New Cystic Fibrosis Treatment Uses Gene Therapy to Improve Lung Function

By James O Malley on at

A new gene therapy treatment could offer hope to cystic fibrosis sufferers following a seemingly successful clinic trial.

The Indy reports that the trial, which took place in Britain, showed "modest and variable" improvement in lung function, without any harmful side effects, after the 136 participants took a monthly dose of gene therapy for a year.

The way the new treatment works is by using a nebuliser spray to inhale "liposomes" that have apparently been wrapped around healthy, synthetic copies of the cystic fibrosis gene. These liposomes are then absorbed by cells in the patient's airway, and they trigger the production of healthy proteins which prevent the mucus which clogs up sufferer's insides. The gene appears to have been especially designed to work with specific cells in mind, to make sure it affects the right place.

The results, which have been published in Medical Journal The Lancet, showed a 5% improvement in lung function amongst patients - with improvements as much as 6% amongst the worst affected.

As the Indy notes though, doctors are very keen to point out it is still early days with the treatment - and don't want to raise expectations as since the cystic fibrosis gene was first identified in 1989, expectations for an imminent cure have been high. But at least this study does offer renewed hope that better treatement might one day be available. [The Independent]