Small Airways Disease in Cystic Fibrosis: Improving efficacy of treatment

Cystic fibrosis (CF) is a severe hereditary and life-threatening disease in the Caucasian population, affecting 70,000 patients worldwide. In the 1950s, a child with CF would rarely live long enough to attend elementary school. Luckily, life expectancy has dramatically improved due to the development of new treatments and treatment approaches. The current median predicted survival is close to 40.
The main cause of mortality and also morbidity is progressive lung disease.3 Patients with CF have an increased susceptibility to airway infections due to the defects in the CF transmembrane conductance regulator (CFTR). Due to a dysfunction in the CFTR channel, ion transfer over the membrane of epithelial cells is abnormal what leads to dehydration of the airway surface liquid in the lungs. This results in thick, viscous mucus that impairs mucociliary clearance and obstructs the airways. Because of these alterations, it is difficult for CF patients to eliminate inhaled bacteria from the lungs. A persistent infection arises, which damages the airways and eventually leads to respiratory failure. The most common mutation causing CFTR dysfunction is the dF508 mutation, but over 2000 mutations have been identified. The disease is complex and the rate of progression of CF lung disease varies widely from person to person, even among patients carrying identical CFTR mutations. Despite the significant improvements in the treatment of the disease, there is still no cure and lung infections remain a serious problem for patients living with CF.