Patients with cystic fibrosis (CF) may see their lives prolonged thanks to a new treatment. Scientists have identified a molecule that could lead to increased airway-hydration and mucus clearance, with improvement in the quality of life of people affected by this disease.
The study, published in the American Journal Of Respiratory And Critical Care Medicine, was intended to test a new compound called QUB-TL1. The molecule is designed to alter cellular ion channels so that airways functions are restored in people with CF.
Chronic bacterial infections
CF patients have defective airway clearance mechanisms. Airway surface liquid levels are reduced, which leads to mucus building up in the airways and lungs and puts people at risk of chronic bacterial infection.
In their study, the scientists have shown that the QUB-TL1 molecule acts by preventing the activation of epithelial sodium channel ENaC, which influences airway surface liquid levels and airway hydration.
Tested in patients, the molecule has appeared to diminish ENaC activity, and was associated with a rise in airways surface liquid levels and a clearance of mucus.
This suggests the molecule has the potential to be an effective treatment against CF, increasing life expectancy for patients.
Independent of genes
CF is an inherited disease, but different genetic mutations can lead to it. Although some treatments can be prescribed, their efficiency will often depend on the type of genetic mutation carried by the patient. Interestingly, this new molecule works no matter the patient's genetic mutation.
"This is an important finding, which could provide a novel therapeutic opportunity relevant to all individuals with CF, as the targeting of ENaC is independent of their underlying CF mutation," lead author Dr Lorraine Martin from the School of Pharmacy at Queen's University Belfast commented.
"This strategy could prevent the significant lung damage that results from chronic cycles of infection and inflammation, with potential impact on quality of life, as well as life expectancy."