There is no cure for the hereditary disease cystic fibrosis, which is characterized by the buildup of thick, sticky mucus in the lungs. A Queen's University Belfast-led team has identified a new molecule that could normalize mucus clearance, improving CF patients’ quality of life and potentially life expectancy.
Defective airway clearance mechanisms in CF patients cause mucus to build up in their lungs, predisposing them to chronic bacterial infection. Recurrent infections and airway inflammation result in the progressive destruction of the airways and ultimately, death. Scientists from Queen’s University Belfast, the Royal College of Surgeons in Ireland and the University of North Carolina discovered that a protease inhibitor that affects the epithelial sodium channel, ENaC, could reduce the frequency of these infections.
The work was conducted in human primary airways cells collected from nasal brushings of CF patients, Dr. Lorraine Martin, senior lecturer in molecular pharmaceutics at Queen’s University Belfast School of Pharmacy, told FierceBiotechResearch in an email. It shows that the inhibition of ENaC-activating proteases can increase airways hydration and normalize mucociliary clearance, she said. The findings are published in the American Journal of Respiratory and Critical Care Medicine.
Current CF drugs, such as Vertex Pharma’s ($VRTX) Kalydeco, only work in the 5% of CF patients who carry a particular CF mutation. And while the combo drug Orkambi could be effective in patients with the most common CF mutation, England’s NICE has not approved the therapy. The protease inhibitor works independently of CF mutation, and so, represents a novel drug development opportunity that applies to all CF patients, Martin said.
The team’s next steps include preclinical testing of compounds that target ENaC as well as trying them out in other chronic airway diseases, such as chronic obstructive pulmonary disease (COPD), Martin said.
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