Drug Repurposing to Treat Celiac Disease Represents a Transformative Opportunity for Millions of Patients
- Jon Bari
- Dec 7, 2025
- 6 min read
Updated: Dec 7, 2025
The Problem
According to Every Cure, approximately 300 million individuals worldwide live with diseases for which no FDA-approved treatments exist. Of the nearly 18,000 recognized diseases, only around 4,000 have available therapies, and millions more contend with treatments that are financially burdensome, highly complex, or limited in accessibility.
The Celiac community knows this struggle all too well. Despite some advances in diagnosis and management, the only available treatment for Celiac remains a restrictive lifelong Gluten Free diet, which is highly burdensome, very costly, challenging to maintain, and insufficient for many patients who continue to experience symptoms and mucosal damage. A therapy capable of directly addressing the genetic and immunologic basis of Gluten reactivity would represent a transformative advance for millions of Celiac patients worldwide. This is why we believe so strongly in the opportunity that drug repurposing provides.
David's Story - Chasing His Own Cure
Dr. David Fajgenbaum is co-Founder & President of Every Cure, and one of the youngest faculty members to ever receive tenure at the University of Pennsylvania School of Medicine. Dr. Fajgenbaum is a world-renowned physician-scientist at Penn Medicine, bestselling author of Chasing My Cure, inspirational speaker, and survivor on a mission to save lives by using AI and relentless hope to unlock hidden cures. David's story is compelling:
"While battling for my life in the ICU, a seemingly minor symptom caught my attention amidst the life-threatening failure of my vital organs. Small bumps, known as blood moles, emerged on my chest and shoulders during my relapses.
This observation became a critical piece of my medical puzzle, hinting at a deeper issue related to the VEGF and mTOR pathways. While experimenting on my own tissue I found that my mTOR pathway was turned into overdrive and I began taking Sirolimus, an mTOR inhibitor never administered for Castleman Disease before.
A couple days later I began feeling better and my blood moles started shrinking away! Leveraging existing medications for new applications can lead to rapid, effective, and often less costly solutions, especially for rare and complex diseases like mine. Now, over a decade into remission, I celebrate not just my survival but the strategy that made it possible.
As Every Cure continues to explore the vast potential of existing drugs, we want to hear from you! What other life-saving discoveries lie within our current pharmacopeia, waiting to be uncovered through the power of repurposing?"
"Drug repurposing could be the game changer we need for millions suffering from Celiac Disease!" -- Jax Bari, Age 12
Make Our Children Healthy Again Strategy Report
Celiac Journey applauds the release of the Make Our Children Healthy Again Strategy Report, which provides a policy roadmap for addressing the childhood chronic disease crisis. In 2025, Jax was invited to the White House twice to inform the MAHA Commission on its work to Make America Healthy Again for kids with chronic diet-related disease! We are grateful that the MAHA Strategy Report details policy reforms that will make a meaningful difference in the lives of 3.3 million Americans with Celiac Disease, including about 729,000 children like Jax. The MAHA Strategy Report includes language on Celiac Disease and requiring the labeling of Gluten as a food allergen:
"Food Allergies: FDA will develop guidance on diagnostics and treatments for food allergies. FDA will also make recommendations about requiring transparency in disclosures of ingredients that impact certain health conditions, such as gluten for those with Celiac disease, and other established food allergens."
In addition, the MAHA Strategy Report includes language on drug reproposing:
"Repurposed Drugs: The NIH and FDA will jointly investigate opportunities to strengthen the use of repurposed drugs for the treatment of chronic disease, while harmonizing authorization processes through collaborative clinical trial designs to achieve FDA approval."
Finding our Sirolimus for the Celiac Community
It's estimated that about 25.7 million Americans have some type of food allergy, auto-immune disease or intolerance to eating Gluten, a protein found in Wheat, Barley, Rye and most Oats (through cross-contamination with Wheat, Barley and Rye).
Celiac Disease is a potentially life threatening food allergy and autoimmune disease triggered by eating Gluten. Gluten is kryptonite to our superman. Just one crumb of Gluten can cause Jax to get violently sick for days with vomiting and diarrhea, and it can damage his small intestine. With each glutening episode, the chances of additional adverse health effects increase, including cancer.
Unlike other food allergies, one cannot outgrow Celiac – it’s lifelong. Additionally, unlike more well-known food allergies with IgE-Mediated mechanisms, there is no rescue medication (i.e., adrenaline) in the event of accidental gluten ingestion. 44% of people with Celiac Disease who follow a strict gluten-free diet still accidentally ingest gluten once a month.
