Marc Rothenberg, M.D., directs the division of allergy and immunology and the Cincinnati Center for Eosinophilic Disorders at Cincinnati Children’s Hospital Medical Center. He’s spent decades trying to understand the mechanisms of allergic reactions, and his work has led to FDA approval for a drug that can help.
It started about 32 years ago when I was a mere grad student and getting my PhD. In the lab, we discovered eosinophils, which are white blood cells, had a characteristic that causes them to grow and survive in ways that weren’t expected.
We identified this particular molecule that was very critical. It was called interleukin 5 or IL-5. We were the first to identify this in humans. It was a leap forward in understanding of the molecular control of allergic reactions.
In the 1990s, when I was recruited by Cincinnati Children’s, we continued to pursue research on eosinophil development. Then around 20 years ago, the drug companies developed a drug that would block IL-5. They did an experiment in 24 patients. That study showed the drug did not have the impact that they expected on asthmatic patients. That was a very big disruption in the theories we had developed.
But we pursued this because it was my belief this was going to be important. We got the drug from the drug company after a lot of arm twisting. We were able to convince them to allow us to continue testing the drug. In 2001, we formed the Cincinnati Center for Eosinophilic Disorders, the first center to focus on these diseases.
We did studies at Children’s and, lo and behold, we were able to show these drugs lowered, almost completely [eroded], the eosinophils. Now companies had new interest in pursuing the research. But the drug companies weren’t interested in the rare disease, hypereosinophilic syndrome (HES). They were more interested in going back to the asthma question.
Asthma affects more than 25 million Americans. HES has less than 10,000 people in the U.S. depending on how you define the disease. It was very disappointing for these patients who were continuing to suffer—and some of them were dying—from HES.
In 2015, the FDA approved a drug that belonged to the new classification of drugs that block eosinophils for asthma. This was the first new class of drugs for asthma in a dozen years. We continued to explore it with a particular focus on the rare disease of hypereosinophilic syndrome. Finally, in September, the FDA approved the drug for HES based on the research my colleagues and I had undertaken.
I’m glad to see this is going to be very helpful for patients. But I’m interested in curing disease. For me, it’s one more step in the process. It’s like conquering a mountain and you just want to go do another one. Scientists shouldn’t be satisfied with what they know. We collaborate with people around the world. The cure is going to happen any day. We can’t put a timeline on it. But we know what we’re doing is opening the door. When the cure comes, it may not be from the Rothenberg lab directly but we have certainly contributed to that knowledge.