Many of the major milestones in understanding eosinophilic esophagitis (EoE), a food allergy that affects the esophagus, began in research laboratories at The Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania.
Investigators discovered the first gene related to the disease, and they identified an immunological pathway that makes EoE distinctive from other food allergies. Now, they are pursuing a novel approach to cure the chronic disorder, as no medications are approved currently to treat EoE.
“If it works, it can potentially revolutionize clinical care,” said Jonathan Spergel, MD, who is leading the proof-of-concept study called SMILEE.
Dr. Spergel is co-director of CHOP’s Center for Pediatric Eosinophilic Disorders, which treats 1,500 patients with EoE from all over the world. About 70 percent of the patients have milk-induced EoE. When Dr. Spergel first began studying EoE, little was known about the disease, but diagnoses of this disorder are increasing rapidly as awareness grows.
“I have been fascinated to understand exactly who gets EoE, whose at risk, and what’s the best way of treating it,” said Dr. Spergel, who is also chief of CHOP’s allergy section.
During an allergic reaction to a food associated with EoE, high quantities of eosinophils, a type of white blood cell, congregate in the esophagus. This muscular tube that carries food from the throat to the stomach gets swollen. Children with EoE experience varying symptoms including belly pain, trouble swallowing, uncontrollable reflux, and failure to thrive.
Physicians currently approach EoE treatment two ways. First, is avoidance of the food that causes the allergic reaction. Second, is prescription of topical steroids to coat the esophagus. Dr. Spergel pointed out that these options do not address the underlying disease; they only control the symptoms.
“Can we do something better? That’s the question this study is asking,” Dr. Spergel said. “We’re trying to induce tolerance using a novel method — epicutaneous immunotherapy (EPITTM).”
EPIT is a desensitization method developed and patented by DBV Technologies (DBV) and uses Viaskin® technology. The application of a Viaskin per day involves maintaining an allergen on the skin of an allergic person for repeated and prolonged periods. DBV will provide a grant to The Children’s Hospital of Philadelphia to support the clinical trial. DBV and Dr. Spergel designed the double-blind, placebo-controlled, randomized trial to assess the effectiveness and safety of using an investigational new drug intended for EPIT, Viaskin® Milk, an allergen extract of milk administered via a skin patch.
“What we think happens is when the milk is absorbed through the skin, it interacts with antigen-presenting cells, which then induce T regulatory cells, and these T regulatory cells then cause the body not to be allergic anymore,” Dr. Spergel said.
Dr. Spergel anticipates that the SMILEE study will include participants ages 4 to 17. If it turns out that EPIT works, Dr. Spergel said it would be a major turning point for EoE treatment.
“We would be able to completely change the way we treat the disease,” Dr. Spergel said. “All these patients who have been unable to drink milk now would be able to drink milk and improve their quality of life.”
Dr. Spergel has other clinical studies under way of Viaksin® Peanut, another form of EPIT for patients with peanut allergies.
The SMILEE study also is supported by donations from grateful CHOP families.