Gene therapy hope for CF patients

Gene therapy can improve lung function in cystic fibrosis patients, a clinical trial has found.

A treatment that replaces the faulty gene responsible for cystic fibrosis has produced encouraging results in a major UK trial.

Improved lung function

One hundred and thirty six patients aged 12 and over received monthly doses of either the therapy or a dummy treatment for one year. Of that number, fifty-five were treated in Edinburgh.

Patients who received the therapy had a significant, but modest, improvement in lung function compared with those receiving a placebo.

The team says more research is needed to improve the effectiveness of the therapy before it will be suitable for clinical use.

Cystic fibrosis

Cystic fibrosis affects around 10,000 people in the UK and over 90,000 worldwide.

Patients’ lungs become filled with thick mucus and they are vulnerable to recurrent chest infections, which eventually destroy the lungs.

The gene mutation that causes cystic fibrosis was identified in 1989. This discovery opened the door to new treatments to repair the damaged gene.

Current therapies have improved life expectancy for patients but are focussed on treating the symptoms of the disease rather than the cause.

UK-wide trial

Patients from across England and Scotland participated in the trial and were treated in two centres, Royal Brompton Hospital in London and the Western General Hospital in Edinburgh.

The study was carried out by the UK Cystic Fibrosis Gene Therapy Consortium, a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian.

The findings are published in The Lancet Respiratory Medicine. The trial was funded by the National Institute for Health Research and the Medical Research Council.

We are naturally encouraged by the outcome of the trial, and are actively investigating how to make non-viral gene therapy for CF more effective by modifying the design of follow-on clinical trials. It is fitting that the Scottish arm of the trial, enrolling patients from NE England and every part of Scotland, took place at the Western General Hospital campus where important early work on CF genetics and gene therapy was pioneered by Professor Julia Dorin and Professor David Porteous.

Dr Christopher BoydCentre for Genomic and Experimental Medicine, University of Edinburgh