New therapies for cystic fibrosis

In the decade since the gene responsible for cystic fibrosis (CF) was identified, our understanding of the pathophysiology of CF pulmonary disease has significantly improved. The current model for CF lung disease suggests several levels at which clinical interventions may be made in an attempt to alter the natural course of disease progression. The first part of this review highlights some of the progress made in novel forms of therapy directed at earlier portions of the pathophysiologic cascade such as gene therapy, protein therapy, and ion-transport regulatory therapy. New developments in well-established modes of therapy such as mucolytic therapy, airway clearance therapy, and antibiotic therapy are discussed next. The review concludes with a look at the use of two forms of therapy that have been adapted to CF care, anti-inflammatory therapy and lung transplantation.