A gene therapy has stabilised and slightly improved cystic fibrosis in some of 136 patients in a trial. Their lungs showed no decline, on average, after they inhaled healthy copies of the gene that causes CF once a month for a year, results published in Lancet Respiratory Medicine show. And the lungs most clogged before the trial showed a 3% improvement. The lungs of patients that did not take the gene therapy showed a decline of 3-4% on average over the same period.
This is the first evidence worldwide which shows that if you give gene therapy to CF patients it has a protective effect Prof Eric Alton, Imperial College. Prof Eric Alton, of Imperial College London, who led the trial, warned: “The effect is modest and it is variable. It is not ready to go straight into the clinic yet.”
Prof Alton and his colleagues at the UK Cystic Fibrosis Gene Therapy consortium, which includes scientists at Edinburgh and Oxford Universities as well as Imperial, are hoping to have a further trial next year. Cystic fibrosis is an inherited condition caused by a faulty gene that leads to a build up of sticky mucus causing debilitating infections in the nose, throat and lungs.