U.S. clears first ‘liv­ing drug’ for tough child­hood leukemia

The Hamilton Spectator - - HEALTH - LAURAN NEERGAARD

The U.S. Food and Drug Ad­min­is­tra­tion on Wed­nes­day ap­proved the first treat­ment that ge­net­i­cally en­gi­neers pa­tients’ own blood cells into an army of as­sas­sins to seek and de­stroy child­hood leukemia.

The CAR-T cell treat­ment de­vel­oped by No­var­tis and the Univer­sity of Penn­syl­va­nia is the first type of gene ther­apy to hit the U.S. market — and one in a pow­er­ful but ex­pen­sive wave of cus­tom-made “liv­ing drugs” be­ing tested against blood can­cers and some other tu­mours, too. The FDA 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’d been near 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 dis­ease­caus­ing 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 and grow hun­dreds of mil­lions of copies. Re­turned to the pa­tient, the revved-up cells can con­tinue mul­ti­ply­ing to fight dis­ease for months or years.

No­var­tis didn’t im­me­di­ately dis­close the ther­apy’s price, but it is ex­pected to cost hun­dreds of thou­sands of dol­lars. It’s made from scratch for ev­ery pa­tient.

This first use of CAR-T ther­apy is aimed at pa­tients des­per­ately ill with a com­mon pe­di­atric can­cer — acute lym­phoblas­tic leukemia — that strikes more than 3,000 chil­dren and young adults in the U.S. each year. While most sur­vive, about 15 per cent re­lapse de­spite to­day’s best treat­ments, and their prog­no­sis is bleak.

In a key study of 63 ad­vanced pa­tients, 83 per cent went into re­mis­sion. It’s not clear how long that ben­e­fit lasts: Some pa­tients did re­lapse months later. The oth­ers still are be­ing tracked to see how they fare long-term.

Still, “a far higher per­cent­age of pa­tients go into re­mis­sion with this ther­apy than any­thing else we’ve seen to date with re­lapsed leukemia,” said Dr. Ted Laetsch of the Univer­sity of Texas South­west­ern Med­i­cal Cen­ter.

Most pa­tients suf­fered side-ef­fects that can be gru­elling, even lifethreat­en­ing. An im­mune over­re­ac­tion called “cy­tokine re­lease syn­drome” can trig­ger high fevers, plum­met­ing blood pres­sure and in se­vere cases or­gan dam­age, re­quir­ing spe­cial care to tamp down those symp­toms with­out block­ing the can­cer at­tack. Also Wed­nes­day, the FDA des­ig­nated a treat­ment for those side-ef­fects.

Ini­tially, No­var­tis’ CAR-T ver­sion — to be sold un­der the brand name Kym­riah — will be avail­able only through cer­tain med­i­cal cen­tres spe­cially trained to han­dle the so­phis­ti­cated ther­apy and its side­ef­fects. Pa­tients’ col­lected im­mune cells will be frozen and shipped to a No­var­tis fac­tory in New Jersey that cre­ates each dose, a process the com­pany says should take about three weeks.

Sci­en­tists around the coun­try also are try­ing to make CAR-T ther­a­pies that could fight more com­mon solid tu­mours such as brain, breast or pan­cre­atic can­cers — a harder next step.

Newspapers in English

Newspapers from Canada

© PressReader. All rights reserved.