'An unmet medical need': Physician weighs in on 1st eosinophilic esophagitis treatment

The GI industry hit a milestone in May with the first FDA approved treatment for eosinophilic esophagitis.

The treatment, Dupixent, was approved to treat eosinophilic esophagitis in adults and pediatric patients 12 and older weighing at least 88 pounds.

Marc Rothenberg, MD, PhD, director of the Cincinnati Center for Eosinophilic Diseases at Cincinnati Children's, spoke with Becker's on June 8 to discuss the approval of the first eosinophilic esophagitis treatment, its significance and the challenges that came with researching the disease.

Editor's note: Responses have been edited lightly for length and clarity.

Question: What makes the approval of the 1st eosinophilic esophagitis treatment so important?

Dr. Marc Rothenberg: [Eosinophilic esophagitis] is an unmet medical need. It's increasing in a growing number of people. This disease involves a breakdown of the immune tolerance to oral antigens or foods and that results in people having very limited diets, which turns out to be a substantial problem in terms of quality of life. Just imagine Thanksgiving without eating turkey with your family at the table or having birthday celebrations or going to a friend's birthday party, especially for kids, without being able to have cake. I know this sounds small, but it turns out that [eosinophilic esophagitis] is a very low quality of life chronic disease of childhood based on our research as well as others.

Q: What was the biggest challenge you faced in your research of eosinophilic esophagitis?

MR: Initially, we had a disease that wasn't necessarily considered a disease when we first started describing this phenomenon. It took a while to convince others of the importance of this disease and change the paradigms in terms of understanding esophagitis. … This was a new form, which we called allergic esophagitis. Elucidating the mechanisms in preclinical systems with a limited amount of funding and support, then seeing fundamental observations we made when in preclinical systems. Particularly in mouse models, would it be applicable to humans? Translation research, approving that and then still working with a rare disease that wasn't of interest to the pharmaceutical industry. But using our know-how, capabilities, dedication and resources personally to perform a proof of principle study, which we conducted at Cincinnati Children's.

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