Posted on: Thursday, 23 December 2010, 08:16 CST

(Ivanhoe Newswire) — Cystic fibrosis (CF) is a common disease which affects the entire body, causing progressive disability and often-early death.  Patients with normal to mildly impaired lung function, however, may be given some new hope thanks to a recent study investigating a novel drug.  It’s called denufosol, and it is an investigational treatment designed to help prevent formation of the sticky mucus that is a characteristic of the disease.

"although the lungs of children with CF are thought to be normal at birth, studies have demonstrated significant lung damage that occurs early in life in children suffering from cystic fibrosis," which lead investigator Frank Accurso, MD, professor of pediatrics, University of Colorado School of Medicine, Denver, was quoted as saying.  "Many patients continue to suffer progressive loss of lung function despite treatment of complications.  because denufosol can be used early in life, it offers hope for delaying or preventing the progressive changes that lead to irreversible airflow obstruction in CF patients."

It’s a family known as ion channel regulators, and denufosol in the newest member.  Drugs in this family aid in balancing the flow of ions through cell membranes, all the while normalizing the airway surface hydration and mucus clearance impairment often present in patients with the disease.  in cystic fibrosis, the ion sodium chloride does not flow normally through cell membranes.  this ultimately results in thick, sticky mucus – a breeding ground for bacteria resulting in respiratory issues – which is alas tough to cough out of the airways.  this ion channel regulator works by increasing chloride secretion, inhibiting sodium absorption as well as increasing the beat frequency of the tiny hairs, or "cilia," lining the airways move to clear mucus.  These combined effects improve airway hydration and assist in the clearing mucus. the drug is different from various other CF medications, which predominantly treat the symptoms rather than the fundamental causes.

Welcome to the first large, phase 3 trial of its kind that is helping cystic fibrosis patients breath a little easier. "Abnormal ion transport and defective mucociliary clearance are fundamental defects that contribute to complications of CF lung disease, including mucus plugging, chronic bacterial infection, inflammation and progressive airway damage," Dr. Accurso added.  "although currently available drugs target these complications, denufosol was designed to treat the underlying defects that cause the complications, and could potentially modify the course of the disease, particularly when administered early in the disease process."

Researchers called upon 352 cystic fibrosis patients above the age of 5 years.  they were enrolled 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.  Patients had their exhalation rates and lung volume measured – in addition to adverse effects, such as cough, congestion, fever and sinusitis – throughout the study.

At the conclusion of the 24-week period, researchers determined patients who received denufosol had better lung exhalation rates than those placed in the placebo group, whose exhalation volumes remained comparatively unchanged from the commencement of the study. Both groups had comparable numbers and types of adverse events, with the denufosol patients experiencing notably less headache occurrences and lower rates of sinusitis and runny nose.Children under five years of age were excluded, although researchers plan on addressing the use of denufosol in the adolescent population.

"considering the evidence that early inflammation and infection results in impaired lung function and structural damage in early childhood, future studies of the effects of denufosol during the first 5 years of life is warranted," Dr. Accurso concluded.

A similar phase 3 trial – the second in fact – is set to incorporate an extended time period for the placebo-controlled treatment phase, which will help scientists determine the effectiveness of denufosol in patients with CF.

SOURCE: the American Thoracic Society’s American Journal of Respiratory and Critical Care Medicine,  December 2010

Source: Ivanhoe Newswire

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