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School of Pharmacy researchers discover new treatment opportunity for cystic fibrosis lung disease

25/04/2016


A ground-breaking programme of work led by Dr Lorraine Martin, and funded by the Cystic Fibrosis Trust, UK has recently been published in the prestigious American Journal of Respiratory and Critical Care Medicine.

This pioneering investigation of a novel compound with potential to treat cystic fibrosis (CF) lung disease, has involved a team of scientists within the School of Pharmacy, notably Drs Lorraine Martin and James Reihill, alongside colleagues at the Royal College of Surgeon’s in Ireland and the University of North Carolina.

The development of lung disease represents the major cause of morbidity and mortality in CF and occurs due to aberrant ion channel function and dehydration of the airway surfaces. Reduced airway surface liquid levels in turn contribute to the build-up of mucous and predisposes the individual to chronic bacterial infection. The ensuing cycles of chronic infection and airway inflammationcause progressive destruction of the airways, which is ultimately fatal.

The group have developed a novel protease inhibitor which reduces the activity of a critical ion channel (the epithelial sodium channel; ENaC) resulting in improved airways hydration and significantly increased mucociliary clearance, which are essential to keep the airways free of infection. Dr Martin said, “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”.

Currently available pharmacological alternatives are only suitable for a small subset of patients depending on the disease-causing genetic mutation.

An image of research findings from Dr Lorraine Martin's work

Image above: Confocal microscopy image showing the fluorescently labelled airway surface liquid layer (red) augmented at the extracellular surface of primary CF airway epithelial cell cultures (green) in the presence of compound.

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