therapy or precision therapy. This article summarizes the AML targeting drugs that have been approved and might be approved in the future, mainly including Fms-like tyrosine kinase (FLT)3 inhibitors, isocitrate dehydrogenase (IDH) inhibitors, B-cell lymphoma (BCL)2 inhibitors, immunotoxins and so on. Key words: Leukemia, myeloid, acute; Molecular targeted therapy; FLT3 inhibitors; IDH inhibitors; BCL2 inhibitors; GO; SGN-CD33A