With the growing number of patients diagnosed with celiac disease, researchers are finally spending time, money and resources searching for new treatment options. A group of researchers are currently working on a vaccine to reduce the effects of gluten exposure for celiac patients. This vaccine, which just emerged from a clinical trial, has just received the green light to go into its next phase.
The manufacturer of the vaccine, ImmusanT, is a Massachusetts-based biotechnology company, focused on innovative approaches to modifying diseases in order to generate immune tolerance. ImmusanT is a pioneer in its field using a proprietary discovery platform for targeted (epitope-specific) immunotherapy, and is taking great strides in the field of celiac disease research and control.
If successful, their methods would be generalized to address other autoimmune conditions, such as type 1 diabetes. Their most recent trial was Phase 1b, which tested the vaccine's safety and tolerability at various doses, including from an initial injection, as well as through a series of booster shots.
Simultaneously, a group of scientists belonging to the University of Chicago is hard at work to determine the cause of the disease that affects millions of people around the world. After much turmoil, the group arrived at the conclusion that it is purely a genetic disorder, which means that everyone with a certain genetic makeup is prone to developing the disease after exposure to certain triggers.
This trigger, according to the group, is a viral infection called the reovirus, which is known to be harmless. What researchers want to target is the disease's genetic makeup. So, their goal is to prevent its onset and the presence of its symptom when gluten is consumed.
Nexvax2, the vaccine, is considered an innovative approach to treating celiac disease, as it consists of modifying the body’s own immune system to treat the underlying disease. The vaccine consists of small dose that are gradually increased through time and help modify the gene commonly associated with celiac disease, HLA-DQ2.5. Through time, the patient’s tolerance to the harmful protein in gluten increases without any type of negative effects.
In the most recent clinical trial, participants were divided into three groups that were given either escalating doses of the vaccine, or a placebo, followed by maintenance doses, which were higher than the ones administered to other participants in previous tests. The results from this test will be used to gauge the safety and tolerability of the drug in the subjects, in order to create a secure dosing regimen for a planned phase 2 study, which will be conducted later this year
The importance of a phase 2 study revolves around using a much higher sample in order to further determine the safety of the vaccine, as well as its effectiveness of protecting the patient from gluten exposure, and whether its benefits far outweigh the risks of using it.
The recruitment of subjects for the phase 2 trials of Nexvax2 began in late 2017. This phase is crucial for the development of any drug, and it's important to note that approximately 63 percent of drugs make it to this phase (according to the Biotechnology Innovation Organization).
While there are many alternatives being peddled as medication for the celiac disease, there is currently no real method to prevent symptoms from appearing. Consequently, the only medications for celiac patients are those that address the symptoms when they appear after consumption, accidental or otherwise, of the harmful proteins in gluten.
ImmusanT aims to address this absence by supplying an innovative drug to reduce the symptoms, and even prevent them from appearing entirely in patients after exposure to gluten.
Nexvax2 contains in its composition 3 vital peptides, which make up the smaller fragments of the entire gluten protein. The smaller peptides are meant to trigger the T-cell reaction caused when the body is exposed to gluten. T-cells are white blood cells that are created to combat foreign contaminants within the body but with celiac disease, these cells mistakenly target random structures after being exposed to gluten. The purpose of the vaccine is to safely induce the immune response in order to build a tolerance to the harmful proteins contained in gluten. The results from the latest clinical trials are meant to provide valuable insights into the dosing and optimization for this ‘cutting-edge’ drugs.
According to Leslie Williams, ImmusanT president, and chief executive officer, Nexvax2 will have the potential to protect against gluten exposure in not just celiac patients, but for everyone looking to avoid the detriments from consuming the harmful substances in the protein. In previous studies conducted with the drug, it was observed that the first dose caused the immune reaction, alongside the typical symptoms in celiac patients. However, after twice-weekly doses of the vaccine, across a period of 8 weeks, the T-cells were no longer active after each shot. After patients treated with Nexvax2 consumed gluten for 3 consecutive days, there were no negative effects.
These improvements in those struggling with celiac disease portray a bright future for the vaccine, as well as for those looking to avoid the detriments from gluten exposure
In the vaccine's roadmap, the team states that the first phase is determining its effectiveness in protecting the patient from gluten exposure. However, future plans suggest that their aim is to develop a method to completely forgo the need for a gluten-free diet.