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The untold story of a new asthma drug

Cincinnati Children’s key part of multi-year research effort

Saturday, November 07, 2015

 CINCINNATI – When the FDA approved the drug mepolizumab this week to help treat severe asthma, it was a blip on the radar screen of health industry news. The medication targets a subset of asthma patients (ages 12 and up) whose current drug regimens are insufficient to control their condition.

Absent from official press announcements about mepolizumab’s approval on Nov. 4 are the many years of research and testing by countless physicians and scientists at institutions around the world. Cincinnati Children’s and its Division of Allergy and Immunology were key parts to this effort.

“After 30 years of research done in part by our team, this is the first new asthma drug approved in over a decade,” says Marc Rothenberg, MD, PhD, director of the Division of Allergy and Immunology and the Cincinnati Center for Eosinophilic Disorders. “A tremendous amount of work has been done behind the scenes to get here, not only by Cincinnati Children’s but also by many others.”

Rothenberg and his research team are considered pioneers in the field, and Cincinnati Children’s has become a global leader in researching and treating eosinophilic conditions.
The institution’s researchers characterized a critical genetic/molecular pathway (involving the protein interleukin 5) that helps fuel severe asthma, which is caused by allergy-associated, inflammatory cells called eosinophils. Mepolizumab inhibits interleukin 5 and blocks the production of eosinophils, which drives certain types of severe asthma.

In addition to basic laboratory studies that established the molecular target for this therapy, Cincinnati Children’s also helped conduct clinical trials testing the safety and efficacy of mepolizumab in patients. Patient studies involved those with asthma and other eosinophilic disorders such as eosinophilic esophagitis.

Rothenberg says mepolizumab is the first among a new class of drugs based on targeting eosinophils. Mepolizumab is marketed under the brand name Nucala and made by GlaxoSmithKline.

Asthma is a chronic disease that causes inflammation in the airways of the lungs. During an asthma attack, airways become narrow, making it hard to breathe. Severe asthma attacks can lead to asthma-related hospitalizations because these attacks can be serious and even life threatening.

According to the Centers for Disease Control and Prevention, as of 2013, more than 22 million people in the U.S. have asthma, and there are more than 400,000 asthma-related hospitalizations each year.

About Cincinnati Children’s
Cincinnati Children’s Hospital Medical Center ranks third in the nation among all Honor Roll hospitals in U.S.News and World Report’s 2015 Best Children’s Hospitals. It is also ranked in the top 10 for all 10 pediatric specialties, including a  #1 ranking in pulmonology and #2 in cancer and in nephrology. Cincinnati Children’s, a non-profit organization, is one of the top three recipients of pediatric research grants from the National Institutes of Health, and a research and teaching affiliate of the University of Cincinnati’s College of Medicine.  The medical center is internationally recognized for improving child health and transforming delivery of care through fully integrated, globally recognized research, education and innovation. Additional information can be found at www.cincinnatichildrens.org. Connect on the Cincinnati Children’s blog, via Facebook and on Twitter