Cincinnati Children's Researchers Publish Positive Findings From Clinical Study Of Investigational Treatment For Debilitating Allergic Disorder

Cincinnati Children's Hospital Medical Center, a recognized leader in pediatric research dedicated to changing the outcomes for children, today announced the publication of positive results from a phase 1/2 clinical trial evaluating mepolizumab, a humanized antibody to interleukin 5 (IL-5), for the treatment of eosinophilic esophagitis (EE). EE is an allergic inflammatory reaction characterized by the accumulation of large numbers of eosinophils (a type of white blood cell associated with allergic reactions) in the esophagus that leads to vomiting and difficulty in swallowing.

"Previous results suggested a key role for interleukin 5 in the accumulation of eosinophils in the esophagus and provided a strong rationale for evaluating an anti IL5 antibody as a potential therapy for EE," said Marc Rothenberg M.D., Ph.D., director of the Division of Allergy and Immunology at Cincinnati Children's Hospital Medical College. "With support of the CCHMC Translational Research Office, this study provided a strong proof of concept for the use of mepolizumab. This data has fueled the pharmaceutical industry to expand this clinical experience with the goal of providing patients with a meaningful and effective treatment option." The paper, published in the December issue of the Journal of Allergy and Immunology, describes positive results from an open-label phase I/2 study evaluating the safety and efficacy of mepolizumab in four adult patients with EE who had long term difficulty in swallowing and esophageal narrowing caused by chronic inflammation. Patients received 3 monthly infusions of mepolizumab without change in their current therapy and were monitored for 28 weeks.

At the conclusion of the study, analysis showed statistically significant reductions in several key indicators of disease including marked decreases in eosinophils in the peripheral blood and in the esophagus. In addition endoscopic examination of the esophagus showed improvements in three out of four patients. The treatment was generally well-tolerated, and all patients reported a better clinical outcome and improved quality of life.

"Cincinnati Children's has long been at the forefront of research into eosinophilic disorders and its commitment was recently underscored by the establishment of the Cincinnati Center for Eosinophilic Disorders," said Ellyn Kodroff, President, CURED: Campaign Urging Research for Eosinophilic Disorders. "Clinical results like this provide important hope for relief from the horrific effects of this condition for patients and their families."

EE is part of a series of chronic and debilitating gastrointestinal disorders associated with allergies that are characterized by elevated levels of eosinophils that attach to the intestinal tract leading to abdominal pain, nausea, vomiting and difficulty swallowing. Patients suffering from these disorders frequently must adhere to a strict diet or acquire nutrients via a feeding tube directly inserted into the stomach; and many endure nausea, abdominal pain and vomiting after every meal. Newly recognized as a disease, data shows a dramatic increase in the diagnosis of EE. It is estimated that at least one in 1,500 people suffer from some form of the disorder.

Allergic disorders are a major public health problem, affecting 50 million people in the United States. Nine million children under 18 have been diagnosed with asthma, and asthma rates in children under the age of five increased more than 160 percent from 1980-1994.

About Cincinnati Children's Hospital Medical Center

Cincinnati Children's Hospital Medical Center, a recognized leader in pediatric research, is dedicated to changing the outcome for children throughout the world. Cincinnati Children's ranks second among all pediatric institutions in the United States in grants from the National Institutes of Health. It has an established tradition of research excellence, with discoveries including the Sabin oral polio vaccine, the surfactant preparation that saves the lives of thousands of premature infants each year, and a lifesaving rotavirus vaccine for hundreds of thousands of infants around the world. Current strategic directions include the translation of basic laboratory research into novel therapeutics for the treatment of disease, and furthering the development of personalized and predictive medicine for children.

Cincinnati Children's Hospital Medical Center
http://www.cincinnatichildrens.org/