Despite FDA approval of nine new drugs for patients with acute myeloid leukemia (AML) in the United States over the last 4 years, AML remains a major area of unmet medical need among hematologic malignancies. In this review, we discuss the development of promising new molecular targeted approaches for AML, including menin inhibition, novel IDH1/2 inhibitors, and preclinical means to target TET2, ASXL1, and RNA splicing factor mutations. In addition, we review progress in immune targeting of AML through anti-CD47, anti-SIRPα, and anti-TIM-3 antibodies; bispecific and trispecific antibodies; and new cellular therapies in development for AML.
Keywords: ASXL1; IDH1; IDH2; RNA splicing; TET2; acute myeloid leukemia; menin; myelodysplastic syndromes.
© 2022 Bewersdorf and Abdel-Wahab; Published by Cold Spring Harbor Laboratory Press.