For 26 years, since researchers first identified the gene responsible for cystic fibrosis, scientists have attempted to use the knowledge to help treat the illness. Now, gene therapy has been shown to help sufferers for the first time.
Cystic firbrosis is the world’s most commonly diagnosed genetic disease. Affecting 70,000 people around the world, it causes a build of thick mucus in the lungs which provides a space where bacteria can flourish. Sufferers tend to die young—the average age of death for sufferers in the UK is 41—and management of the illness requires regular use of antibiotics and physiotherapy.
Now, a team from the University of Oxford has developed a gene therapy technique for the disease which has been shown to be effective. The treatment sees patient inhale vapour which contains fatty bubbles—known as liposomes—that are laced with healthy DNA. In the lungs, this enters the cells.
In a trial of 116 participants, 62 received monthly doses of the therapy and a control group of 54 inhaled saline solution. Their lung function was regularly tested by measuring how much air they could exhale in a single second. The results show that over the course of 12 months, those taking the treatment exhibited 3.7 per cent better lung function than those taking the placebo.
That may not sound like much, but it’s worth noting that lung function of people with cystic fibrosis typically declines by 2 to 3 per cent a year. New Scientist points out that the difference afforded by the drug “could mean the difference between needing a lung transplant at the age of 60 instead of 40.”
That’s obviously wonderful news for sufferers of the illness, though wider testing will be required before it’s used as a conventional treatment. Equally interesting, though, is the fact that the work demonstrates that gene therapies can be effectively delivered into the lungs—opening the door for the delivery of other gene therapies.
Image by Yale Rosen under Creative Commons license