A combination therapy, developed by US pharmaceutical company, Vertex, improves lung function and reduces hospitalisations for patients with the most common type of CF.
In particular, it reduced lung infections by between 30 and 39% and helped people with CF to gain weight.
Prof Stuart Elborn, an international authority in respiratory medicine at Queen’s University, Belfast, who played a pivotal role in studies of the new treatment, said it was a “very significant” breakthrough for people with CF.
CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1,200 children and adults in the Ireland and 70,000 worldwide.
“While we had previously found an effective treatment for those with the ‘Celtic gene’ this new combination treatment has the potential to help roughly half of those with cystic fibrosis — those who have two copies of the F508DEL mutation,” said Prof Elborn, who worked with colleagues from the US and Australia.
Two Phase 3 studies of the drugs ivacaftor and lumacaftor, which included over 1,100 patients worldwide, built on previous studies of ivacaftor in patients with G551D and other related mutations.
Ivacaftor, also known as kalydeco, is the first drug to treat the underlying causes of cystic fibrosis rather than just its symptoms and is currently approved for patients with the ‘Celtic gene’ mutation carried by about four per cent of all patients and 10-15 per cent of patients in Ireland.
The latest trial looked at the treatment of patients with two copies of the F508DEL gene mutation which is carried by roughly half of all cystic-fibrosis patients.
Research and development officer with Cystic Fibrosis Ireland, Katie Murphy, said the results of the study that combined kalydeco with the new drug, lumicaftor, were “extremely promising and statistically significant.”
Ms Murphy, who was speaking on RTE radio yesterday, said the treatment allowed mucus to be cleared away from the lungs more easily, reducing infection and damage to the organs.