Life-changing gene therapy for cystic fibrosis sufferers could be available in the next five years following a landmark trial in Edinburgh and London.
For the first time, a gene-altering treatment was shown to improve or stabilise the lung function of patients and lead to significant health gains.
It’s feasible that we’ll be able to offer this treatment by the end of the decadeProfessor Eric Alton
The £3 million trial, involving 136 patients aged 12 and over, is seen as the curtain raiser for a much larger study next year.
Scientist say there is more work to be done but believe they are on track to deliver a therapy that can make a meaningful difference to patients’ lives.
Professor Eric Alton, from Imperial College London, who co-ordinated the trial, said: “For the first time in the world, we have a significant benefit compared with placebo in cystic fibrosis patients.
“If the bigger study shows big benefits then it’s feasible that we’ll be able to offer this treatment to patients by the end of the decade.”
Cystic fibrosis is a rare but devastating inherited disease caused by a single defective gene that causes the lungs to secrete thick mucus. The airways become clogged up and vulnerable to recurrent life-threatening infections.
Lung damage caused by infections is responsible for 90 per cent of deaths of people with CF, who are not likely to live beyond the age of 35. Since the genetic basis of CF was discovered in 1989, scientists have been working on ways to replace the defective gene. But all attempts so far have been thwarted by the body’s protective mechanisms for clearing unwanted material out of the lungs and fighting off foreign invaders such as viruses, which are used to ferry corrective DNA into cells.
The new treatment does not employ viruses but fat globules, or liposomes, that carry the genetic material and are inhaled through a nebuliser. The trial compared the effects of inhaled gene therapy and a dummy “placebo” treatment on patients with CF aged 12 and over.
The results, published in the Lancet Respiratory Medicine journal, showed that, at the end of the trial, lung function was 3.7 per cent better in patients who received the “active” treatment.
Prof Alton said: “Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group and there were no safety concerns. Whilst the effect was inconsistent, with some patients responding better than others, the results are encouraging.”
He stressed that even though “a whole swathe” of patients improved, the treatment was not yet ready for use in clinics.