How close are we to CAR T-cell therapy for AML?

Chimeric antigen receptor (CAR) T-cell therapy for acute myeloid leukemia (AML) has thus far been elusive, in part owing to the absence of truly AML-specific surface antigens, making AML difficult to target. However, progress has been made toward the use of CAR T-cell therapy in this disease, prompting the topic of this paper. Discussion and clinical examples of potential solutions to creating a safe and effective CAR T cell for AML include: (1) Decreasing the potency or activity of CAR T cells to enhance the therapeutic window; (2) Using transient or depletable CAR T cells as part of pre-transplant conditioning; and (3) Using a gene-edited allogeneic donor hematopoietic stem cell transplant in order to allow safe and protracted anti-AML CAR T-cell function.