Novel and Investigational Therapies in Acute Myeloid Leukemia

Abstract

Acute myeloid leukemia (AML) is classified into four broad categories in the revised World Health Organization classification for myeloid neoplasms; AML with recurrent genetic abnormalities, AML with myelodysplasia-related changes, therapy-related myeloid neoplasms, and AML not otherwise specified. With better understanding of the disease biology, many novel agents have been developed and are now incorporated in the treatment paradigm for AML. We summarize in this chapter the molecular landscape of AML, novel therapies that have received regulatory approval, as well as selected investigational agents that are in early stages of development (first-in human trials) and focus on those in later stages of development. Each section will emphasize selected investigational agents based on their mechanism of action or targeted pathway.