Aerosol Therapies for Cystic Fibrosis

As our understanding regarding the pathophysiology of cystic fibrosis lung disease has progressed, clinicians and basic researchers have worked to improve the treatment of this condition. Much of the new research is related to the recent discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) [1–3]. As the pathophysiological defects in ion transport leading to the clinical entity of cystic fibrosis (CF) are dissected, many of the associated secondary abnormalities of this disease are better understood. This has led to novel therapies that will be discussed in this review. For example, amiloride and nucleotide triphosphates are being investigated for the treatment of CF because of their effects on ion transport in the respiratory epithelium. Recombinant human DNase is an agent that serves to thin mucus from CF airways, making it easier for patients to clear viscous secretions from the respiratory tract. More established therapies with a longer history of use, such as nebulized bronchodilators and antibiotics, will also be discussed.