Introduction: Long-term outcome of patients with acute myeloid leukemia (AML) remains dismal, especially for those with high-risk disease or who are refractory to conventional therapy. CAR T-cell therapy provides unique opportunity to improve outcome by specifically targeting leukemia cells through genetically engineered T cells.
Areas covered: We summarize the progress of CAR T-cells therapy in AML. We examine its shortcomings in AML therapy and the strategies that are being implemented to improve its safety and effectiveness. PubMed Central, ClinicalTrials.gov, and ASH annual meeting abstracts were searched. Search terms used to identify clinical trials were 'CAR T-cells in AML' OR CAR T-cells in leukemia". Relevant clinical trials and CAR T-cell research data were reviewed from June 2009 till July 2021.
Expert opinion: CAR T-cell therapy has shown promise as a novel therapy, but there are number of barriers to overcome to achieve its full therapeutic potential in AML. Targeting leukemia-specific antigen such as CLL1, to avoid myelotoxicity, incorporating checkpoint inhibitors to overcome leukemia-induced immunosuppression and allogenic CAR T cells to increase accessibility to patients with proliferative disease are among the strategies that are being explored to make CAR T cell a successful immunotherapy for patient with AML.
Keywords: CAR T-cells; CD123/CD33 CAR T-cells; CLL1 CAR T-cells; acute myeloid leukemia; allogeneic CAR; checkpoint inhibitors; gene editing; myelotoxicity secondary to AML CAR.