Break­through ther­apy for child leukemia OK’d

Austin American-Statesman - - FRONT PAGE - By Lauran Neergaard

Open­ing a new era in can­cer care, U.S. health of­fi­cials 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 “CAR-T cells” at $475,000, but said there would be no charge for pa­tients who didn’t show a re­sponse 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 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 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. Re­turned to the pa­tient, the revved-up cells can con­tinue mul­ti­ply­ing to fight dis­ease for months or years.

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. CAR-T cell ther­apy gives pa­tients stronger T cells to do that job.

“We’re en­ter­ing a new fron­tier in med­i­cal in­no­va­tion with the abil­ity to re­pro­gram a pa­tient’s own cells to at­tack a deadly can­cer,” said FDA Com­mis­sioner Scott Got­tlieb.

The first CAR-T ver­sion, de­vel­oped by No­var­tis and the Univer­sity of Penn­syl­va­nia, is ap­proved for use by sev­eral hun­dred pa­tients a year who are des­per­ately ill with acute lym­phoblas­tic leukemia, or ALL. It strikes more than 3,000 chil­dren and young adults in the U.S. each year and while most sur­vive, about 15 per­cent re­lapse de­spite to­day’s best treat­ments.

In a key study of 63 ad­vanced pa­tients, 83 per­cent went into re­mis­sion soon after re­ceiv­ing the CAR-T cells. Im­por­tantly, 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, one of the study sites. “I wouldn’t say we know for sure how many will be cured yet by this ther­apy. There cer­tainly is a hope” that some will be.

Most pa­tients suf­fered side ef­fects that can be gru­el­ing, even life-threat­en­ing. An im­mune over­re­ac­tion called “cy­tokine re­lease syn­drome” can trig­ger high fevers, plum­met­ing blood pres­sure and in se­vere cases or­gan dam­age, side ef­fects that re­quire so­phis­ti­cated care to help pa­tients with­out block­ing the can­cer at­tack. The FDA des­ig­nated a treat­ment for those side ef­fects Wed­nes­day.

“This is re­mark­able tech­nol­ogy,” said Dr. Mikkael Sek­eres of the Amer­i­can So­ci­ety of He­ma­tol­ogy. But, he cau­tioned that CAR-T “isn’t a panacea.”

Among con­cerns, some­times leukemia can de­velop re­sis­tance, and some­times pa­tients worsen while wait­ing for their new cells, said Sek­eres, who di­rects the Cleve­land Clinic’s leukemia pro­gram and wasn’t in­volved with CAR-T test­ing.

“Un­for­tu­nately, leukemia grows so rapidly that it can evade even the smartest of our tech­nolo­gies,” he added.

To bet­ter en­sure pa­tient safety, the FDA is re­quir­ing No­var­tis to of­fer CAR-T ther­apy only through med­i­cal cen­ters spe­cially trained and cer­ti­fied to han­dle the com­pli­cated treat­ment. No­var­tis ex­pects to have 32 cen­ters around the coun­try, mostly in large cities, run­ning by year’s end, with the first 20 of­fer­ing care within the next month.

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. The $475,000 price tag doesn’t in­clude the cost of needed hos­pi­tal­iza­tions, travel to a cer­ti­fied hos­pi­tal and other ex­penses.

On a con­fer­ence call Wed­nes­day, No­var­tis ex­ec­u­tives said the com­pany is work­ing with the Med­i­caid pro­gram and pri­vate in­sur­ers and ex­pects broad cov­er­age, and will of­fer some fi­nan­cial as­sis­tance with such things as co-pay and travel costs. But they didn’t prom­ise all pa­tients would be able to get the ther­apy.

For some pa­tients, the new CAR-T ther­apy might re­place bone mar­row trans­plants that cost more than half a mil­lion dol­lars, noted Grupp, who led the No­var­tis study.


Emily White­head is seen in May, five years after she be­came the first child­hood leukemia pa­tient in the world to re­ceive ex­per­i­men­tal CAR-T treat­ment that sent her can­cer into re­mis­sion.

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