Sodium channel blockers for cystic fibrosis

Cystic fibrosis–related metabolic defects: crosstalk between ion channels and organs

“…Cystic fibrosis (CF) is a genetic condition that affects the cystic fibrosis transmembrane conductance regulator (CFTR) protein ( 1 , 2 ). Patients with CF have defects in the movement of chloride, sodium, and water in and out of cells ( 3 ). In the lung epithelium, this defect dramatically increases the viscosity of the mucus, resulting in life-threatening blockages ( 4 ).…”

Aerosol Medications for Treatment of Mucus Clearance Disorders

“…The epithelial sodium channel inhibitor amiloride has not been shown to be effective in the treatment of CF, and there is some evidence that its use is associated with deterioration in lung function. 13 …”

Cystic fibrosis transmembrane conductance regulator dysfunction and its treatment

“…12,13 Three subsequent randomized control trials 14 -16 failed to show any benefit of amiloride on respiratory function tests over a 6 to 12 month period (see also Cochrane review 2006). 17 It has been suggested that the lack of clinical benefit of amiloride is due to its short half-life on the airway surface because of rapid absorption. Higher potency sodium channel blockers with longer airway surface half-lives have been developed, most recently a compound called PS552.…”