A gene therapy for cystic fibrosis has successfully been used to improve the health of patients for the first time, it was announced today.
The benefits were “modest” – but were significant for the first time since the CF genes were discovered in 1988.
The treatment, tested in the UK, involved patients inhaling molecules of DNA into the lungs.
Researchers said some patients fared better than others – but that the results are “encouraging.”
Some 136 patients aged 12 or over took part in the study, reported in The Lancet Respiratory Medicine.
Some 62 patients took the therapy for a year – and at the end their lung function was assessed as having improved by 3.7% compared with patients taking a placebo treatment.
Among patients with the worst lung function, the improvement was 6.4%.
Researcher Professor Eric Alton, of Imperial College, London, UK, said: “Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group and there were no safety concerns.
“Whilst the effect was inconsistent, with some patients responding better than others, the results are encouraging.”
Fellow researcher Professor Stephen Hyde, of Oxford University, said the search to find effective ways of getting healthy DNA into the lungs would continue.
And another researcher, Dr Alastair Innes, from Western General Hospital, Edinburgh, added: “Publication of this trial is a landmark for cystic fibrosis patients and we are particularly grateful to the many patients across the UK who gave their time and effort to participate and make this collaborative venture a success.”
E.W.F.W. Alton et al. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. Lancet Respir Med 3 July 2015
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