Are We Close to a Cure for Celiac Disease?

Q: Have scientific advances been made in the search for a cure or treatment of celiac disease?

According to Dr. Alessio Fassiano, director at the Center for Celiac Research and Treatment at Massachusetts General Hospital, Boston, pharmaceutical companies were not interested in developing drugs for celiac disease until about 15 years ago. At the time, researchers knew that for those with the condition, consuming gluten — a protein found in wheat, rye and barley — caused damage to the small intestine. But they didn’t understand how or why gluten had this effect. Dr. Fasano stated that there was a simple way to manage celiac diseases: eat gluten-free.

For the estimated 1 percent of peopleFor people with this autoimmune condition, avoiding gluten is the best way to prevent small intestine damage. the various symptoms of the diseaseThis can include nausea, diarrhea, constipation and depression.

But consuming even minuscule amounts of gluten — just a bread crumb from a cutting board, for example — can re-trigger symptoms and intestinal damage. Dr. Fasano stated that maintaining a gluten-free diet for life is difficult in an environment full of hidden gluten-containing foods. This makes it risky to eat out, travel, and go to school.

In a survey published in 2014341 people with celiac diseases rated the burden associated with managing their condition as worse than those with chronic acid reflux or high pressure. This is similar to those who have diabetes or kidney disease that require dialysis. Many people with celiac disease cannot avoid gluten. 30 percent of people with celiac disease still have symptoms, said Dr. Elena Verdú, a professor of gastroenterology at McMaster University in Ontario, Canada.

Gluten-free food can also be available more expensive than their gluten-containing counterparts, and many people don’t have access to the support of a dietitian to help them plan a balanced, gluten-free diet, Dr. Verdú said.

As it’s become clearer that maintaining a gluten-free diet is neither simple nor satisfactory for many celiac patients, researchers have also made recent strides in grasping how the disease works. We now understand “almost step-by-step the march, the progress from the moment in which you break down gluten to the point in which you destroy your intestine,” Dr. Fasano said. “An entire world opens up in terms of new treatments.”

There are currently 24 potential therapiesAccording to the Celiac Disease Foundation there are different stages of development. Dr. Fasano stated that different stages of the disease path are being targeted by the enzymes being tested. Some enzymes help to break down gluten into smaller and less harmful pieces. Other approaches make the lining of the small intestine less porous, so that it’s more difficult for partially digested gluten to enter the body. Dr. Fasano stated that other approaches target the immune system to stop it from damaging the intestinal lining in response to gluten.

If proven safe and effective, these potential therapies probably would not be cures for celiac disease or “a free ticket for high-gluten consumption,” but they could mitigate the effects of accidentally eating small amounts, Dr. Verdú said.

That being said, they’re still likely at least a few years away from being approved for use. “Drug design and approval is a really very lengthy path,” said Dr. Verdú, whose clinic is participating in several trials but who does not have any financial ties to the drugs.

Of the potential therapies in development, the one furthest along — currently being tested in a Phase 3 trial — is a drug called larazotide, which decreases the porosity of the small intestine and has shown promise from earlier trials. Larazotide could be approved in the best case scenario and put on the market within two to 3 years, according to Dr. Fasano. He was involved in the development of the drug, and has a financial stake in it.

He added that only one or two of the five to six drugs in Phase 3 trials will be approved. Dr. Fasano indicated that several other potential therapies are currently in Phase 2 trials. This could mean that it could take up to six years for them to be on the market.

Celiac therapies will vary in cost. The larazotide and digestive enzyme treatments are relatively cheap — they “cost cents to produce,” Dr. Fasano said — but drugs targeting the immune or inflammatory response would be more expensive.

Dr. Fasano stated that vaccine-like treatments for celiac disease are being explored. These therapies would teach the immune system how to tolerate gluten. He called this approach the “holy grail” because it could allow people to safely consume larger amounts of gluten. Phase 2 trials of one such therapy was discontinued in 2019It was ineffective. Still, Dr. Fasano said, “we hold a lot of hope in this approach.”

With so many different types of therapies in the pipeline, Dr. Verdú said she hopes to eventually have several medications, some of which may be used in combination, to offer to her celiac patients. Dr. Fasano speculated that they might be beneficial for other autoimmune conditions. In one recent studyFor example, larazotide was found to be helpful in a few children with Multisystem Inflammatory syndrome in Children (MIS-C). The drug is currently being studied in a laboratory. Phase 2 trial.

Source: NY Times

Leave a Reply

Your email address will not be published.

Back to top button