Update on clinical trials in the treatment of pulmonary disease in patients with cystic fibrosis.
Cystic fibrosis is a congenital disease resulting from an abnormality of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. A defect in ion transport leads to poor clearance of viscoelastic secretions and a susceptibility to bacterial infection. This initiates a self-perpetuating cycle of infection and inflammation that accounts for the chronic endobronchial sepsis and pulmonary damage observed in patients with cystic fibrosis. Recent studies have attempted to correct the gene defect, enhance the expression and function of the CFTR protein and correct the ion transport defect. Improving the rheological properties of airway secretions, enhancing host defence and controlling inflammation are the other key strategies.
Shah
PL
Royal Brompton Hospital, Sydney Street, London SW3 6NP, UK. pallav.shah@ic.ac.uk