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For those with celiac disease, even a little bit of gluten can cause damage to the intestine.
The only available solution is a strict gluten-free diet.
Yet even when foods are carefully scrutinized, accidents can be made, and symptoms can persist in up to 40 percent of patients.
Better options are in the pipeline.
Several new pharmaceuticals are being investigated by drug companies, and one just passed a phase 2 clinical trial.
The medicine is called FB102, which admittedly doesn't sound very glamorous, but don't judge a book by its cover.
This intravenous medicine has the potential to protect the gut against accidental gluten exposure, a feat that no approved drug can currently achieve.
The company that owns the pharmaceutical, argenx SE, has now announced positive topline results from its phase 2 clinical trial.
The results are not yet peer-reviewed, but patients with celiac disease who were treated with FB102 demonstrated better preservation of the intestinal lining during gluten exposure compared to a placebo.
"These positive Phase 2 results strengthen our conviction in the potential of FB102 to address a significant unmet need for people living with celiac disease, where there are currently no approved therapies," says Luc Truyen, the chief medical officer at argenx.
The recent clinical trial enrolled 126 patients with confirmed celiac disease, who were symptom-free and who had been on a strict gluten-free diet for at least the past year.
Participants underwent a controlled, eight-week oral gluten challenge. Some were randomized to receive a placebo during this time, while others received intravenous infusions of FB102.
Neither participants nor researchers knew who was receiving what.
At day 78, researchers analyzed how much gluten-induced damage had occurred in the mucosal lining compared to at the start of the trial.
When people with celiac eat gluten, it tends to shorten the tiny projections that stick out from their intestinal walls while also deepening the grooves between them.
But FB102 seems to protect against this.
An illustration comparing healthy intestinal villi (left) with the flattened, damaged villi associated with celiac disease (right). These tiny, finger-like projections help the small intestine absorb nutrients. (Koh et al., _Future Generation Computer Systems_ , 2019)
Participants in the trial who received this novel drug showed fewer signs of injury from eating gluten. They also showed a smaller inflammatory immune response in the mucosal lining.
This does not mean that FB102 will one day allow patients with celiac disease to eat gluten without care.
This drug was given under controlled conditions, and it's unclear if its protective effects work in a real-world setting.
Still, it's worth investigating further, especially as there is currently no way to protect the gut against accidental gluten exposure.
"Combined with the consistent safety profile observed across studies," says Truyen, "these findings support advancing FB102 into Phase 3 development and further exploring its broader potential across immune-mediated diseases."
The FB102 drug has been in clinical trials for several years now. It was specifically designed to target a receptor on immune cells called CD122.
This receptor responds to inflammatory messengers, and in 2009, researchers showed that if this receptor was blocked in mice with gut autoimmune issues, it led to a reversal of intestinal damage.
That led to the hypothesis that CD122 and its interaction with inflammatory signals are critical in the development and maintenance of celiac disease.
To that end, drug researchers at Forte Biosciences Inc (later acquired by argenx) developed FB102.
This drug is designed to block CD122 receptors on only some disruptive T cells, while preserving the receptor on other ones.
It shows promise in treating not just celiac disease, but other autoimmune diseases, too, like vitiligo.
This is a rare chronic autoimmune condition that causes skin to lose its pigment in patches.
Researchers suspect the drug could also work for alopecia areata.
But there's still more work to be done.
According to the United States Food and Drug Administration's (FDA) draft guidance, a chronically administered compound like FB102 would need to show efficacy in clinical trials for at least 52 weeks, and it would need to demonstrate efficacy without a gluten challenge.
Researchers at argenx are still working up to that.
"The study builds on the Phase 1b trial, providing consistent clinical evidence that CD122 blockade can protect against gluten-induced intestinal damage and prevent emergence of patient symptoms, even under a more intense gluten challenge, validating CD122 as a promising therapeutic target in celiac disease," says Truyen.
This article was fact-checked by Fiona MacDonald and edited by Fiona MacDonald. While we pride ourselves on our process, we are only human. If you spot a mistake, please let us know.
