For the first time, non-viral gene therapy for cystic fibrosis (CF) has had a beneficial effect on lung function (Alton et al., 2015).
Pre-clinical studies including plasmid engineering, selection of the best liposome formulation, and toxicology studies in mice and sheep supported a randomised, double-blind, placebo-controlled, phase 2b trial. CF patients received 5ml of either nebulised plasmidliposome complexes or 0·9% saline (placebo) every 28 days for 12 months.
A significant treatment effect was observed in the Active group versus Placebo after 12 months, based on in the primary outcome measure (FEV1; 3·7%, 95% CI 0·17·3; p=0·046), associated with a stabilisation of lung function.
The treatment was well-tolerated with no significant difference in treatment attributable adverse events between groups. These results indicate the potential to reduce deterioration of lung function in young CF sufferers and further clinical trials are planned to assess the formulation for transfer to clinical care.