Cystic fibrosis sufferers offered new hope with gene therapy

3 Jul 2015

There has been a significant breakthrough in the treatment of cystic fibrosis (CF), with patients in testing showing a considerable reduction in symptoms after undergoing a new form of gene therapy.

According to the team of researchers that developed this new gene therapy, their treatment was tested on 136 patients and in nearly all cases it was shown to stabilise and offer slight improvements to those with CF.

In terms of figures, those who received the gene therapy showed a 3pc decline in mucus build-up, while those taking on the role of the control showed a decline in their lung health by 3-4pc in the same period. The team published its results online.

The faulty gene that causes CF leads to a build-up of mucus in the throat, nose and lungs of those who suffer from it, which not only makes daily life that much more difficult, but means their average life expectancy is only 41.

Ireland has a particularly strong attachment with the treatment and search for a solution to cure CF, as people in the country are statistically the most likely to receive the faulty gene, with an average of one person in 19 carrying it.

Speaking to the BBC, one of those successful patients during the trial, Kieran Kelly, said the therapy has been hugely beneficial to his daily life.

“I did feel a lot healthier,” he said. “It might have been psychological, but I did feel better in myself.”

However, the team of researchers from Imperial College London said that there is still a long way to go before it can be released to the public as a treatment.

Speaking of the trials, lead author of the paper Prof Eric Alton said: “The effect is modest and it is variable. It is not ready to go straight into the clinic yet.”

Graffiti of lungs image via Quinn Dombrowski/Flickr

Colm Gorey was a senior journalist with Silicon Republic