New Drug is in Clinical Trials
Dr Robert Anderson, PhD from Australia announced that after several years of research his vaccine was ready for a Phase I trial. I remember meeting Dr Anderson a few years ago at a National Foundation for Celiac Awareness conference ,where he shared with me his goals about this research.
The study was composed of 34 celiacs, 19 people of whom received the vaccine while 15 received a placebo of saline water.
The participants followed a strict gluten-free diet and were of the DQ2 genotype (genetic profile) for celiac disease. DQ2 is the most commonly known gene for celiac and it is estimated that 80% of celiacs carry the gene.
The goal of the phase I trial was to assess the weekly injectable vaccine for safety, tolerability and efficacy. The vaccine is derived from wheat and barley and contains 3 peptides (or protein fragments) that commonly create an immune reaction.
What Were the Side Effects?
The side effects of the 19 people tested included:
GI distress, typically similar to that felt with inadvertent gluten ingestion.
One individual had to withdraw from the study because the digestive complaints were too severe.
7 out of the 19 suffered from nausea,
2 suffered from severe nausea and
2 others vomited shortly after receiving the injection.
The mechanism of the vaccine is to target the immune cells (T cells) that are specific for celiac disease and have them instead be created specifically against the vaccine. In turn these T cells, once specific immunity is developed, create an immune substance called gamma interferon, which is critical in developing a specific immunity. This mechanism would be similar to the one used in allergy shots where someone is desensitized to something they’ve been reacting to. The injections are designed to prevent the typical immune reaction of the offensive substance from occurring.
As a side note, gamma interferon, when aberrant, is associated with autoimmune disease, meaning the immune system gets overstimulated to a point where it starts attacking the body itself instead of the foreign agent.
Four out of Nineteen Participants Had a Positive Response
Only 4 of the 19 participants generated these desired type of T cells, although Dr Anderson seemed pleased with this response, since he considered the trial a success.
There are considered to be over 60 peptides (pieces of protein) that are responsible for the reactions against gluten. This vaccine includes only 3 peptides, but its creators feel that these account for the majority of the negative reactions that are seen.
As mentioned above, this particular vaccine is only applicable for those who carry the DQ2 genes. In regard to celiac disease, we know that patients with the gene DQ8 can also develop the disease and it is thought that many other genes are involved as well. DQ2 is the most prevalent so it definitely made sense that a gene specific vaccine would be created for it, but the vaccine would not be universally effective on all celiacs.
Of course this vaccine does nothing for those suffering from gluten sensitivity, who have a different genetic profile. But gluten sensitivity is a newer player in the gluten intolerance arena, so no big surprise that a vaccine would focus on those with celiac disease.
With 4 individuals out of 19 generating the desired T cell response, that puts the “success” rate at 21%. I personally wouldn’t consider that a success, but I’m often surprised at what is acceptable in the drug and vaccine field.
While the trial was considered successful, even if all goes according to plan there would not be a vaccine on the market before 2017.
So what are my personal conclusions? From the science perspective I share Dr Anderson’s excitement in developing a mechanism that causes the immune system to react in the way he had envisioned it.
But from a clinical perspective I have concerns. There has never been a drug or vaccine with zero side effects and I’m sure this one, should it make it to market, will be no different. What will these side effects be and how long will the vaccine need to be on the market before they are realized? No one yet knows.
Is a 21% Positive Outcome Really Considered Effective?
The “effectiveness” according to this trial was 21% of those who received the vaccine. But remember that the vaccine is only appropriate for about 80% of the celiac population. With its current efficacy, a population of 100 people would only find 17 potentially benefiting from this vaccine, excluding any side effects.
With No Drug on the Market, What Should We Do in the Meantime?
So where does this leave us? No different really than we were before this data came out. The truth of the matter is, that at this point and likely for several years into the future, our goal must be timely diagnosis of celiac disease and gluten sensitivity, gluten elimination, and an effective program to heal the gut while reversing as much gluten-induced damage as possible.
This is what we specialize in at my clinic HealthNOW Medical Center. Since writing our book The Gluten Effect” we have come to realize the overwhelming demand for a clinic that would provide such a service. In response to that need we created a destination clinic so that we could see patients from across the country as well as internationally. This has been very successful and our destination clinic patients have enjoyed dramatically improved their health.