Gene ther­apy for cys­tic fi­bro­sis promis­ing for some

Not yet ready for wide use


Doc­tors who gave chil­dren with cys­tic fi­bro­sis a re­place­ment copy of a de­fec­tive gene say it ap­peared to slow the ex­pected de­cline of some pa­tients' lungs, but called the re­sults “mod­est” and say there must be ma­jor im­prove­ments be­fore of­fer­ing the treat­ment more widely.

Cys­tic fi­bro­sis is an in­her­ited con­di­tion that fills the lungs with mu­cus, mak­ing peo­ple sus­cep­ti­ble to in­fec­tions that can even­tu­ally de­stroy the lungs; the av­er­age life ex­pectancy is about 37 years. Af­ter sci­en­tists iden­ti­fied the ge­netic se­quence that causes cys­tic fi­bro­sis in 1989, many ex­perts hoped the dis­ease could be cured by re­plac­ing the prob­lem gene. But be­fore the new study, all at­tempts at such gene ther­apy - where a nor­mal ver­sion of the faulty gene is given to pa­tients - failed to show a ben­e­fit.

In the new study, doc­tors in Lon­don and Ed­in­burgh gave 78 chil­dren with cys­tic fi­bro­sis a monthly dose of gene ther­apy us­ing a spe­cial in­haler in the hos­pi­tal while 62 oth­ers got a placebo treat­ment. All of the par­tic­i­pants were older than 12.

Chil­dren who got the gene ther­apy showed about a four per cent bet­ter lung func­tion com­pared to those who got the placebo. But doc­tors said the per­ceived ben­e­fit was just a sta­bi­liza­tion of the lungs from de­te­ri­o­rat­ing fur­ther, rather than an ac­tual im­prove­ment. The re­sults could also have been sim­ply due to chance. Both groups were fol­lowed for one year.

“Our only hope was that some of the pa­tients, on some oc­ca­sions, might see some ben­e­fit,” said Dr. Eric Alton of Im­pe­rial Col­lege Lon­don, the study's lead au­thor. The re­search was pub- lished online Thurs­day in the jour­nal, Lancet Res­pi­ra­tory Medicine. Alton said that while some chil­dren re­ported feel­ing bet­ter, oth­ers didn't feel any dif­fer­ence.

Some ex­perts said the trial could spur re­lated gene ther­apy stud­ies.

“This is a long-term prob­lem and the so­lu­tion is go­ing to take time,” said Dr. Wil­liam Skach, vice-pres­i­dent for re­search at the Cys­tic Fi­bro­sis Foun­da­tion in the U.S., who was not in­volved in the study.

Skach de­scribed the re­sults as en­cour­ag­ing, but said sci­en­tists need to fig­ure out how to im­prove de­liv­ery of the needed gene to pa­tients to get bet­ter re­sults.

“That could trans­late into ex­tended lives for pa­tients,” he said. “If we re­ally want to im­pact on the dis­ease in a ma­jor way, we need to think about how to do this more ef­fec­tively.”

“This is a long-term prob­lem and the so­lu­tion is go­ing to take time.” Dr. Wil­liam Skach, vice-pres­i­dent for re­search, Cys­tic Fi­bro­sis Foun­da­tion in the U.S.

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