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

Article Abstract:

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.)

Research, Physiological aspects, Lung diseases, Sodium channels, Aerosol therapy

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A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis

Article Abstract:

Transfer of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to nasal cells in people with cystic fibrosis appears to be ineffective in correcting these cells' function. The CFTR gene controls chloride and sodium transport in cells lining the respiratory tract. Viruses with and without the normal CFTR gene were applied in different doses to the nasal passages of 12 people with cystic fibrosis. Genetic testing of nasal cells indicated that only those cells exposed to the higher viral doses incorporated the normal CFTR gene. Measurement of the potential difference across the nasal cells indicated that cells had not gained normal transport function. Two of the three patients receiving the highest viral doses experienced side effects including inflammation of the nasal passages, earache, and jaw ache.

Evaluation, Gene therapy

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Mucus clearance and lung function in cystic fibrosis with hypertonic saline

Article Abstract:

The hypothesis that pretreatment with amiloride would extend the duration of hypertonic saline-induced acceleration of mucus clearance and enhance improvement in lung function in patients with cystic fibrosis was tested. The findings reveal that inhalation of hypertonic saline four times daily provides a modest improvement in the lung function and respiratory symptoms without substantial adverse events.

United States, Medicinal and Botanical Manufacturing, Medicinals and botanicals, Saline Solution, Dosage and administration, Physiologic salines

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