Emerging therapies in cystic fibrosis

Abstract

The life expectancy of people with cystic fibrosis (CF), a lethal inherited disease, has been greatly extended by advances in therapy. Currently, there are a number of potential drugs for treatment of CF lung disease in clinical trials. These therapies are targeted at all points in the pathogenesis of lung disease, from gene transfer to drugs that treat mucus, infection and inflammation in the airways. An exciting development is that of modulation of the abnormal protein that causes CF, the cystic fibrosis transmembrane conductance regulator (CFTR), where drugs are targeted at specific defects in CFTR transcription, processing or functioning. Inhaled therapies are being developed to augment airway surface liquid height, either by modulating the abnormal ion channel function in the airway epithelial cell or by rehydrating with osmotic agents. Anti-inflammatory therapy is also of great interest in CF and there are several candidate drugs in clinical trials. A number of antibacterial agents formulated for inhalation are at various stages of study or newly approved, which should improve options for chronic management of airway infection. Hopefully, many of these potential therapies will come to market and will further extend the life expectancy of people with CF.