Hydrator therapies for cystic fibrosis lung disease

Abstract

Cystic fibrosis (CF) provides an extreme example of the consequences of failed host defense of the lung. Investigation into the nature of the CF defect has revealed that abnormal ion transport activities lead to dehydration of airway surfaces and defective mucus clearance. This article will review the normal regulation of airway surface liquid volume and the abnormalities in CF that are felt to initiate lung disease. Fortunately, a number of therapeutic approaches might be employed to restore airway surface liquid hydration in CF, and the current state of their development will also be discussed. Finally, a case will be made for the early use of this emerging class of airway “hydrators.” Although these agents have the theoretical potential to significantly delay the onset of clinical disease by targeting its underlying pathophysiology, a great deal of research is required to prove this concept and to optimize their efficacy. However, the advent of therapies aimed at the earliest steps of disease pathogenesis provides a great deal of hope for the immediate future. Pediatr Pulmonol. 2008; 43:S18–S23. © 2008 Wiley-Liss, Inc.