Celiac.com 05/27/2008 - People with celiac disease know all too well that the only effective treatment at present is faithfully following a gluten-free diet. There’s been a lot of talk about various therapies and enzyme treatments that would allow people with celiac disease to return to a normal diet. Talk to anyone who suffers from celiac disease and they’ll likely have a personal horror story about a time when they had an unhappy episode of cross-contamination.

So, the idea of a drug that would prevent such symptoms is appealing, and the goal, desirable. The chief cause of recurring symptoms in celiac disease is accidental gluten exposure, usually through cross-contamination. Cross-contamination doesn’t always mean food. Gluten is a common ingredient in many medicines and vitamins, and exposure in celiacs can cause diarrhea, weight loss, abdominal pain, anemia and oral ulcerations in the short-term, and myriad other problems in the long-run.

The drug AT-1001 is a good example of how the realities are playing out on the front-lines of science. AT-1001 is an enzyme therapy that has promised some degree of protection from gluten exposure in people with celiac disease.

A team of researches recently set out to assess the effectiveness of AT-1001 in preventing gluten from crossing the gut barrier by reversing the defective barrier mechanism. This required evaluating intestinal permeability between those exposed to gluten after taking AT-1001, those exposed without AT-1001, and control groups. The of intestinal function is done by gauging the absorption rates of various sugars.

Early testing of AT-1001 showed some progress and a significant rate of protection of celiac patients exposed to wheat proteins. The research team looked at 86 subjects with celiac disease. The patients were divided into three groups. The first group was given placebo AT-1011 and challenged with gluten, the second group was given either active or placebo AT-1001, while the third group was given gluten and active AT-1001.

After the first week, all subjects showed improvement, possibly due to closer adherence to a gluten-free diet. At three weeks, those given AT-1001 showed substantial improvement over the group given gluten and placebo AT-1001, including reduced intestinal permeability and fewer symptoms of gluten toxicity.

The problem is that while AT-1001 shows a degree of promise, the results are so far underwhelming. The research team noted that the degree of improvement did not match the primary study. The results are, however, strong enough to encourage researchers to conduct a larger trial of AT-1001, which is currently underway.

It’s important to remember that celiac disease is an immune disorder and no immune disorder has ever been fully cured. So, the idea of people with celiac disease being able to take a pill and head out for a night of pizza and beer without the standard celiac-related reactions is far-fetched at best. At best, such drugs would likely help to prevent cross-contamination, rather than conveying immunity to gluten proteins.

Until then, stay tuned…best of luck with the gluten-free diet!

Presented by Dr. Leffler at the 2009 Digestive Disease Week on Tuesday, May 20 at 10:45 a.m. Pacific Time in room 10, San Diego Convention Center.

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