New data on risk vs ben­e­fit for po­tent CAR-T cancer drugs

Kuwait Times - - HEALTH & SCIENCE -

NEW YORK: A promis­ing but risky new group of cus­tom­ized cancer drugs will be in fo­cus this week­end at the an­nual meet­ing of the Amer­i­can So­ci­ety of Hema­tol­ogy (ASH), where clin­i­cal trial re­sults will help clar­ify their po­ten­tial for doc­tors and in­vestors.

Ex­per­i­men­tal chimeric anti­gen re­cep­tor Tcells, or CAR-Ts, are made by ge­net­i­cally al­ter­ing a pa­tients’ own T-cells in the lab to help the im­mune sys­tem find and kill cancer cells. The al­tered cells are then in­fused back into the pa­tient.

Early ex­cite­ment over the drugs has pro­pelled in­vestor in­ter­est in biotech Kite Pharma Inc, whose shares have tripled since a 2014 ini­tial pub­lic of­fer­ing, as well as ri­val Juno Ther­a­peu­tics Inc, whose ther­apy JCAR015 has gen­er­ated con­cerns af­ter five leukemia pa­tients died due to se­vere brain swelling. Juno shares now trade about 14 per­cent be­low their IPO price.

“Juno has dug them­selves into such a deep hole,” said Brad Lon­car, man­ager of the Lon­car Cancer Im­munother­apy ETF. “My guess is that they may drop the JCAR015 pro­gram.” He will be watch­ing closely to see whether data at ASH on an­other Juno can­di­date, JCAR017, shows sim­i­lar is­sues.

Data to be pre­sented

Data on CAR-T drugs from Kite and No­var­tis AG will also be pre­sented at the meet­ing. In early tri­als, CAR-Ts elim­i­nated all trace of leukemia and lym­phoma in 40 per­cent to 90 per­cent of pa­tients who had run out of other op­tions. But it is not yet clear how long those re­mis­sions will last. Also, the drugs can over­es­ti­mate the im­mune sys­tem, which can cause dan­ger­ous side ef­fects.

If they work safely, Wall Street ex­pects an­nual sales for CAR-T ther­a­pies in the bil­lions of dol­lars.

Shares of Blue­bird Bio Inc closed 14 per­cent higher on Thurs­day af­ter a small study showed that its CAR-T, bb2121, in­duced re­mis­sion in sev­eral pa­tients with ad­vanced mul­ti­ple myeloma with no wor­ri­some side ef­fects. The Blue­bird drug is be­ing de­vel­oped in part­ner­ship with Cel­gene Inc.

“The data look good, but these are very small tri­als,” said Les Funt­ley­der, health­care port­fo­lio man­ager at E Squared As­set Man­age­ment in New York, which does not cur­rently hold po­si­tions in CAR-T com­pa­nies. “We would def­i­nitely like to see larger sam­ple sizes and longer du­ra­tion.” He is also look­ing for clar­ity on pric­ing. Es­ti­mates have run into hun­dreds of thou­sands of dol­lars per treat­ment. It is not yet clear if pa­tients will need more than one treat­ment or whether that can even be done safely.

The CAR-T tech­nique is be­ing tried ini­tially against blood can­cers, which con­tain spe­cific pro­teins that can be dif­fer­en­ti­ated from nor­mal tis­sue, lim­it­ing a pos­si­ble im­mune sys­tem at­tack on healthy or­gans. Drugs like Juno’s JCAR015 tar­get a pro­tein called CD19, which is found on the sur­face of a type of white blood cell.

Juno is slated to present early-stage data at the ASH meet­ing in San Diego on JCAR017, which also tar­gets CD19. Juno Chief Ex­ec­u­tive Hans Bishop said JCAR017, and the sim­i­lar JCAR014, are safer than JCAR015 be­cause of a man­u­fac­tur­ing process that al­lows for ac­tive con­trol of the com­po­si­tion of the T-cells that make up the fi­nal prod­uct, said “We think when you get to the ex­tremes ... that is likely a source of vari­abil­ity,” he said.

No­var­tis plans to file next year for US Food and Drug Ad­min­is­tra­tion ap­proval for its drug, CTL019, in chil­dren with acute lym­phoblas­tic leukemia who have ex­hausted other op­tions, based on Phase 2 trial re­sults to be re­leased at ASH on Satur­day.

The study re­sults show “what it looks like when you roll this out to a bunch of dif­fer­ent cen­ters across the world,” said Dr Stephan Grupp, re­search di­rec­tor at Chil­dren’s Hospi­tal of Philadel­phia’s child­hood cancer cen­ter and the study’s lead in­ves­ti­ga­tor.

He said most pa­tients in the No­var­tis trial ex­pe­ri­enced a se­ri­ous side ef­fect known as cy­tokine re­lease syn­drome, or CRS, as well as neu­ro­log­i­cal tox­i­c­ity, in­clud­ing con­fu­sion and seizures, but all re­sponded to treat­ment for those side ef­fects and none had se­vere brain swelling.

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