Discovered Molecule May Prolong Life of Cystic Fibrosis Patients, Researchers Say

Discovered Molecule May Prolong Life of Cystic Fibrosis Patients, Researchers Say

Scientists at Queen’s University Belfast discovered a new molecule with the potential to prolong the life of individuals with cystic fibrosis (CF). The molecule, QUB-TL1, may be a potential treatment option for patients with CF, by altering cellular ion channels and leading to improved airway hydration and significantly increased mucus clearance.

The study, “Inhibition of Protease-ENaC Signaling Improves Mucociliary Function in Cystic Fibrosis Airways,” was published in the American Journal of Respiratory and Critical Care Medicine.

CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. In people with CF, a defective gene (CFTR gene) causes a thick, buildup of mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage, and eventual respiratory failure.

Patients with CF have a reduction in airway surface liquid (ASL) height, which compromises mucociliary clearance, favoring mucus plugging and chronic bacterial infection. Inhibitors of the epithelial sodium channel (ENaC) have therapeutic potential in CF airways, as they can reduce the hyper-stimulated sodium and fluid absorption to levels which can restore airway hydration.

The new study, funded by the Cystic Fibrosis Trust, was conducted by a team of researchers from Queen’s University and colleagues from the Royal College of Surgeons in Ireland and the University of North Carolina. It aimed to determine if a novel compound (QUB-TL1) designed to inhibit protease/ENaC signaling in CF airways can restore ASL volume and mucociliary function.

The researchers found that the QUB-TL1 was able to restore the ASL volume and the mucociliary function in CF airways.

Referring to the breakthrough, Dr. Lorraine Martin from the School of Pharmacy at Queen’s University Belfast said a recent news release: “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. 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.”

Ed Owen, chief executive of the Cystic Fibrosis Trust, said the organization is “thrilled with these initial findings” and is excited to see the next stage of preclinical testing begin.

“Research is the biggest single area of investment for the Cystic Fibrosis Trust and it’s wonderful to see projects like this making such positive progress in our fight for a life unlimited,” Owen said.

Pharmacological therapies currently available for CF, suit only a small subset of patients depending on the disease-causing genetic mutation.

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