For people with celiac disease, there’s one way to avoid symptoms: don’t eat gluten. But what if a vaccine and some booster shots could let you eat all the wheat-filled food you want, without all the nasty consequences? That’s the kind of thing a Cambridge, MA-based startup called ImmusanT is trying to prove is possible.
Sometime within the next few months, ImmusanT will likely report data from two separate early-stage clinical trials for a vaccine, NexVax2, that it’s developing for celiac disease—an autoimmune disorder in which eating gluten triggers a sufferer’s immune system to attack his small intestine. Those data, from two separate studies in the U.S. and Australia, will represent the first signals as to whether ImmusanT is on to something in treating celiac disease. It’s also the first move in a broader effort to prove that the company’s concept of a peptide immunotherapy platform is viable.
“It’s a very exciting first half of the year with the data that is pending,” says CEO Leslie Williams.
Of course, even if those studies are a success, they’re really the first step down a long road for ImmusanT. These two studies are Phase 1b trials, designed to make sure NexVax2 is safe and to find a range of potential doses the company might be able to use in its next studies. There will be several clinical hurdles to come. Meanwhile, there are plenty of other companies trying to find treatments for celiac disease. Both San Carlos, CA-based Alvine Pharmaceuticals and Baltimore, MD-based Alba Therapeutics, for instance, are developing drugs that are supposed to be taken in combination with a gluten-free diet. Sitari Pharmaceuticals, a startup emerging from a joint venture between GlaxoSmithKline and Avalon Ventures, recently raised $10 million to pursue treatments for the digestive disorder.
But even with all those forces raging around ImmusanT, Williams says that the company stands out for one key reason: if its plan succeeds, celiac patients might one day be able to eat all the gluten they want.
“We are the only treatment in development to date that is disease-modifying,” she says. “Our focus is disease modification so patients can resume an unrestricted diet.”
When people with celiac disease digest gluten—a protein found in wheat, barley, and rye—their immune system mistakes certain fragments of the protein as invaders and mounts an attack. This causes inflammation and damage in the intestine, impairing its ability to absorb nutrients and leading to things like abdominal pain, vomiting, and diarrhea. Left untreated, celiac can lead to much more significant problems, such as growth delays, osteoporosis, or infertility. Celiac disease affects about 3 million people in the U.S., though only about 10 percent of them are officially diagnosed through an intestinal biopsy, Williams says.
Right now, the only real option for celiac patients is to completely avoid gluten. ImmusanT’s idea, however, is to inject patients with a vaccine containing engineered version of the three gluten fragments that trigger the immune response in most celiac patients. These peptides, each about 15 amino acids long, are supposed to train the immune system to see gluten as food, so it doesn’t trigger an attack in the gut.
The approach may sound a little counter-intuitive, given that most vaccines teach the immune system to attack the injected molecules. But Williams likens the company’s approach to
