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Small molecule, big deal

Research could pave the way for drug that helps reduce excess mucus in lungs.

A research project funded by the Cystic Fibrosis Trust has identified a small molecule that can ultimately improve the clearing of excess mucus from the lungs.

Dr Lorraine Martin and her team at Queen’s University Belfast (QUB) also discovered that the molecule has beneficial effects against products released by bacteria (Pseudomonas aeruginosa) which normally cause tissue damage.

The next stage is to develop a drug based on this molecule, which will then have to undergo rigorous safety and efficacy testing, including clinical trials.

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.”

Dr Janet Allen, Director of Strategic Innovation at the Cystic Fibrosis Trust, said: "The scientific approach by this team of researchers is very novel as they have targeted the way the ENaC channel is activated and so effectively inhibit its action. The work is an example of the rich and diverse approaches to beat cystic fibrosis for all, and also provides a great example of how basic academic work can be translated to the benefit of people with cystic fibrosis.

"Of course, this new approach now needs to move from the laboratory to clinical trials and so is not  available to use right now. However, ENaC is the target for a number of researchers and companies. I think everyone now believes there will be no 'magic bullet' for CF so having as many 'shots on goal' is excellent news."

This work was presented by Dr James Reihill, who worked on the project, at a number of national and international meetings which included the annual European and North American Cystic Fibrosis conferences and was recognised by a number of awards. Dr Martin also presented this work at the Trust’s Sixty Five Roses Club formal dinner, highlighting how donations are enabling cutting-edge research.

This research has recently been published in the American Journal of Respiratory and Critical Care Medicine.

The Trust invested nearly £200,000 into this research over three years, as part of its commitment investing in ground-breaking research to create a brighter, healthier future for everyone affected by the condition.