'Very encouraging' results for Irish cystic fibrosis patients in drug trials

A NEW drug has been shown to improve lung function, weight and quality of life in people who have a particular strain of cystic fibrosis (CF), a conference on the condition was told at the weekend.

Dr Edward McKone, consultant respiratory physician at St Vincent’s University Hospital, in Dublin, said the results were “very encouraging” for all CF patients.

Cystic fibrosis is the most common life-threatening genetic condition in Ireland, with one in 1,400 babies born with it. It affects the CFTR gene, which regulates salt in cells. If this gene is mutated it leads to a build-up of mucus in the lungs, liver and pancreas. Ireland has the highest rate of people who carry the mutated gene in the world and has the highest incidence of CF in the world.

Eleven per cent of people in Ireland have a type of gene mutation called G551D, which is known as the Celtic mutation and is associated with severe CF.

Dr McKone told the Cystic Fibrosis Association of Ireland national conference in Westport, Co Mayo, that a new drug, VX-770, produced by Vertex Pharmaceuticals and the US Cystic Fibrosis Foundation, targets that specific mutation.

People being treated at St Vincent’s, the national referral centre for adults with cystic fibrosis, took part in the clinical trials for the drug along with people from the United States, Australia and other parts of Europe.

“The results demonstrated significant increases in lung function, weight and quality of life in CF patients with the G551D mutation who received VX-770 compared to a placebo,” Dr McKone said.

He said the tests indicated the drug was working on “the cause of CF”, rather than on treating the complications of CF.

A second study, which included children over six years of age, reported similar findings, he said.

Dr McKone said further research was under way to identify therapies that would work on other CFTR mutations. “But the results of this trial are very encouraging for all CF patients as it proves that therapies that target CFTR can lead to significant clinical benefits for CF patients,” he said.

Also at the conference, Philip Watt, chief executive of the Cystic Fibrosis Association of Ireland, said some Health Service Executive cost-saving measures were directly affecting CF services.

He said one hospital was without a physiotherapist for three months, which resulted in many children with CF not getting physiotherapy; another had lung function equipment that could not be used because it didn’t have the resources to employ a technician. And a third hospital had a CF adult consultant who could not see adults with CF because he didn’t have a proper consulting room.

Mr Watt also asked if the Government would offer funding and support to ensure new CF treatments can be offered in Ireland.

Opening the conference, Taoiseach Enda Kenny said the new dedicated CF unit for St Vincent’s was “on track, on time, on budget” and it was his intention to keep it that way. He said the forthcoming newborn screening for CF would be introduced as part of the existing national newborn bloodspot screening programme this summer. He committed to “doing everything possible to secure improvements in services for people with CF”.

Mr Kenny presented a special recognition award to Prof Charles Gallagher of St Vincent’s hospital for his services to CF.