European Cystic Fibrosis Society (ECFS) president and Cystic Fibrosis Trust professor Stuart Elborn was one of the contributors of the latest breakthrough in cystic fibrosis (CF) research, and he believes that the positive results of two phase 3 studies of the drugs ivacaftor (Kalydeco) and lumacaftor may change the course of the disease treatment. This is the first time that a combination of medications target not only the symptoms, but also the causes of CF.
The combination of ivacaftor and lumacaftor, developed by Boston based biotech company Vertex, is currently in late-stage trials, and the latest studies have demonstrated that it is able to improve lung function as well as reduce the risk of hospitalization in patients with the most common type of cystic fibrosis.
Dr. Elborn, a professor at Queen’s University Belfast (QUB), director of Queen’s Centre for Infection and Immunity, Dean of the School of Medicine, Dentistry and Biomedical Sciences at QUB, and an international authority in respiratory medicine, worked in collaboration with colleagues from the United States and Australia, as he led the pivotal studies of the new combination treatment for CF patients.
“This is a very significant breakthrough for people with cystic fibrosis,” Dr. Elborn noted, as quoted in an article in the Belfast Newsletter. “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 the F508DEL mutation. This is another example of how Queen’s scientists are working internationally to change lives around the world.”
Ivacaftor is the first drug treatment that not only treats the symptoms, but also the underlying causes of CF, and is currently approved for patients with the ‘celtic gene’ mutation carried by about four percent of all patients. The two phase 3 studies performed by Vertex tested the combination of ivacaftor and lumacaftor in over 1,100 patients worldwide, built on previous studies of ivacaftor in patients with G551D and other related mutations.
Professor Stuart Elborn was recently one of the main panelists at the 37th European Cystic Fibrosis Conference, one of the events organized by the ECFS for the international cystic fibrosis research community, which gathered leading CF caregivers and researchers in Gothenburg, Sweden.