Gene Therapy May Benefit Patients With Cystic Fibrosis

By Dr Deepu

Gene Therapy May Benefit Patients With Cystic Fibrosis

The AP (7/3, Cheng) reported that physicians “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.” However, they “called the results ‘modest’ and say there must be major improvements before offering the treatment more widely.” The findings (pdf) were published in Lancet Respiratory Medicine.

        Bloomberg News (7/3, Kitamura) reports that although “the therapy yielded a modest benefit in some patients, there was wide variability among the 136 people at least 12 years old who received monthly doses or a placebo for a year.” Investigators found that after one “year of treatment, in the 62 patients who received the gene therapy, lung function was an average of 3.7 percent greater than in” those given a placebo.

        Also covering the story were Reuters (7/3, Kelland), BBC News (7/4, Ghosh), HealthDay (7/3, Dallas), MedPage Today (7/3, Walker), theHuffington Post (UK) (7/3), the Telegraph (UK)(7/4, Knapton), and The Guardian (UK) (7/3)