Novel Pharmaceutical Approaches for Treating Patients with Cystic Fibrosis

Before the cloning of the CFTR gene in 1989, there were relatively few treatment options for the many phenotypes associated with cystic fibrosis (CF). The advancement of research in areas such as immunology, molecular biology and pharmacology have provided new insights into the mechanism and evolution of CF. More than 40 systematic clinical trials evaluating new therapies for CF are presently registered with the NIH. A great deal of effort is focused on the main cause of mortality: chronic and persistent lung infections. Intestinal malabsorption, pancreatic insufficiency, reduced bone mineral density and reproductive abnormalities are other manifestations of this disease that have been targeted by innovated treatments which are giving renewed hope to CF patients and their families. The following review is a summary of the novel pharmaceutical approaches for the treatment of cystic fibrosis aimed at improving both the quality and the longevity of the lives of patients afflicted with this devastating disease.