National coverage analysis of CAR-T therapies — policy, evidence, and payment

New England Journal of Medicine

15 August 2018 - In December 2017, the Boston radio station WBUR chronicled the case of the first patient at Massachusetts General Hospital to receive axicabtagene ciloleucel (Yescarta, Gilead) outside a clinical trial. 

The chimeric antigen receptor T cells (CAR-Ts) were made from 71-year-old Barbara Kearney’s own T cells, which after genetic modification ex vivo were turned on to target the CD19 receptor. CD19 is a commonly expressed surface antigen on B cells, including those of Ms. Kearney’s non-Hodgkin’s lymphoma, and also inhabits the surface of some other normal cells.

In a trial involving 101 patients with non-Hodgkin’s lymphoma, axicabtagene ciloleucel produced complete remission in 51, which led to its approval by the FDA. Tisagenlecleucel (Kymriah, Novartis), another anti-CD19 CAR-T therapy, led to complete responses in 22 of 69 patients (32%). Juno Therapeutics also has a development program for anti–CD19 CAR-T therapies.

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Michael Wonder

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Michael Wonder