FDA ad­vis­ers en­dorse gene ther­apy treat­ment

San Francisco Chronicle Late Edition - - OPINION - By Matthew Perrone Matthew Perrone is an As­so­ci­ated Press writer.

SIL­VER SPRING, Md. — A po­ten­tially ground­break­ing treat­ment for a rare form of blind­ness moved one step closer to U.S. ap­proval Thurs­day, as fed­eral health ad­vis­ers en­dorsed the ex­per­i­men­tal gene ther­apy for pa­tients with an in­her­ited con­di­tion that grad­u­ally de­stroys eye­sight.

The panel ex­perts to the Food and Drug Ad­min­is­tra­tion voted unan­i­mously in fa­vor of Spark Ther­a­peu­tics’ in­jectable ther­apy, which aims to im­prove vi­sion by re­plac­ing a de­fec­tive gene needed to process light.

The vote amounts to a rec­om­men­da­tion to ap­prove the ther­apy. The FDA has un­til mid-Jan­uary to make its de­ci­sion and does not have to fol­low the panel’s rec­om­men­da­tion, though it of­ten does.

If ap­proved, Lux­turna would be the first gene ther­apy in the U.S. for an in­her­ited dis­ease and the first in which a cor­rec­tive gene is given di­rectly to pa­tients. Though the ther­apy from Spark Ther­a­peu­tics tar­gets a small group of pa­tients — about 2,000 in the U.S. — ex­perts say it could pave the way for other ge­netic treat­ments for a va­ri­ety of in­her­ited con­di­tions.

Pan­elists de­bated sev­eral specifics of the treat­ment, split­ting on whether to re­quire a min­i­mum age for treat­ment; the com­pany says the drug is in­tended for those 3 years and older. Pan­elists con­cluded that the sci­ence be­hind the drug — based on re­search from the Chil­dren’s Hospi­tal of Philadel­phia — was re­mark­ably strong with few se­ri­ous side ef­fects.

Sev­eral pa­tients at­tended the meet­ing to urge pan­elists to sup­port the drug, re­lat­ing ex­pe­ri­ences of see­ing snow, stars and the moon for the first time. In most cases, the pa­tients’ travel ex­penses were paid for by the drug’s de­vel­oper.

Kate­lyn Corey, 24, de­scribed her life be­fore the treat­ment as “a black-and-white film.”

“Within days of the first treat­ment I could see vi­brant colors again,” Corey said. “I could see the clock tower of Philadel­phia City Hall at night when mere days be­fore I thought it was the moon.”

Ash­ley Carper, a mother of two chil­dren with the dis­or­der, said treat­ment has al­lowed her 11-year-old son Cole to read large-print books and write his own home­work. Pre­vi­ously, he could only read with Braille. Cole said the im­prove­ments ex­tend beyond school­work.

“I can stay out later when my friends are out­side play­ing and now I feel like part of the group,” he said. “My vi­sion is not per­fect, but what I do have is still re­ally im­por­tant to me.”

Re­searchers tested the treat­ment by record­ing pa­tients’ abil­ity to com­plete an ob­sta­cle course at vary­ing lev­els of light, sim­u­lat­ing real-world con­di­tions like nav­i­gat­ing a dark stair­well. A hall­mark of the in­her­ited dis­or­der is dif­fi­culty see­ing at night. Pa­tients with the mu­ta­tion gen­er­ally start los­ing their sight be­fore age 18, al­most al­ways pro­gress­ing to to­tal blind­ness.

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