For a number of years, an American company called ImmusanT has been working on a potential vaccine to protect celiac sufferers from the effects of exposure to gluten and the gastrointestinal symptoms that can result such as diarrhea, abdominal pain and bloating.
The Massachusetts biotech firm says it has completed its first phase 1b trial of Nexvax2.
As many of us are aware, celiac disease is remarkable among chronic diseases in that it is very common but has no proven, approved treatment beyond dietary restriction. It affects around 1% of people – though an estimated 90% remain undiagnosed. In most celiac cases, the disease is thought to be caused by a mutation in the HLA-DQ2 gene, which is involved in immune regulation.
Developing effective treatments is important as, despite being on the gluten-free diet, around a third of people with celiac disease still suffer from symptoms as well as damage to the cells lining the intestines – often without even realizing it.
This is rather complex, but ImmusanT’s big idea is to use three “peptides” (an amino acid-containing compound within our bodies) as an immunotherapy that it hopes will encourage the T cells involved in the inflammatory reaction in celiac disease to become tolerant to gluten. After a first course of the vaccine, to induce tolerance, the company hopes that this tolerance can be maintained by periodic re-injection with the vaccine.
It’s a case of “so far, so good”, with the phase 1b trial in 38 patients revealing no concerns about safety or tolerability and showing that the immunotherapy seemed to have the desired effects on the immune system.
The study also allowed ImmusanT to select a dosing regimen for planned phase 2 trials that will see if Nexvax2 can be used alongside a gluten-free diet to protect patients when they are accidentally exposed to gluten, which ImmusanT sees as the quickest route to approval in the United States.
Depending on the results, a follow-up program is planned that will focus on an immunotherapy that could do away with the need for the gluten-free diet entirely. The company is also developing a companion diagnostic for the vaccine which could guide its use and help improve diagnosis rates.