Gene Therapy for Cystic Fibrosis

An article in The Lancet Respiratory Medicine reports the first ever successful testing of a gene therapy for Cystic Fibrosis (CF). CF is caused by having two faulty copies of the CFTR gene, this results in a thick heavy mucous that builds up in the lungs providing a ripe breeding ground for bacterial infections. Treatments include a brutal regimen of physiotherapy to dislodge and bring up the mucous and plenty of antibiotics to keep infections at bay. Even with the best treatment available, though, 50% of sufferers are dead by the age of 41.

Researchers used a plasmid to deliver normal copies of the CFTR gene into the lungs of patients using an inhaler. They had treatment once per month for a year. Once the study was complete they compared lung function with a year earlier and also against a control group that was given only saline. The treatment group saw an increase in lung function of about 3% whist the control group saw a decrease in function. Whilst the improvement was modest if treatment could be started early in life then the chronic degradation of the lungs that is so characteristic of the disease could be stopped in its tracks. The team are now working on a virus based delivery system.