Researchers at the Center for Childhood Cancer Research (CCCR), including Shannon L. Maude, MD, PhD and Stephan Grupp, MD, PhD, and in partnership with the University of Pennsylvania and Novartis, are developing and evaluating an exciting new immunotherapy called chimeric antigen receptor (CAR) T cells to treat children with acute lymphoblastic leukemia (ALL).
T-cells can be engineered to target specific antigens on the surface of leukemic cells. After infusion into patients, CAR T-cells bind to tumor target antigens, which in turn induces an immune effector cell cytotoxic response that kills malignant cancer cells.
Remission rates exceeding 90 percent have been seen in a phase I/IIa trial of CAR T cells targeting CD19, but some patients relapse. To overcome one type of relapse, a Phase 1 trial, led by Dr. Maude, is underway at the CCCR to evaluate the safety and efficacy of humanized CAR T cells targeting CD19 (CART19) as a treatment for pediatric patients with chemotherapy resistant or refractory CD19+ ALL and lymphoma.
Another clinical study being conducted at the CCCR is assessing the safety and efficacy of CAR T-cells targeting CD22 (CART22) in children who relapse after CART19 treatment with CD19 negative leukemias. Like CD19, CD22 is another antigen that is expressed on ALL cancer cells.
Both studies will help to better define the utility of CAR T-cell immunotherapy as a treatment for high-risk pediatric leukemias and pediatric lymphomas.