Minimal Effect of Nonviral Gene Therapy for Cystic Fibrosis

Nebulized nonviral CFTR gene therapy had a modest effect on stabilizing lung function after 1 year.

Chronic lung disease is the major complication of cystic fibrosis. Researchers evaluated nonviral CFTR gene therapy in a randomized, double-blind, placebo-controlled phase 2b trial at 18 sites in the U.K. During a 1-year period, 256 patients aged 12 years and older with any combination of CFTR mutations received saline or a plasmid DNA-liposome complex (pGM169/GL67A) by nebulization monthly.

The relative change in forced expiratory volume in 1 second from before to after therapy was −4.0% in the placebo group compared with −0.4% in the treatment group (P=0.046). There was also a significant treatment effect for forced vital capacity and gas trapping. Treatment effects did not differ significantly with sex, age, or CFTR mutation. Incidence of…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

Minimal Effect of Nonviral Gene Therapy for Cystic Fibrosis

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Reviewing Author