A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis

One consequence of cystic fibrosis is the excessive absorption of sodium by cells lining the respiratory tract, which may contribute to the drying of airway secretions. The effects of aerosolized amiloride, a substance that blocks sodium channels, was tested in 14 cystic fibrosis patients in a randomized study. Each patient underwent two 25-week treatments, one with amiloride and one with only the vehicle (the inactive substance that carries the amiloride). The drug or placebo was administered four times each day. Tests of lung function were performed and symptoms of pulmonary dysfunction noted. Results showed that forced vital capacity (the amount inhaled by forcible inhalation) decreased almost twice as much from the baseline level during treatment with vehicle than with amiloride. The decrease in forced expiratory volume in one second was not different for vehicle and amiloride. The rheologic values (a measure of flow properties) values of sputum, including elasticity and viscosity, approximated normal values for patients during amiloride treatment, while the solid content of sputum did not change during the two periods. Cough clearance also improved with the drug. It appears that amiloride is a moderately effective agent for treating adults with lung disease resulting from cystic fibrosis. (Consumer Summary produced by Reliance Medical Information, Inc.)