Q: Is there any scientific research that has made progress in curing or treating celiac disease?
Alessio Fasano, director of the Center for Celiac Research and Treatment at Massachusetts General Hospital in Boston, said that until about 15 years ago, pharmaceutical companies showed little interest in developing therapeutics. celiac disease. At the time, researchers knew that for people with the condition, the consumption of gluten — a protein found in wheat, rye, and barley — caused damage to the small intestine. But they don’t understand how or why gluten has this effect. And, Dr. Fasano said, there seems to be a simple way to manage celiac disease: adopt a gluten-free diet.
For estimate 1 percent of people In people with this autoimmune condition, avoiding gluten is currently the only method for preventing small bowel damage and soothing various symptoms of the diseasemay include abdominal pain, diarrhea, constipation, depression, fatigue, headache, skin rash, and iron-deficiency anemia.
But consuming even tiny amounts of gluten — such as breadcrumbs on a cutting board — can reactivate symptoms and damage the gut. And maintaining a strict, gluten-free diet for life in a world rife with gluten-containing ingredients requires constant vigilance and makes eating out, traveling, and going to school a breeze. risky and anxiety-provoking, says Dr. Fasano.
In one survey published in 2014, 341 people with celiac disease rated their burden of managing the condition worse than those with chronic acid reflux or high blood pressure, and were similar to those living with diabetes or kidney disease that required running kidney. Despite trying to avoid gluten, many 30 percent of people Elena Verdú, a professor of gastroenterology at McMaster University in Ontario, Canada, says celiac disease still has symptoms.
Gluten-free foods can also more expensive Dr. Verdú said.
As it becomes increasingly clear that maintaining a gluten-free diet is neither simple nor feasible for many celiac patients, researchers have also made recent strides in understanding how. activity of the disease. We now understand “almost every step of the journey, the progression from the moment you break down gluten to the moment you destroy your gut,” says Dr. Fasano. “A whole world opens up to new treatments.”
Current 24 potential therapies at different stages of development, according to the Celiac Disease Foundation. Fasano said the people being tested target different steps in the infection process. Some are enzymes to improve the digestion of gluten, breaking it down into smaller, less harmful pieces. Other approaches make the lining of the small intestine less porous, so it’s 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, says Dr.
If proven safe and effective, these potential therapies likely won’t be a cure for celiac disease or “a free ticket to consuming more gluten,” but they could minimize the effects. effects of accidentally eating small amounts, says Dr. Verdú.
That being said, they are still likely to be at least a few years away from being approved for use. “Drug design and approval is a really long road,” said Dr. Verdú, whose clinic is involved in several trials but does not have any financial ties to the drug.
Of the potential therapies being developed, one of the most distant – currently being tested in a Phase 3 testing – is a drug called larazotide, which reduces the porosity of the small intestine and has shown promise from earlier test. In a best-case scenario, larazotide could be approved and on the market within two to three years, said Dr. Fasano, who was involved in the drug’s development and has financial concerns for it. know.
However, he added, for every five or six drugs tested in Phase 3 trials, only one or two are approved in the end. Several other potential therapies are currently in phase 2 trials; Dr. Fasano said it could be five to six years away from the market.
The cost of celiac therapies will vary. Treatments with larazotide and digestive enzymes are relatively cheap – they “cost pennies to produce,” said Dr. Fasano, but drugs that target the immune or inflammatory response are more expensive.
Fasano said vaccine-like therapies to teach the immune system to tolerate gluten, are also being studied. He calls this approach the “holy grail” because it can allow people to safely consume larger amounts of gluten. Phase 2 trial of such a therapy discontinued in 2019 because it doesn’t seem to work. However, Dr. Fasano said, “we have a lot of hope in this approach.”
With so many different types of therapy in the works, Dr. Verdú said she hopes to eventually have several drugs, some of which can be used in combination, to offer her celiac patients. . And perhaps they could be helpful for other autoimmune or inflammatory conditions, Dr. Fasano said. In a recent studyeg: larazotide seems to help some children Multisystem inflammatory syndrome in children, or MIS-C. The drug is currently being tested for this purpose in a Test phase 2.