For the Celiac community, drug repurposing represents a particularly meaningful opportunity. Despite the seriousness and prevalence of Celiac Disease, there are still no approved treatments that allow patients to safely manage the condition beyond lifelong, strict gluten avoidance -- which as discussed is difficult, burdensome, and often insufficient.
Repurposed drugs could help fill this gap by targeting key mechanisms involved in Celiac pathophysiology, such as intestinal permeability, immune activation, inflammation, or enzyme pathways that break down gluten. Because these drugs have already been tested in humans, early signals of efficacy in Celiac Disease can be generated much faster than with novel compounds.
Drug repurposing also democratizes innovation and provides new market opportunities to the pharmaceutical industry. It allows small research groups, nonprofit organizations, and patient-driven initiatives to pursue therapeutic leads without the enormous financial barrier of traditional drug discovery. For chronic diseases like Celiac Disease -- long underfunded and under-prioritized -- repurposing can bring new momentum, new collaborators, and new options for patients who have waited decades for meaningful progress. In short, drug repurposing is not only a smart scientific strategy; it is a hopeful and pragmatic path toward delivering the first effective treatments for the millions of people living with Celiac Disease.
The Economics in Support of Drug Repurposing: A Case Study with GSK Pulling the Plug on Celiac Disease R&D
Buried on page 33 of GSK’s Full Year and Q4 2022 Results investor and analyst presentation, the global biopharma company quietly disclosed in February 2023 that its TG2 inhibitor for Celiac Disease, 3915393, had been removed from the R&D pipeline as part of its “Innovation: R&D pipeline changes since last quarter.”
As Fierce Biotech reported, GSK terminated its Celiac Disease R&D program that it had acquired from Sitari Pharma in 2019 and which Sitari previously had under clinical development for many years.
This was such disheartening news, especially since it was reported that GSK CEO Emma Walmsley said, "I don't think there are any further plans to pursue Celiac Disease."
Sitari had launched with support from GSK and Avalon Ventures, shortly after the partners established a $495 million investment fund. The biotech secured a $10 million Series A to advance early discoveries from Stanford University researcher Chaitan Khosla, Ph.D., on TG2 inhibition. In 2020, GSK advanced Sitari's lead candidate, GSK3915393, into clinical development.
GSK is a bellwether company for the pharma industry. After GSK had acquired Sitari in 2019, it signaled to other large pharma companies that there was a valuable marketplace to pursue Celiac treatments. Since 2023, our worry has been that GSK's pullback has been a negative signal to investors and other big pharma companies.
Fierce Biotech's reporting suggested that GSK's termination of Celiac R&D is because Celiacs do not represent a large enough market opportunity for return on investment.
"It will be, again, simply a question of really putting as much discipline as we can into our capital allocation. We do that now across the full portfolio of vaccines and medicines, and we just try and look to see where we're going to not only get the right impact for patients, but the right of return on that R&D investment with the right kind of portfolio mix in terms of risks and rewards." -- GSK CEO Emma Walmsley
When I worked for Capitol Records during college, there was a general rule of thumb in the music industry that only 1 in 10 musical artists signed to a major record label would become a commercial success. That meant that it was a numbers game in finding the next big artist, and often times the 9 in 10 artists who failed to attain enough radio airplay and music sales were dropped by their label. The same general principle applies in biotech, but the stakes are much higher and can mean the difference between life and death.
Developing a new therapy and advancing it through the approval process is an arduous undertaking. It can take more than a billion dollars and longer than a decade. Nearly nine out of ten drugs that enter clinical trials do not succeed in reaching patients. Patients without any treatments do not have that much time to wait.
While we remain hopeful that some other pharmaceutical companies are successful with their limited drug development pipeline to treat Celiac Disease, we believe that the best hope for a near-term therapeutic treatment for Celiac will come from drug repurposing.
In addition, since food is our only medicine, we must all call on the FDA to take next steps from the MAHA Strategy Report and issue rulemaking now to require the labeling of Gluten grains (Barley, Rye and Oats) as Major Food Allergens, just like Gluten must declared on all food labels in 87 countries worldwide. Until there is any treatment other than a Gluten Free diet and a rescue medicine in the event of accidental ingestion of Gluten, we need Gluten labeled now.
Let's go!
