Research Drug May Delay Progression of Cystic Fibrosis
Patients with cystic fibrosis (CF) with normal to mildly impaired lung function may benefit from a new investigational drug designed to help prevent formation of the sticky mucus that is a hallmark of the disease, according to researchers involved in a phase 3 clinical trial of the drug. Called denufosol, the investigational medication can be given early in the CF disease process, and may help delay the progression of lung disease in these patients, the researchers found.
The findings were published online and distributed by Newswise ahead of the print edition of the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine.
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.
Denufosol works by increasing chloride secretion, inhibiting sodium absorption and increasing the beat frequency of the cilia, lining the airways to move and clear mucus. The drug is different from other CF medications, which primarily treat the symptoms rather than the underlying causes, said lead investigator Frank Accurso, MD, professor of pediatrics at University of Colorado School of Medicine and director of the university's cystic fibrosis center.
This study is the first large, phase 3 trial of an ion channel regulator in CF patients with little or no baseline pulmonary function impairment.
Researchers enrolled 352 cystic fibrosis patients 5 years of age or older to receive either inhaled denufosol or placebo three times daily for 24 weeks, followed by a 24-week open-label period when all patients received denufosol. At baseline, most patients enrolled had mild impairment of lung function and were taking multiple medications to control their symptoms. Patients' exhalation rates and lung volume were measured throughout the study. Patients also were monitored for adverse events, such as cough, congestion, fever or sinusitis. At the end of the 24-week period, researchers determined patients who received denufosol had better lung exhalation rates than those in the placebo group, whose exhalation volumes remained relatively unchanged from the start of the study. Both groups had similar numbers and types of adverse events, with the denufosol patients experiencing significantly fewer headaches and lower rates of sinusitis and runny nose.