An international research collaboration, including scientists from Dundee University, has described a new approach for the treatment of cystic fibrosis.
In a study published in a prominent medical journal, Dundee scientists helped demonstrate how the combination of two drugs reversed the key features of cystic fibrosis in a pilot trial.
Study in Dundee has been supported by the Wellcome Trust since 1991 and initially by the UK Cystic Fibrosis Trust.
Researchers found the two drugs given together reduced inflammation in nine out of 10 patients’ airways and dramatically reduced levels of salt in sweat.
Salty sweat is a feature of cystic fibrosis that can place a baby into therapy for life.
Researchers hope to set up a large-scale trial to prove the drugs’ effectiveness across a wider group.
Dr Anil Mehta, a clinical reader at Dundee’s Medical Research Institute, has been working on CF for more than 20 years and described the findings as an extremely encouraging first step.
He said: “The results suggest it might be possible to arrest the disease.
“Obviously we are still at an early stage but if these results are replicated in a placebo-controlled clinical trial, then I believe it could be a potential game-changer. However, we will need more support to make this happen.
“These drugs are already licensed, and being off-patent means the cost of developing a drug therapy should not be prohibitive.”
Cystic fibrosis is the most common inherited disease in the UK, affecting more than 10,000 people.
About one in 25 of the population in the UK carry a mutation in a gene called CFTR, which makes it hard for the body to efficiently fight off infection by bacteria, particularly in the lungs in CF-affected children.
As a result of screening in Scotland since 2003, it is known that in Scotland one child in every 2,345 is born with CF.
Dr Mehta’s long-term ambition is to develop drugs to give to babies soon after diagnosis to prevent their lungs declining.