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FDA panel recommends approval of first gene therapy in US

BMJ 2017; 358 doi: https://doi.org/10.1136/bmj.j3443 (Published 14 July 2017) Cite this as: BMJ 2017;358:j3443
  1. Owen Dyer
  1. Montreal

A 10 member expert panel convened by the US Food and Drug Administration has voted unanimously to recommend approval of a cancer treatment that could transform the survival prospects of young people with refractory leukemia, though at a steep price.

The Novartis treatment, called CTL019, or tisagenlecleucel, is a chimeric antigen receptor T cell (CAR-T) treatment, a class whose career in development has already earned them a name for astronomical cost, alarming side effects, and spectacular results.

In treatment with CTL019 T cells are extracted from a patient’s blood, frozen, and sent to a Novartis plant in New Jersey. There they are genetically modified to hunt and destroy cancer causing B cells, multiplied, refrozen, and shipped …

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