In the past few years, we have finally begun to realize the promise of co-opting a patient's immune system as a form of cancer therapy. An exciting form of immunotherapy involves the genetic retargeting of T cells with chimeric antigen receptors (CARs). These engineered receptors usually contain a monoclonal antibody-derived single-chain variable fragment (scFv) that confers specific binding to a tumor antigen. At the 2013 annual meeting of the American Society of Hematology, three groups reported remarkable successes in clinical trials targeting chemotherapy-refractory, relapsed B-cell acute lymphoblastic leukemia (B-ALL) using CD19-targeted CAR T cells. Complete remission (CR) rates-which would be expected to be <40% with chemotherapy alone-ranged from 70 to 90% in approximately 50 patients among the three trials.
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