CFTR Modulator Drugs and Inflammation in children with CF
Research type
Research Study
Full title
Identifying the effects of CFTR Modulators on the biochemical signalling pathways involved in inflammation in children with cystic fibrosis
IRAS ID
304246
Contact name
Daniel Peckham
Contact email
Sponsor organisation
The Secretariat, University of Leeds
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Cystic Fibrosis is a life-limiting inherited condition of the lungs, digestive, and reproductive systems. It affects 1 in 2500 people and the gene causing it is carried by 1 in 25 people on the UK. The Cystic Fibrosis Transmembrane Regulator (CFTR) protein channel is responsible for moving Chloride and Bicarbonate in and out of cells and with this Sodium and then water follows. In Cystic Fibrosis, the CFTR channel does not work properly leading to thick mucus secretions, which is particularly crucial to lung function. The thick, difficult to clear, mucus in the lungs leads to recurrent lung infections, damage to the lung tissue and end stage respiratory failure if left untreated.
Recent advances in CF have led to the development of CFTR Modulator drugs that improve the function of this channel and cause stabilisation and improvement of lung function and chest symptoms.
There has recently been a link between the CFTR protein, and the Epithelial Sodium Channel (ENac) established. This Sodium Channel causes an exaggerated inflammatory (swelling) response (NLRP-3 Inflammasone) without infective triggers being present.
Further studies have shown that CFTR modulator drugs may also influence this pathway and reduce the inflammatory process in CF. This has been achieved by monitoring levels of pro-inflammatory markers (IL-18, IL-1b, caspase-1 activity and ASC speck release) from patients before and after starting these new therapies.
This hypothesis has not been tested in children. With this study we aim to take blood samples from children with Cystic Fibrosis immediately before and at 3 months following the commencement of CFTR Modulator drugs. The blood tests would be taken alongside samples needed for routine care therefore there would be no need for additional painful procedures in these children.REC name
South Central - Oxford C Research Ethics Committee
REC reference
22/SC/0344
Date of REC Opinion
1 Sep 2022
REC opinion
Favourable Opinion