New Gene Therapy could help Cystic Fibrosis sufferers

Turns out, a new gene therapy for Cystic Fibrosis sufferers, helps moderately improve or stabilise the lung function of patients. An early-stage trial of the gene-altering medicine has yielded promising results.

Cystic fibrosis, an inherited chronic disease, damages the lungs and digestive system. It affects 1,200 children and adults in Ireland.

A defective gene is the cause behind it, leading the body to produce thick mucus that clogs the lungs; risk of life-threatening infections looms over the sufferer.

Scientists in London and Edinburgh conducted the trial of this new gene therapy, involving 136 CF patients aged 12 and over.

Every month for a year, fat droplets were inhaled by the participants to carry genetic material into their lungs. DNA slipped into lungs after the globules fused with fatty cell membrane walls.

Details of the treatment have been published in the journal Lancet Respiratory Medicine. The trial so far showed 4% better lung infection in those taking the therapy. A 6.4% health gain was experienced by participants with the worst lung function at the start of the study.

The scientists have said the more work is needed before confirming potential of the treatment to become a viable clinical one.

“If I was on the board of a pharmaceutical company, I would require further studies to determine the best dose and whether the current treatment could be combined with other drugs to increase the effect”, said Prof Stuart Elborn, of Queen's University in Belfast. He, however, said the results of the study are encouraging.