Chasing cancer with chimeric antigen receptor therapy.

Many attempts to use genetically modified T cells to halt tumor progression have been met with disappointment and significant challenges in the successful application within human patients. Porter et al., however, describe the use of genetically modified lymphocytes bearing a chimeric antigen receptor that bypasses many of the common limitations of adoptive lymphocyte therapy. Through incorporation of a costimulatory domain within the chimeric antigen receptor, the investigators engineered lymphocytes with significantly higher tumor rejection activity and demonstrated significant expansion and prolonged survival after in vivo transfer to a single patient who showed a complete regression of refractory chronic lymphoid leukemia. This recent success in using genetically modified T cells to kill chronic lymphoid leukemia tumor cells is an encouraging advancement in the development of specific and targeted immune-based therapies against cancer.