A new treatment for a rare blood disorder

2019

Challenge

PDGFRA-negative hypereosinophilic syndrome is a chronic and debilitating disorder with limited and ineffective treatment options that tend to have significant side effects. It’s one of a group of rare disorders characterized by an excess of eosinophils, a type of white blood cell, in the blood and bodily tissues. Patients with the illness face substantial and long-term clinical manifestations that can include intractable itch, digestive problems, heart complications, and blood clots.

Advance

IRP researchers led by Amy Klion, M.D., completed a randomized, double-blind, placebo-controlled, Phase 2 clinical trial assessing the ability of a therapeutic called benralizumab to destroy eosinophils. Benralizumab, which targets a receptor found on the surface of human eosinophils, was highly effective in reducing eosinophil levels in blood and tissue while presenting few or no toxic effects in patients with a severe hypereosinophilic syndrome that is resistant to currently available treatments.

Impact

The discovery of a potentially effective therapy for the illness that has limited side effects will allow patients to taper down other treatments that have substantial long-term toxic effects.

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