Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33 CAR T) in Children and Young Adults with Relapsed/Refractory Acute Myeloid Leukemia

Pediatric Blood Cancer
Steven Devine, MD
Be The Match Foundation


While a first remission is possible for pediatrics patients with acute myeloid leukemia (AML), only 60% will remain in long-term remission despite intensive therapy and allogeneic stem cell transplant (stem cell transplant from a donor). Thus, there is a need to increase remission rates, decrease relapse, and to improve overall survival. CD33 is a cellular protein expressed on >80% of AML cells and as a result represents a potential target. The investigators will utilize CD33-redirected (targeted) CAR T-cells to combat AML. CAR T-cell therapy is when a patient’s own T-cells (white blood cells that kill cancer cells) are collected via blood draw, then engineered to target specific cancer cells, multiplied, and infused back into the patient. This phase I/II study aims to determine the maximum tolerated dose in the phase I portion and the percentage of patients who achieve remission in the phase II portion of the study.

Trial Registration: Identifier: NCT03971799