Genetics On the Forefront of New Cystic Fibrosis Treatment

 Ivacaftor went through clinical trials like any drug before being FDA approved. The clinical trials looked at ivacaftor’s tolerability and clinical effect on patients with a specific type of CFTR mutation. The researchers looked at the CFTR function, lung function, and quality of life in the study participants. Results showed that ivacaftor was generally well tolerated among the study subjects. There were significant improvements in sweat chloride concentrations in the patients who received the drug. Additionally, patients who received the ivacaftor showed increased forced expiratory volume (FEV) from their previous baseline. This first study was of course not without limitations. It was of a relatively small sample size which makes its applicability to the larger CF population perhaps a bit less realistic. A second study also demonstrated that ivacaftor has the potential to greatly improve lung function in patients with CF. Common adverse effects that were seen in patients in subsequent studies were headaches, nasal congestions, upper respiratory infection, rash and dizziness.