Larazotide acetate treatment of celiac disease appears to significantly reduced symptoms in a large controlled clinical trial.

Celiac disease is considered a disease of the digestive system as well as an autoimmune disease.  The disease affects people worldwide, including more than 2 million people in the United States.   It causes damage to the small intestine, which interferes with the absorption of nutrients from food and triggers an abnormal immune response to foods containing gluten. This abnormal activity involves the villi, which are small, fingerlike protrusions that line the small intestine. The villi are supposed to allow nutrients from food to be absorbed into the bloodstream, but in celiac disease, the immune system destroys or damages the villi when gluten is ingested. As a result, it’s impossible for a person with celiac disease who eats gluten-containing foods to be properly nourished, as the digestive system cannot absorb nutrients in food.

Gluten, which is a protein found in wheat, rye, and barely, may also be present in products such as medicines, vitamins, lip balms, and many processed foods. Treatments are needed because inadvertent exposure to glutens and non-adherence to gluten-free diets are common with more than 70% of individuals with celiac disease reporting continued symptoms despite dietary restrictions.”

Larazotide prevents gluten uptake by closing tight junctions in the gastrointestinal (GI) tract to inhibit transport of gluten peptides. By preventing gluten, immunogenic peptide uptake the cycle of events that includes cytokine-mediated inflammation with celiac disease is prevented. In this clinical trial 342 patients from74 research sites in North America where they were treated with one of 3 doses of larazotide or placebo and compared.

The primary goal of the study was to determine whether larazotide could improve diarrhea, indigestion and abdominal pain as measured on the Celiac Disease Gastrointestinal Symptom Rating Scale (CeD GSRS).

One dose of larazotide was found to increase the proportion of patients who reported no or very few symptoms by 31%.  Improvement for several additional end points including reductions in headache and tiredness was also observed. Patients taking larazotide experienced a 26% reduction in the number of days with severe celiac symptoms compared with those taking placebo.

According to the study author Joseph A. Murray, MD, of the Division of Gastroenterology and Hepatology at Mayo Clinic, in Rochester, Minn. “This is the first large randomized and blinded trial to associate a therapeutic agent with significant symptom reduction in celiac disease,”

The investigators believe that larazotide has the potential to become the first pharmacologic therapy for celiac disease.  The therapy is not expected to replace gluten-free diets, but to act as an adjunct because of the frequency with which patients experience symptoms despite avoiding gluten.

Reference: Wang C, Rasmussen H, Perrow W, et al. Larazotide Acetate, a First In-Class, Novel Tight Junction Regulator, Meets Primary Endpoint and Significantly Reduces Signs and Symptoms of Celiac Disease in Patients on a Gluten-Free Diet: Results of a Multicenter, Randomized, Placebo Controlled Trial. Digestive Disease Week (DDW) 2014. Abstract 929b. Presented May 7, 2014.

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