Breakthroughs of CAR T-cell therapy in acute myeloid leukemia: updates from ASH 2024

While chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment landscape for lymphoid malignancies, its greatest challenge remains in the treatment of acute myeloid leukemia (AML). Its success in AML has been limited by the ideal target antigen, myelosuppression, and immunosuppressive leukemia microenvironment. The 2024 ASH Meeting highlighted several cutting-edge advancements in AML-directed CAR T therapies, including clinical trials targeting CD33, CD123, CLL1, CD19, and IL1RAP, as well as novel engineering strategies such as dual-targeting CARs, inhibitory CAR designs, and genome-editing approaches to enhance safety and efficacy. Here, we summarize key findings from both clinical and preclinical studies, offering insights into the evolving landscape of CAR T-cell therapy for AML.