The results of two clinical trials show that a new combination of medications can successfully treat the underlying causes of cystic fibrosis in patients ages 12 and older.

The international trial, which studied more than 1,000 cystic fibrosis patients age 12 and older, revealed that a combination of the drugs Kalydeco (ivacaftor) and lumacaftor, an experimental drug that has not yet been approved by the Food and Drug Administration (FDA), successfully treated the defective CF protein and improved lung function. The drugs also helped patients achieve a 40 percent reduction in pulmonary exacerbations, the leading cause of death in cystic fibrosis patients.

“Being able to treat not just the symptoms, but the underlying cause of cystic fibrosis by targeting this specific gene mutation, is a major game changer because it is by far the most common in patients with this disease,” said Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and professor of Pediatrics at the University of Washington, who presented the study’s findings at the Center for Drug Evaluation and Research advisory committee meeting on May 12.“Previously, we found that Kalydeco treated just four percent of cystic fibrosis patients, but this new combination treats the defective CF protein for a much greater number of patients.”

View the full story at www.newswise.com