Today, even in the midst of our COVID-19 pandemic crisis, celiac disease patients need treatments and a cure. The COVID-19 pandemic, as terrible and as disruptive as it is, must eventually pass. When it does, celiac disease patients will still be in need of treatments and a cure. The Celiac Disease Foundation must, therefore, persist in our work: funding disease research and advocating on Capitol Hill and at the National Institutes of Health (NIH) for increased investments in disease research and improved diagnostics. There are 3 million Americans who are counting on us to persevere and to be successful, even under these horrific circumstances. And we are. That is our mission. It has not changed.

As you know, the Foundation has organized a concerted advocacy effort in Washington, DC, to secure from NIH increased investments in celiac disease research with a focus on identifying treatment alternatives to the gluten-free diet and a cure. During this process, which has included meetings with key appropriators in the U.S. Congress, as well as leadership of the National Institute of Allergy and Infectious Diseases (NIAID) and National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), specific questions have arisen as to where an infusion of research capital by NIH might be deployed, the timing of proposed research investments, and at what dollar amounts.

In an effort to answer these questions, on March 8, 2020, at the invitation of the Celiac Disease Foundation and the Society for the Study of Celiac Disease, leading celiac disease researchers and clinicians gathered at the Faculty House on the campus of Columbia University for the First SSCD Consensus Workshop: Research Opportunities in Celiac Disease 2020. Despite COVID-19, the Consensus Workshop was attended by leadership from the nation’s premier academic research centers; Mayo Clinic, University of Chicago Celiac Disease Center, Harvard Medical School Celiac Research Group, Columbia Celiac Disease Center, and the Colorado Center for Celiac Disease, as well as multiple representatives from biopharma.

The Consensus Workshop was also attended by the scientist from NIAID responsible for coordinating NIH’s response to Congress on celiac disease.

As a stakeholder in this effort, I would like to share with you a summary of the highlights.

The Consensus Workshop was organized into three threads, each of which featured a small panel of experts who presented, and a robust Q&A led by an expert moderator. Attendees debated about unmet needs in understanding celiac disease pathogenesis, diagnosis, and management.

Pathogenesis: Celiac disease research can, and has, revealed proof of concepts for treating other autoimmune diseases because:

  • there are clear biomarkers,
  • the antigen (gluten) is known and can be introduced and removed with no long-term harm to the patient,
  • fresh tissue samples from all disease stages are readily available for analysis,
  • animal models exist that can speed testing, and
  • the patient community is large and engaged.

However, attendees acknowledged that there is limited understanding of pathways of disease tolerance and tissue destruction. This gap in knowledge is limiting the development of options for preventing celiac disease when propensity for the disease in a patient is established. As well as whether there are—as suspected by many in the research community—a variety of celiac disease phenotypes which might require differing treatments.

Diagnosis: By epidemiological standards, celiac disease is an epidemic, showing rapid growth over the last several decades. Based on child cohort studies in Finland and Colorado, there is evidence that disease penetration is deeper than commonly assumed. Researchers do not know why disease prevalence is growing. Major questions have emerged about who to screen for celiac disease and when. There is broad agreement around the screening of first-degree relatives, but not enough data to determine if there is a benefit to screening asymptomatic patients, or if mass screening is appropriate. As well, there is disagreement between U.S. and European diagnostic standards as to the necessity of a biopsy. There is also debate surrounding best practices for gluten challenge in patients and, critically important, how to meet FDA demands for “real world” clinical trial designs to secure drug approvals.

Disease Management: Many studies show that patients are not able to maintain a strict gluten-free diet due to the great difficulty of adherence, but, as we are all aware, no alternatives exist. It is known that histological recovery (villi/gut healing) is inconsistent, and it appears that younger patients recover better than older patients. Researchers acknowledge that the long-term implications of undiagnosed celiac disease are poorly understood. There was broad acknowledgement among attendees that poor understanding of the disease in the medical community and broader population, coupled with exploitation by the food industry, has confused many about the seriousness of celiac disease. There was also consensus that the lack of celiac disease academic research centers, as compared to the many for inflammatory bowel disease and type 1 diabetes, and the lack of young researchers studying celiac disease, was a major impediment. The dearth of pharma-economic data that supports appreciation of disease burden by employers, insurers, and government, and project potential returns on investment in celiac disease, has also severely limited the development of treatment alternatives.

In the end, consensus emerged around several action items required to accelerate diagnosis and the development of treatments and a cure. These are:

  • Increasing NIH funding, as academic and industry funding is not sufficient to achieve common goals.
  • Improving understanding of celiac disease as a serious medical condition in the healthcare provider community, government, and the public.
  • Developing an international database platform that links clinical, microbiome, genetic, and immunological adult and child patient data into one accessible tool which can promote mechanistic study of human disease in clinical trials. This is aligned with what has been done with TrialNet in the diabetes research community and ImproveCareNow for IBD, both of which have accelerated research in their respective fields.

There is obviously a lot in this note to digest. Please know that the Celiac Disease Foundation is committed to the implementation of these action items. If you have any questions, please reach out to me and I will make sure we have your answer. Your continued support makes all of this possible.

May you be safe and well,

Marilyn G. Geller, Chief Executive