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
• perceptions
• survey
• public
• patient

Acknowledgments

The authors thank all the participants that took part in the study, the CF organisations that helped distribute the questionnaire through their online channels, and Dr Ivana Osenk for her assistance with data collection.

Author contributions

I Prichard conceived the study, and designed the questionnaires with input from M Donnelley and D Parsons. I Prichard performed statistical analysis. M Donnelley and I Prichard wrote the manuscript. All authors edited and approved the final manuscript.

Declaration of interest

M Donnelley declares funding from CF Australia via the Australian Cystic Fibrosis Research Trust. Robinson Research Institute Career Development Fellowship, NHMRC GNT1160011 and Cystic Fibrosis Foundation grant DONNEL21GO. D Parsons declares funding from CF Australia via the Australian Cystic Fibrosis Research Trust. NHMRC GNT1160011 and Cystic Fibrosis Foundation grant DONNEL21GO. I Prichard declares funding from CF Australia via the Australian Cystic Fibrosis Research Trust. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Ethics approval

The study was approved by the Flinders University Human Research Ethics Committee (SBRECProject 7425). Completion of the survey was considered as informed consent.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14712598.2022.2150544

Additional information

Funding

This paper was funded by CF Australia via the Australian Cystic Fibrosis Research Trust, Cystic Fibrosis Foundation (Grant: DONNEL21GO) and the National Health and Medical Research Council (Department of Health, Australian Government; grant: GNT1160011).