Journal
GENES & DEVELOPMENT
Volume 36, Issue 5-6, Pages 259-277Publisher
COLD SPRING HARBOR LAB PRESS, PUBLICATIONS DEPT
DOI: 10.1101/gad.349368.122
Keywords
ASXL1; acute myeloid leukemia; IDH1; IDH2; menin; myelodysplastic syndromes; RNA splicing; TET2
Categories
Funding
- Leukemia and Lymphoma Society
- National Institutes of Health (NIH) [R01 CA251138]
- NIH/National Cancer Institute [1P50 254838-01]
- Edward P. Evans MDS Foundation
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This review discusses the development of promising new molecular targeted approaches for AML, as well as progress in immune targeting of AML through various antibodies and cellular therapies. Despite FDA approval of new drugs for AML, it remains a major area of unmet medical need among hematologic malignancies.
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-SIRP alpha, and anti-TIM-3 antibodies; bispecific and trispecific antibodies; and new cellular therapies in development for AML.
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