The beginning of a new therapeutic era in acute myeloid leukemia.

Abstract

In the field of AML, the early 2000s were shaped by the advent of novel molecular biology technologies including high‐throughput sequencing that improved prognostic classification, response evaluation through the quantification of minimal residual disease, and the launch of research on targeted therapies. Our knowledge of leukemogenesis, AML genetic diversity, gene‐gene interactions, clonal evolution, and treatment response assessment has also greatly improved. New classifications based on chromosomal abnormalities and gene mutations are now integrated on a routine basis. These considerable efforts contributed to the discovery and development of promising drugs which specifically target gene mutations, apoptotic pathways and cell surface antigens as well as reformulate classical cytotoxic agents. In less than 2 years, nine novels drugs have been approved for the treatment of AML patients, and many others are being intensively investigated, in particular immune therapies. There are now numerous clinical research opportunities offered to clinicians, thanks to these new treatment options. We are only at the start of a new era which should see major disruptions in the way we understand, treat, and monitor patients with AML.