Celiac disease could be reversed by promising nanotechnology treatment, clinical trial suggests

New nanoparticle technology has the capability to induce immune tolerance in people who suffer from celiac disease and could potentially allow them to tolerate gluten in their diet, according to results from a phase-two clinical trial of the treatment.

After receiving treatment with the nanoparticle technology for a week, the patients were able to consume gluten for 14 days with about a 90 percent reduction in immune inflammation. The results suggest that the treatment could protect patients’ small intestine from gluten exposure.

“Celiac disease is unlike many other autoimmune disorders because the offending antigen (environmental trigger) is well known — gluten in the diet,” said Dr. Ciaran Kelly, professor of medicine at Harvard Medical School and director of the Celiac Center at Beth Israel Deaconess Medical Center. “This makes celiac disease a perfect condition to address using this exciting nanoparticle induced immune tolerance approach.”

The technology was developed in the lab of Stephen Miller, a professor of microbiology and immunology at Northwestern University Feinberg School of Medicine, who has been refining the new technology for decades.

Celiac disease is an autoimmune disorder that affects only about one percent of the population, and thus far there has been no effective treatment. Immune suppressants can provide some relief for those with autoimmune diseases, but they can have toxic side-effects.

The new nanoparticle treatment functions quite differently. Instead of suppressing the immune system, it reverses the course of celiac disease.

The nanoparticle acts like a Trojan horse, hiding the allergen in a friendly shell, to convince the immune system not to attack it. The nanoparticle is loaded with allergens and then injected into the bloodstream, and the immune system then interprets the particle as innocuous debris. A macrophage (a cell that clears cellular debris and pathogens from the body) then consumes the nanoparticle, and, in turn, the hidden allergens.

“The vacuum-cleaner cell presents the allergen or antigen to the immune system in a way that says, ‘No worries, this belongs here,’” Miller said. “The immune system then shuts down its attack on the allergen, and the immune system is reset to normal.”

In this particular trial geared at treating celiac disease, the nanoparticle, CPN-101, was filled with gliadin, the major component of dietary gluten. After one full week of treatment, patients were fed gluten for 14 days.

The patients treated with the nanoparticle showed 90 percent less immune inflammation response than untreated patients.

“This is the first demonstration the technology works in patients,” said Miller, the Judy Gugenheim Research Professor of Microbiology and Immunology. “We have also shown that we can encapsulate myelin into the nanoparticle to induce tolerance to that substance in multiple sclerosis models, or put a protein from pancreatic beta cells to induce tolerance to insulin in type 1 diabetes models.”

When a celiac patient eats gluten without the nanoparticle treatment, it causes immune responses to gliadin, which results in significant damage to the small intestine.

By eliminating the inflammatory response, the CPN-101 nanoparticle is able to protect the intestines from injury that results from gluten consumption.

The CPN-101 nanoparticle is the first viable treatment for celiac disease.“Doctors can only prescribe gluten avoidance, which is not always effective and carries a heavy social and economic toll for celiac patients,” Miller said. The nanotechnology was licensed to COUR Pharmaceuticals Co., and the biotech company was granted fast track status from the U.S. Food and Drug Administration to go through clinical trials.

Takeda Pharmaceuticals announced Tuesday that it acquired an exclusive global license to develop and commercialize the celiac treatment, and COUR will focus on developing clinical trials on iterations of the nanotechnology which would be used to treat peanut allergy and multiple sclerosis.

This story was reported from Los Angeles.