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expert reaction to results from gene therapy trial for cystic fibrosis

Genetic disorders underlie many diseases, and gene therapies have the potential to alleviate the severity of some conditions or even cure them. Researchers publishing in the The Lancet Respiratory Medicine journal have detailed their use of a gene therapy to treat cystic fibrosis, reporting a modest and variable effect compared to placebo. These comments accompanied an SMC briefing.


Prof. Dominic, Wells, Professor in Translational Medicine, Royal Veterinary College, said:

“The new paper by Alton and colleagues describes the outcome of a large randomised double-blind placebo-controlled phase 2b clinical trial of a non-viral gene therapy for cystic fibrosis. This trial was based on a substantial body of work using experimental animals and was preceded by earlier phase 1 and 2a clinical trials. Although the efficiency of non-viral gene delivery is generally much lower than that achieved with viral vectors, immune responses and differences in cellular targeting can limit the clinical use of the latter. Despite the use of a non-viral gene delivery (plasmid and liposome combination), the authors report that monthly dosing for one year produced a significant although modest difference in the primary outcome measure, FEV1 (the forced expiratory volume in 1 second), suggesting a stabilisation of lung function in the treated group compared to the placebo. No clinically important adverse events attributable to treatment were observed. The authors are suitably cautious in avoiding over-interpreting their findings and rightly point out that this study will not lead directly to a clinical treatment. However, this important study lays a strong foundation for the development of more potent gene transfer systems for the treatment of cystic fibrosis, a severe life-limiting condition.”


Repeated nebulisation of non-viral CFTR gene therapy inpatients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial’ by Alton et al. published in The Lancet Respiratory Medicine on Friday 3rd July. 


Declared interests

Prof Dominic Wells: Member of the British Society for Gene and Cell Therapy, Member of the American Society for Gene and Cell Therapy. Also a member of the Scientific Advisory Board for Akashi Therapeutics, a company developing treatments for Duchenne muscular dystrophy. Currently on Grant panels for Telethon (Italy), AFM (France), MDUK. Previously a grant panel member for BBSRC and NC3Rs. Research grant funding Current and Previous from the BBSRC, MRC, Wellcome Trust, AFM, MDUK and other charities

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