Cystic Fibrosis Trust in UK To Co-Fund Study To Develop Solution for Dehydration

In the United Kingdom, The Cystic Fibrosis Trust along with the children’s charity Action Medical Research, are funding a laboratory test of an inhaled treatment targeting lung dehydration in Cystic Fibrosis.

Cystic fibrosis (CF) is a condition that usually affects children and young people and includes symptoms such as persistent coughing, shortness of breath, chest infection, and ultimately pneumonia.  Dehydration in the lungs can cause severe symptoms in cystic fibrosis patients, allowing damaging bacteria to grow and trigger mucus accumulation.

Estimations are that over 10,000 people in the UK and 70,000 worldwide have a CF diagnosis. The progressive lung damage that occurs makes cystic fibrosis a condition that is still associated with low life expectancy.

Although some treatments have been developed to treat the causes of CF, studies have shown that they only work in 4% of patients, making it necessary to develop new treatments targeting the biological underlying mechanisms associated with CF.

A team of researchers from the University College London led by Professor Steve Hart aims to develop an inhaled treatment that protects the lungs from becoming dehydrated and that eases young people’s symptoms, thus reducing chest infections.

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The inhaled treatment that is being tested uses a synthetic molecule called siRNA, that turns off the gene ENaC, thus protecting the lung from the dehydration. The team of researchers think that this procedure will then ease cystic fibrosis symptoms. Specialists are testing a procedure where they wrap the molecule siRNA with a mixture of lipids (fat) and peptides (proteins), so the lung can easily absorb siRNA. Laboratory tests are being conducted to formulate the medicine and to test safety and efficacy of this procedure. The project is estimated to cost £150.000, with the Cystic Fibrosis Trust contributing one-third of the amount.
In a recent press release Professor Steve Hart from the University College London, project leader, said: “We envisage that children would need to take the new medicine just once per month, by inhaling it.”

“We hope the medicine will one day help children to breathe more easily and stop them from getting so many bacterial infections — something which could dramatically change children’s lives for the better,” Professor Steve Hart added.