Possible Celiac Disease Nanotechnology Treatment Breakthrough Revealed

The results of a clinic trial suggest medical nanotechnology could lead to new effective treatments for celiac disease and a rash of other ailments.

The treatment, called CNP-101/TAK-101, was developed at Northwestern University. It utilizes microscopic nanoparticles in an attempt to train the body's immune system to tolerate gluten. Celiac is an autoimmune disorder that results in a person's immune system being unable to tolerate the protein gluten, triggering inflammatory attacks on the digestive system when the substance is introduced.

In the study, researchers formed a harmless shell-like structure around a tiny amount of gliadin, a component of gluten, and administered it to celiac patients for about a week before testing their reaction to gluten for around two weeks. The phase 2 clinical trial showed a significant reduction in adverse immune response to gluten in patients who received the treatment, versus those who were given a placebo. The randomized double-blind study was relatively small, with 34 participants. Six of the patients did not complete the trial due to gluten-related symptoms.

The technology is owned by COUR Pharmaceutical Development Company, which was co-founded by one of the scientists who developed the treatment, Dr. Stephen Miller of Northwestern University. At the conclusion of the trial, Japanese pharmaceutical company Takeda bought the license for the technology's use in treating celiac disease, in a deal worth up to $420 million. COUR retains rights to the tech for use in the possible treatment of other diseases.

"This is the first demonstration the technology works in patients," said Miller in a Tuesday press release. "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."

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In addition to celiac disease, the nanotechnology-based treatment could also potentially be applied to other autoimmune disorders and allergies. Getty

Miller explains that the treatment works when the nanoparticle involved in the treatment, acting as a kind of Trojan horse, is disregarded by the a person's immune system and consumed by macrophages, a "vacuum cleaner" cell that the body uses to clear debris and pathogens.

"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."

Although the results of the study do suggest that the technology could lead to effective treatments, it seems premature to expect that a miracle cure is around the corner.

Phase two clinical trials are the first stage of human trials where treatments are tested for their effectiveness and the Food and Drug Administration states that while around 33 percent of all phase two trials display positive results, only 25-30 percent of treatments that make it to phase three are then shown to be effective and safe. If CNP-101/TAK-101 does make manage to pass those hurdles, it will still likely be several years before the general population of patients would be able to receive the treatment.

If the celiac treatment ultimately proves effective and safe, the method is expected to be developed for use in several other treatments. COUR has targeted autoimmune diseases like multiple sclerosis in particular. A wide range of allergies are also possible candidates for application of the technology.