On March 30-31st, 2015, Celiac Disease Foundation Chief Executive Officer, Marilyn G. Geller, attended the FDA Public Workshop on Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics (GREAT3) near Washington, D.C. This landmark conference addressed endpoints and outcome measures for trials for products intended to treat celiac disease and Irritable Bowel Disease. The second day of this meeting focused on testimony from leading experts (including a number of members of the Foundation’s Medical Advisory Board) to establish endpoints and benchmarks to assess potential treatments for celiac disease emerging from research. Marilyn is proud to sit on the GREAT3 steering committee. Founder, Elaine Monarch, has called this FDA meeting the most important federal meeting on celiac disease in more than 20 years. This workshop indicates that great strides have been made in the recognition of celiac disease as a serious autoimmune disorder with broad implications for patients, caregivers, and society. In this conference, the FDA also acknowledged the central role that disease advocacy organizations, such as Celiac Disease Foundation, can play in assessing disease impact and in educating healthcare professionals and the public on disease symptoms and possible implications.

GREAT3 panelists, including researchers, clinicians, and FDA representatives, largely concurred that the gluten-free diet can no longer be the only treatment for the disease, and that alternatives are needed. Dr. Alessio Fasano, Medical Advisory Board Member, explained that the gluten-free diet is not the 100% effective treatment that we expected it to be, and declared that a celiac therapeutic was needed to compliment the gluten-free diet. Dr. Joseph Murray, also a CDF Medical Advisory Board Member, argued that therapies that replace the gluten-free diet for celiac disease are needed. Beyond these statements, the following points were made:

  • Celiac disease is one of the few diseases where treatment is exclusively the burden of the patient and their caregivers.
  • Celiac disease is among the best-understood and well-documented auto-immune diseases, yet there are still no approved therapies beyond the gluten-free diet.
  • More than half of celiac patients report dissatisfaction with the gluten-free diet. A large percentage refuse strict adherence to the diet.
  • Children in particular face exceptional burdens adhering to the gluten-free diet, including, limited appreciation of the risk/reward, peer pressure, and eating away from home.
  • Asymptomatic celiac patients also struggle to adhere to a strict gluten-free diet despite the potential for long-term damage from continued gluten exposure.
  • The gluten-free diet can have negative side effects for some patients, including bloating and weight gain.
  • 70% of patients are accidentally exposed to gluten while on the gluten-free diet.

A significant part of the day was spent understanding from the FDA the burdens of proof that drug makers had to meet at various stages along the therapeutic pipeline to have celiac therapies approved. Other points discussed included: the low levels of diagnosis, who are potential targets for therapy and clinical trials within the celiac community, what precisely the focus should be of the initial round of therapies (i.e., improving quality of life, symptom relief, villi healing, and disease vaccine), and the psychological impacts of celiac disease on patients and caregivers.

There are currently five therapies in the pipeline that hope to make living with celiac disease less of a burden. The mere fact that GREAT3 was convened by the FDA and so well attended means there is progress being made. The presence of pharmaceutical companies with drugs in the pipeline is tremendous progress. The Foundation is proud, through your support, to have been a co-funder of this breakthrough event. But we still aren’t there yet. With your ongoing support and participation, the Celiac Disease Foundation will continue to make your voices heard in the legislative, medical, and research communities.