Cure for celiac disease is distant, but science invests in quality of life

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Has scientific research made any progress towards curing or treating celiac disease?

Until about 15 years ago, pharmaceutical companies showed little interest in developing drugs for celiac disease, said Alessio Fasano, director of the Center for Celiac Research and Treatment at Massachusetts General Hospital in Boston.

At the time, researchers knew that for people 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. And, according to Fasano, it appeared that there was already a simple way to control celiac disease: adopt a gluten-free diet.

For the roughly 1% of people who have this autoimmune condition, avoiding gluten is the only method to prevent damage to the small intestine and alleviate the various symptoms of the disease, which can include abdominal pain, diarrhea, constipation, depression, fatigue, headache, blistering rash, and iron deficiency anemia.

Consuming minuscule amounts of gluten — just a crumb of bread off a board, for example — can reactivate symptoms and intestinal damage. And maintaining a strict gluten-free diet in a world full of hidden ingredients that contain the substance requires constant vigilance and makes eating out, traveling and going to school risky and anxiety-provoking, Fasano said.

In research published in 2014, 341 people with celiac disease rated the effort to manage their condition as worse than those who had chronic acid reflux or high blood pressure, and similar to those living with diabetes or kidney disease that required hemodialysis.

Despite trying to avoid gluten, up to 30% of people with celiac disease still have symptoms, said Elena Verdú, a professor of gastroenterology at McMaster University in Ontario (Canada).

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

As it has become clearer that maintaining a gluten-free diet is neither simple nor satisfying for many celiac patients, researchers have also made recent advances in understanding how the disease works. We now understand “almost step-by-step the progress from the time you digest gluten to the point where it destroys your gut,” Fasano said. “A whole world opens up in terms of new treatments.”

There are 24 potential therapies at various stages of development, according to the Celiac Disease Foundation. Those being tested target different stages in the course of the disease, Fasano said. Some are with enzymes designed to improve the digestion of gluten, breaking it down into smaller, less harmful fragments.

Other approaches make the lining of the small intestine less porous, so it is more difficult for partially digested gluten to enter the body. Still others target the immune system to prevent it from damaging the gut in response to gluten, Fasano said.

If proven safe and effective, these potential therapies would likely not be cures for celiac disease, but they could mitigate the effects of accidentally eating small amounts, Verdú said.

Still, they are probably at least a few years away from being approved. “Drug design and approval is a really long way to go,” said Verdú, whose clinic is participating in several trials but has no financial ties to the drugs.

Of the potential therapies in development, the most advanced — now being tested in a Phase 3 study — is a drug called larazotide, which decreases the porosity of the small intestine. At best, larazotide could be approved and on the market within two to three years, said Fasano, who participated in the drug’s development and has a financial interest in it.

But, he added, for every five or six drugs tested in Phase 3, only one or two will be approved. Several other potential therapies are now in Phase 2 trials, which could take five to six years to enter the market, Fasano said.

The cost of celiac therapies can vary. Treatments with larazotide and digestive enzymes are relatively inexpensive — “they cost pennies to produce,” Fasano said — but drugs that target the immune or inflammatory response would be more expensive.

Luiz Roberto M. Goncalves

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