Current and future therapies for acute myeloblastic leukemia

Abstract

The management of acute myeloid leukemia (AML) has undergone a remarkable transformation over the past decade, as advances in genomic profiling and drug development have expanded therapeutic options and enabled increasingly personalized treatment approaches. This review summarizes the evolving therapeutic landscape of AML, including current intensive and lower-intensity treatment backbones, approved molecularly targeted therapies, and emerging investigational strategies. We discuss the role of intensive cytarabine plus anthracycline-based regimens and venetoclax-based lower-intensity approaches, as well as targeted therapies directed against FLT3, IDH1/2, NPM1, and KMT2A-rearranged AML. We further examine efforts to optimize existing treatment paradigms through the incorporation of venetoclax into intensive chemotherapy, rational targeted combinations, and novel triplet regimens, in addition to the emerging use of highly active lower-intensity therapies in younger fit patients. Finally, we highlight promising future directions in AML, including therapies targeting RAS/MAPK signaling, emerging approaches for TP53-mutated disease, and immunotherapeutic strategies. AML treatment is rapidly evolving from broadly applied chemotherapy-based approaches toward increasingly molecularly informed and individualized therapeutic strategies. Continued advances in molecular diagnostics, targeted therapies, and synergistic combination regimens have the potential to further increase remission durability, reduce relapses, and improve long-term outcomes for patients with AML.