The Food and Drug Administration on Wednesday approved a new therapy to treat leukemia in kids and young adults—a decision whose importance is as much symbolic as it is practical.
Kymriah, from the Swiss pharmaceutical company Novartis, is a cancer therapy that represents several things at once: a game-changing way to treat cancer through genetic engineering, a novel paradigm for the biotech business, and the latest turn in the debate over just how astronomically expensive a life-saving therapy can be.
Kymriah is strikingly effective for young patients with acute lymphoblastic leukemia, or ALL, but it is far more involved than taking a pill or getting an infusion. It requires inserting a human-designed gene into a patient’s own T cells so they recognize and ferociously attack cancer cells. Researchers began modifying T cells for patients in the 1990s—and now the technology called CAR T-cell therapy is finally ready for prime time in treating cancer.
(To clear up any possible confusion about terminology: The FDA and others have chosen to call CAR T-cell therapy a form of gene therapy—and thus deemed it the first gene therapy to be approved in the United States.
The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.