For the first time, gene therapy has offered hope to cystic fibrosis sufferers after its successful trials.
Gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomised trial. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.
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, said senior author Eric Alton from the National Heart and Lung Institute at Imperial College London.
He added that whilst the effect was inconsistent, with some patients responding better than others, the results are encouraging.
Overall, the gene therapy was well tolerated and patients in the treatment and placebo groups experienced similar rates of adverse events.
According to senior co-author Stephen Hyde from the Gene Medicine Research Group at the University of Oxford, Stabilisation of lung disease in itself is a worthwhile goal. Researchers are actively pursuing further studies of non-viral gene therapy looking at different doses and combinations with other treatments, and more efficient vectors.
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Senior co-author Alastair Innes from Western General Hospital, Edinburgh, UK added that 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.
The study is published in The Lancet Respiratory Medicine journal.