FDA ap­proves break­through drug

Albuquerque Journal - - NEWS - AS­SO­CI­ATED PRESS

WASH­ING­TON — Open­ing a new era in can­cer care, U.S. health of­fi­cials on Wed­nes­day ap­proved a break­through treat­ment that ge­net­i­cally en­gi­neers pa­tients’ own blood cells into an army of as­sas­sins to seek and de­stroy child­hood leukemia.

The Food and Drug Ad­min­is­tra­tion called the ap­proval his­toric, the first gene ther­apy to hit the U.S. mar­ket. Made from scratch for ev­ery pa­tient, it’s one of a wave of “liv­ing drugs” un­der de­vel­op­ment to fight ad­di­tional blood can­cers and other tu­mors, too.

No­var­tis Phar­ma­ceu­ti­cals set the price for its one-time in­fu­sion of so-called “CART cells” at $475,000, but the com­pany said there would be no charge for pa­tients who don’t re­spond within a month.

“This is a brand new way of treat­ing can­cer,” said Dr. Stephan Grupp of Chil­dren’s Hos­pi­tal of Philadel­phia, who treated the first child with CAR-T cell ther­apy — a girl who’d been near death but now is can­cer­free for five years and count­ing. “That’s enor­mously ex­cit­ing.”

CAR-T treat­ment uses gene ther­apy tech­niques not to fix dis­ease-caus­ing genes but to tur­bocharge T cells, im­mune sys­tem sol­diers that can­cer too often can evade. Re­searchers fil­ter those cells from a pa­tient’s blood, re­pro­gram them to har­bor a “chimeric anti­gen re­cep­tor” or CAR that ze­roes in on can­cer, and grow hun­dreds of mil­lions of copies.

It’s a com­pletely dif­fer­ent way to har­ness the im­mune sys­tem than pop­u­lar im­munother­apy drugs called “check­point in­hibitors” that treat a va­ri­ety of can­cers by help­ing the body’s nat­u­ral T cells bet­ter spot tu­mors.

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