Cystic fibrosis clinical trials

Abstract

The ion transport abnormalities in cystic fibrosis are becoming increasingly well defined, although how these lead to lung pathology is still speculation. Correction of these defects could theoretically be achieved either through pharmacological means or via gene therapy. Pharmacological approaches include increasing the amount of CFTR protein that reaches its correct localisation in epithelial cells. Secondly, approaches have been suggested which could increase the function of the protein already present at this correct localisation. Finally, it may be possible to identify alternative channels which could subserve the function of CFTR. Gene therapy is theoretically an attractive proposition as it should circumvent each of the identified abnormalities in cystic fibrosis. The principal difficulty at present relates to delivering sufficient copies of the normal CFTR gene into the appropriate cell population in vivo. A number of clinical trials have now been undertaken and steady and encouraging progress has been made in moving this approach from theory to practice.