Gene therapy for cystic fibrosis shows encouraging trial results

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

One hundred and thirty six patients aged 12 and over received monthly doses of either the therapy or the placebo for one year.

The clinical trial reached its primary endpoint with patients who received therapy having a significant, if modest benefit in lung function compared with those receiving a placebo.

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

The trial is the first to show that repeated doses of gene therapy can have a meaningful effect on the disease, and change the lung function of patients. However, the team say more research is needed to improve the effectiveness before the therapy will be suitable for clinical use. Imperial College London