The experimental drug named denufosol used early in life could help in delaying or preventing lung disease progression in children, teenagers and young adults with cystic fibrosis. The American Journal of Respiratory and Critical Care Medicine study said those given the drug and not a placebo for six months fared better in lung tests.
Frank J. Accurso, M.D., Head of Pulmonary Medicine, Director of Cystic Fibrosis Center at University of Colorado, Denver, and the lead principal investigator for TIGER-1 stated, “These exciting data suggest that denufosol may have promise in this mild patient population. The improvement in lung function observed during the placebo-controlled portion and the open-label extension of the TIGER-1 trial is notable given that the patient population studied had little to no pulmonary function impairment.”
Denufosol belongs to a class of drugs known as ion channel regulators. These drugs help balance the flow of ions through cell membranes, helping normalize the airway surface hydration and mucus clearance impairment present in patients who suffer from the disease. in cystic fibrosis, the ion sodium chloride does not flow normally through cell membranes, resulting in thick, sticky mucus which is difficult to cough out of the airways.
Denufosol works by increasing chloride secretion, inhibiting sodium absorption and increasing the beat frequency of the tiny hairs, or “cilia,” lining the airways move to clear mucus. Combined, these effects enhance airway hydration and aid in clearing mucus.
The drug is different from other cystic fibrosis medications, which mainly treat the symptoms rather than the underlying causes, said Accurso, who is also the director of University of Colorado’s cystic fibrosis center.