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Cystic fibrosis drugs may treat celiac disease- research suggests

Celiac disease is an autoimmune disease that is very common all around the world. Only in the US, 1 in 141 people is suffering from celiac disease. This condition triggers when the patient consumes any food with gluten it. Gluten is a protein that is naturally a part of wheat, rye, barley, bread, pasta, and all baked foods. (Click here to read more about it.)

Any person who has celiac disease is unable to eat anything that has gluten in it. It makes the body’s immune system to attack the mucus linings of intestines and trigger the disease symptoms. For many people, these symptoms appear as bloating, nausea, vomiting, pain, and diarrhea.

The current medication is unable to completely overcome celiac disease and the patients are advised to avoid taking gluten. This new research published in The EMBO Journal finds a better medication for celiac disease. To everyone’s surprise, these are the same medicines that are recommended for cystic fibrosis. (Click here to read the complete research findings)

The research study was conducted in the San Raffaele Scientific Institute in Milan, Italy in collaboration with the University of Naples Federico II in Italy and Paris Descartes University in France.

What is notable about this research?

The research team explains how the disease starts and progresses. The celiac disease is three times more prevalent in patients of cystic fibrosis. This is a medical condition in which the thick mucus layer accumulates inside the lungs and intestines of a person.

This occurrence of these two conditions at a time shows that there is a hidden linkage between these two, however, it’s on a molecular level. A person gets cystic fibrosis due to the gene mutations. These genes are usually those which encodes a protein called cystic fibrosis transmembrane conductance regulator (CFTR).

This cystic fibrosis transmembrane conductance regulator or CFTR works as an ion transport protein for mucus fluid. Any mutation to this gene will add abnormality in mucus fluid; it will make it stickier, causing clogs.

The same genetic mutations of cystic fibrosis transmembrane conductance regulator also activate the immune system which starts to show reactions in the lungs and intestines. All these changes take place inside people who are gluten sensitive i.e. celiac disease patients.

The researcher team calls it their motivational reason to study the molecular chain patterns between these two conditions in detail. The similarities between these conditions interested them to discover any new information for a better disease control.

The research project is on human cell lines from gluten intolerant people that identifies a peptide called P31-43 binds to CFTR. This binding inhibits its function and ends up causing cellular stress and inflammation. This is a clear sign that cystic fibrosis transmembrane conductance regulator (CFTR) has a crucial position in gluten sensitivity.

Drugs that are CFTR potentiators may also treat celiac disease

For next, the research team identified a compound called VX-770, which can stop P31-43 from spoiling CFTR’s function. This compound VX-770 was given to the gluten-intolerant mice. It showed a significant prevention from all intestinal symptoms in the rats.

After these results were achieved, the team replicated the same in human cell lines. The experiment showed that pre-incubation with VX-770 actually stopped the P31-43 peptide from triggering the immune response. It categorized VX-770 as a CFTR potentiator. In pharmaceutical terms, it is a compound that is given to treat cystic fibrosis in patients.

The new treatment for celiac disease

The researchers concluded that CFTR is a molecular target of gluten and it has a direct role in celiac disease’s pathogenesis. It is a clear hint that the scientific rationale for repurposing CFTE potentiator will prevent the symptoms of celiac disease. Thus it can be a potential medicine for celiac patients.

Lastly, the research team added that more clinical trials are necessary to explore the accurate dosage and administration of CFTR potentiators. Only with these trials, pathogenesis of celiac disease can be controlled and the patients will find an effective medicine without changing their diet.

 

Areeba Hussain

The author is a Medical Microbiologist and a healthcare writer. She is a post-graduate of Medical Microbiology and Immunology with distinction. She is an author of six research papers and currently working as a research associate in a Research Lab.

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