ADVANCES IN THE PHARMACOTHERAPY OF CYSTIC-FIBROSIS

The quality of life and life-span of cystic fibrosis patients have been improved substantially in the last 20 or 30 years. This has been accomplished largely through improved early diagnosis and advances in traditional pharmacotherapy which mainly consists of antibiotic therapy. In the past few years, the basic cellular defect of cystic fibrosis, as well as the gene determining the disease, have been described. These milestones in the quest for knowledge about the disease open the door for other innovative forms of therapy. While corrective gene therapy may be the ultimate result of advances made based upon this new knowledge, we will probably see earlier breakthroughs in the form of immune reaction inhibition and correction of ion flux disturbances. Each of these present and future forms of therapy is explored in this paper.