Acute myeloid leukemia: advancing clinical trials and promising therapeutics

ABSTRACT

Recent progress in understanding the biology of acute myeloid leukemia (AML) and the identification of targetable driver mutations, leukemia specific antigens and signal transduction pathways has ushered in a new era of therapy. In many circumstances the response rates with such targeted or antibody-based therapies are superior to those achieved with standard therapy and with decreased toxicity. In this review we discuss novel therapies in AML with a focus on two major areas of unmet need: (1) single agent and combination strategies to improve frontline therapy in elderly patients with AML and (2) molecularly targeted therapies in the frontline and salvage setting in all patients with AML.

KEYWORDS:

• AML
• clinical trials
• molecular therapy
• monoclonal antibodies
• multikinase inhibitors

Financial and competing interests disclosure

This manuscript was supported in part by the MD Anderson Cancer Centre Leukaemia Support Grant (CCSG) CA016672 and generous philanthropic contributions to the MD Anderson Moon Shots Program. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.