Perceptions of airway gene therapy for cystic fibrosis

Abstract

Background: Airway gene therapy could produce lasting benefit for cystic fibrosis (CF) lung disease, however patient and public support is critical for successful adoption.

Research design and methods: Two separate quantitative online surveys were conducted to examine perceptions towards airway gene therapy for CF among people with CF, their families, and members of the public. Data was collected from a total of 213 participants across both studies, with 43 having a diagnosis of CF, 122 having a family member with CF, and 135 knowing someone with CF.

Results: Participants in both studies displayed positive perceptions towards gene therapy and were supportive of involvement in CF gene therapy trials. Around 50% hoped gene therapy could provide a cure. In Study 1 gene therapy was the most important research area, but in Study 2 this was new daily drugs. Almost all thought gene therapy was still required even if modulators already improved quality of life.

Conclusion: The factors that influence acceptance, whether trials would be positively viewed, and whether individuals with CF are receptive to gene therapy, are essential to determine prior to clinical trials. Our findings indicate people have positive opinions about airway gene therapy for CF, but further education is vital.

Keywords: Cystic fibrosis; gene therapy; patient; perceptions; public; survey.