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the body misidentifies fragments of gluten—a protein found in barley, wheat, and rye—as a foreign invader and mounts an attack. These attacks damage the gut, impair the ability to absorb nutrients, and cause abdominal pain, vomiting, and diarrhea. If left unchecked, celiac disease can cause more serious complications like anemia or osteoporosis. It affects about 1 percent of the U.S. population.
There are no FDA-approved treatments. The only remedy is to avoid gluten. Some companies, like San Carlos, CA-based Alvine Pharmaceuticals and Alba Therapeutics, of Baltimore, MD, are developing treatments to be taken in combination with a gluten-free diet. Cambridge, MA-based ImmusanT is advancing a vaccine that, if proven effective in clinical studies, would enable patients to eat gluten without getting sick.
Celimmune isn’t aiming to treat the entire celiac population with AMG-714; just the roughly 50 percent of diagnosed celiac patients, Leon says, who aren’t responding to gluten free diets. Within that group is a much smaller group with a rare condition called “refractory” celiac disease. This is when symptoms persist despite several months of gluten-free eating due to permanently damaged villi—the tiny protrusions sticking out of the intestinal walls that help the body absorb nutrients and move food through the gut.
Refractory celiac affects about 1 percent of celiac sufferers. It’s a form of lymphoma; a type of white blood cell (intraepithelial lymphocytes) keeps multiplying and invading the gut, even without being triggered by gluten.
Leon says AMG-714 might help by blocking IL-15, which is thought to play a key role in the development of the invading white blood cells that damage the intestinal lining. Cutting off IL-15 could destroy those cells and give refractory patients relief. That’s the theory, anyway.
“It’s far from proven,” Palmer cautions.
Palmer and Leon will test that hypothesis with Amgen’s drug. They won’t say when those trials will begin, but to run them Celimmune will form partnerships with some of the world’s major celiac disease centers in New York, Paris, and Amsterdam. Patients will take what’s known as a ‘gluten challenge’ to their system: eating gluten to see what the effects are.
If the patients ingest gluten for a few weeks, take AMG-714, and don’t show celiac symptoms, Celimmune believes that will be a signal the drug is having a positive effect. And with that signal, perhaps Amgen will once again take a shot on a program it shelved many years ago.