Celiac Disease: A Significant, Unmet Clinical Need

muffinsImagine not being able to eat bread, pasta, cookies, cereal, crackers, or anything else that contains wheat. At restaurants, you may have to grill the waitstaff on ingredients and preparation methods, and you’re lucky if you can find something on the menu that won’t make you sick. Reading labels on packaged products becomes a necessary habit.

This is the everyday reality for the more than 2 million people in the U.S. affected by celiac disease. Patients with celiac disease cannot digest wheat, rye, or barley. Aside from avoiding common culprits like bread or cake, patients must also be cautious around processed foods that may contain hidden gluten, such as soy sauce and canned soups. Certain medications and the glue used in postage stamps may contain gluten.  In certain cases reminiscent of peanut allergies, patients may even be unable to tolerate gluten-free foods that have been prepared in the same kitchen as foods containing gluten.

Symptoms of celiac disease vary widely, but can include abdominal discomfort, diarrhea, vomiting, joint pain, and fatigue. The condition can lead to long-term health complications such as malnutrition and intestinal cancer. At the moment, the only way to control celiac disease is through a gluten-free diet.

A disease that affects a significant number of Americans, that requires extensive and often frustrating lifestyle modifications, and that has no approved pharmaceutical treatment: It’s an enticing opportunity for drug companies. According to a 2009 report by DataMonitor, medications for celiac disease are expected to be available by 2014. Drug sales could reach $8 billion by 2019.

A new study holds promise for developing celiac disease medications: Scientists believe they have identified three protein fragments in gluten that are responsible for celiac disease. Only blood samples from patients with a genetic predisposition to celiac disease were tested, so the study isn’t relevant to every patient with the disease. However, according to researchers, many celiac patients share this genetic background.

Although a cure for celiac disease may not be in sight, a few companies are developing drugs that could allow patients to ingest at least small amounts of gluten. Among the list is Nexpep, which partially funded the study mentioned above. The Australian company is conducting Phase I trials on a therapeutic vaccine utilizing the three protein fragments discovered in the study.

Another company in this space is Alvine Pharmaceuticals, a biopharmaceutical company focused on autoimmune and gastrointestinal disease. The company’s lead product candidate is ALV003, an orally administered combination of two proteases engineered to digest gluten in patients with celiac disease. 

Alba Therapeutics has been testing AT-1001 (larazotide acetate) in patients with celiac disease. AT-1001 has received Fast Track designation from the U.S. Food and Drug Administration. Although the company experienced a setback last October when Shire Pharmaceuticals dropped out an agreement to develop AT-1001, Alba is still moving ahead with the drug candidate. AT-1001 was in Phase IIb clinical trials as of January 2010.

Have you heard of any other treatments in the works for celiac disease? Feel free to share them in comments.