There are currently 22 potential therapies at various stages of development, according to the Celiac Disease Foundation. Those being tested target different steps in the disease pathway, Dr. Fasano said. Some are enzymes meant 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 that it’s more difficult for partially digested gluten to enter the body. Still others target the immune system to prevent it from damaging the intestine in response to gluten, Dr. Fasano said.
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.
A drug called larazotide, which is thought to decrease the porosity of the small intestine and had shown some promise in earlier trials, was being tested in a Phase 3 trial until June, when the trial’s sponsor stopped it after an analysis found that the drug did not provide a clinically significant benefit among study participants. Of drugs tested in Phase 3 trials, about half are typically approved, said Dr. Fasano, who was involved with the development of larazotide and has a financial interest in it. Several other potential therapies are now in Phase 2 trials; which could be five to six years from market, he said.
The cost of celiac therapies would vary. 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.
Vaccine-like therapies for celiac disease, which would teach the immune system to tolerate gluten, are also being investigated, Dr. Fasano said. He called this approach the “holy grail” because it could allow people to safely consume larger amounts of gluten. A Phase 2 trial of one such therapy was discontinued in 2019 because it appeared to be 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. And perhaps they may prove useful for other autoimmune or inflammatory conditions, Dr. Fasano said. In one recent study, for example, larazotide seemed to help a handful of children with Multisystem Inflammatory Syndrome in Children, or MIS-C. The drug is currently being tested for this purpose in a Phase 2 trial.