Novel therapies for AML: a round-up for clinicians

Introduction: Acute myeloid leukemia (AML) is a deadly disease associated with poor outcomes. For over four decades, therapeutic options for AML were limited to high dose cytotoxic chemotherapy. Scientific breakthroughs have not only enhanced our understanding of the molecular underpinnings of this disease but also resulted in the development of several targeted therapies with superior efficacy and lesser toxicities than conventional chemotherapy. The FDA approval of small molecule inhibitors for specific AML subsets highlights the importance of genetic and molecular profiling to optimally personalize AML therapy in the modern era. Areas covered: In this article, we review the medical literature from PubMed on recent FDA approved drugs for AML by their mechanism of action: small molecule inhibitors, antibody-drug conjugate, cytotoxic, and epigenetic agents. We describe how to incorporate these agents into the current treatment paradigm for specific AML patients. Expert opinion: Knowing the molecular characteristics of patients with AML is of utmost importance to plan the best management. There are promising drugs targeting leukemogenesis by various mechanisms. It is important to consider clinical trial options for patients if and when available. We have provided a brief overview of the most promising agents on the horizon for AML therapy.