“What's in a name?” CAR‐T Gene Therapy

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 (ALL). This ex vivo genetic manipulation of a patient's own T-lymphocytes marks a major success in translational re...

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Bibliographic Details
Published in:The Hastings Center report 2017-11, Vol.47 (6), p.inside back cover-inside back cover
Main Author: Kodish, Eric
Format: Article
Language:English
Subjects:
Bioethics
Gene therapy
Genetic Therapy - methods
Humans
Jargon
Medical ethics
Medical research
Precursor Cell Lymphoblastic Leukemia-Lymphoma - drug therapy
Receptors, Antigen, T-Cell - therapeutic use
Terminology as Topic
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Summary: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 (ALL). This ex vivo genetic manipulation of a patient's own T-lymphocytes marks a major success in translational research, a landmark in cancer history, and a sign of good things to come in personalized medicine. Unfortunately, since CAR-T infusion is often associated with a life-threatening cytokine release syndrome, the approval was limited to a small number of centers with the expertise to manage this toxicity. The question of what to name this activity reflects a longstanding debate in research ethics. Is it ever correct to talk about “therapeutic research?” What are the risks of promoting false hopes? During the era in which many aspire to build a learning health care system, perhaps one size does not fit all. High-risk, high-reward research like that which led to the development of CAR-T therapy seems to me to be categorically different from low-risk population-based research that leverages the electronic medical record and the power of big data. Thoughtful attention to such distinctions and to key transition points like FDA approval is the foundation for thinking about research, innovation, progress, and treatment in biomedicine. So, as long as there is a commitment to quality informed consent processes, count me in as comfortable talking about “therapeutic research.”
ISSN:0093-0334
1552-146X
DOI:10.1002/hast.786