Rev­o­lu­tion­ary gene ther­apy ap­proved

Irish Examiner - - World News -

US of­fi­cials have ap­proved the first treat­ment that ge­net­i­cally en­gi­neers pa­tients’ own blood cells to seek and de­stroy a child­hood leukaemia.

The CAR-T cell treat­ment de­vel­oped by No­var­tis and the Uni­ver­sity of Penn­syl­va­nia is the first type of gene ther­apy to hit the US mar­ket — and one in a pow­er­ful but ex­pen­sive wave of cus­tom-made “liv­ing drugs” be­ing tested against blood can­cers and some other tu­mours.

The Food and Drug Ad­min­is­tra­tion called the ap­proval his­toric.

“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 had been close to death, but now is can­cer-free for five years and count­ing. That’s enor­mously ex­cit­ing.”

CAR-T treat­ment uses gene ther­apy tech­niques not to fix dis­ease-caus­ing genes, but to “turbo-charge” T cells, im­mune sys­tem “sol­diers” that can­cer can of­ten evade. Re­searchers fil­ter the cells from a pa­tient’s blood, re­pro­gramme them with a “chimeric anti­gen re­cep­tor” that tar­gets can­cer, and grow hun­dreds of mil­lions of copies.

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