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“What’s in a name?” CAR-T Gene Therapy

During a four-year stint I finished in 2010 on the National Institutes of Health Recombinant DNA Advisory Committee, the committee was repeatedly reminded that we should not speak of “gene therapy.” “Gene transfer research” (the preferred terminology) was resurgent in those years as the science made progress in safety and efficacy, and some time had passed since the tragic death of Jesse Gelsinger as a direct result of his participation in such a study. But by no means was the language of treatment considered appropriate—at least according to the powers that be at NIH. I suspect that investigators and patients who were considering enrollment on gene-transfer clinical trials might have had a different opinion. The former were attempting to develop treatments for a variety of diseases, and the latter wanted to benefit from the clinical science. Fast forward to August of 2017, when the Food and Drug Administration approved chimeric antigen receptor T cell (CAR-T) therapy for relapsed childhood acute lymphoblastic leukemia.

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