Cystic fibrosis is a genetic condition in which the lungs become clogged with thick sticky mucus. For the first time, gene therapy has offered hope to cystic fibrosis sufferers in a phase 2 randomized trial. It has shown a significant benefit in lung function compared with placebo.
The gene therapy for cystic fibrosis involves replacing 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. Senior author Eric Alton from the National Heart and Lung Institute at Imperial College London 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."
Alton further added, "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."
Senior co-author Stephen Hyde from the Gene Medicine Research Group at the University of Oxford said, "Stabilization 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."
Senior co-author Alastair Innes from Western General Hospital, Edinburgh, UK 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."
The study has been published in The Lancet Respiratory Medicine.