Drug Repurposing to Treat Celiac Disease Represents a Transformative Opportunity for Millions of Patients

Dr. David Fajgenbaum and Jax Bari Met to Discuss Drug Repurposing for Celiac Disease

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."

Dr. Jay Bhattacharya, Director of the National Institutes of Health, and Jax Bari

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.