The Molecular Similarities of Coeliac Disease and Cystic Fibrosis - European Medical Journal

The Molecular Similarities of Coeliac Disease and Cystic Fibrosis

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TREATMENT of coeliac disease may now be possible using a compound originally developed for cystic fibrosis. While current remedies for coeliac disease involve avoiding the consumption of gluten, which is the main trigger of the condition, new research has investigated the molecular similarities between coeliac disease and cystic fibrosis, with a hope of finding novel treatment targets for effective therapeutics.

With the prevalence of coeliac disease approximately 3-times higher in cystic fibrosis patients compared with the general population, a research team led by Prof Luigi Maiuri, San Raffaele Scientific Institute, Milan, Italy, hypothesised that the two diseases are connected at a molecular level. In addition, they both exhibit immune dysfunction that causes changes in the intestines. The team investigated human cell lines from gluten-intolerant people and discovered that the P31-43 peptide binds to the cystic fibrosis transmembrane conductance regulator (CFTR), indicating the crucial role of CFTR in gluten sensitivity.

The researchers then focussed their attention on finding a compound to inhibit this autoimmune effect, successfully identifying VX-770, a CFTR potentiator used in the treatment of cystic fibrosis. When given to gluten-intolerant mice, VX-770 was shown to prevent coeliac disease-like symptoms; these results were replicated in human cell lines, with VX-770 pre-incubation preventing an immune response by P31-43.

The team noted that these new findings identify CFTR as a molecular target of coeliac disease pathogenesis, suggesting that CFTR potentiators can be successfully repurposed for the treatment of the autoimmune disease. With 1 in 141 people in the USA affected by coeliac disease, these findings may be of great significance for a vast number of patients for whom quality of life is reduced due to the condition. Future clinical trials are now expected to focus on oral administration of all CFTR potentiators and their ability to disrupt coeliac disease pathogenesis. If successfully approved, individuals with the disease may be able to consume gluten in the diet while experiencing no autoimmune symptoms.

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