Abstract
Cystic fibrosis is a serious chronic disease, which was first described less than 70 years ago. Since then great progress in understanding the nature of the disease has been made.
Patients' prognosis improved significantly in the mid 1950s, when new antibiotics were introduced. The discovery of chloride transport across the epithelial membrane and especially the discovery of the CFTR gene in 1989 enabled the comprehension of disease aetiology.
They also offered the opportunity to search for new therapeutic approaches. The aims of classical symptomatic treatment were to prevent/treat bacterial infection, to improve airway clearance and nutritional status.
New methods have now been added; they are focused on better modes of medicament application to the airways, improving airway clearance, fighting inflammation, and searching for, preventing, and treating complications. Great expectations are set on the evolution of new methods targeting the pathophysiology of the disease.
Several new medicaments are going through clinical trials. Their aim is to repair the CFTR protein according to the class of mutations, to inhibit the sodium channel and thus to improve the quality of airways surface liquid.
Others are focusing on suppressing infection and inflammation. Several of these drugs have received the status of orphan drugs in EU, which enables their quicker development and registration for the indication of cystic fibrosis.
Currently new therapeutic approaches only supplement the classical therapy. But they have tremendous promise of potential future benefit in ion transport and in ameliorating or replacing CFTR function.