STOPPING AN INFLAMMATION TRAIN
HLH is a rare but deadly childhood disease that overstimulates the immune system and causes hyperinflammation, which leads to widespread organ and tissue damage. Clinicians find about 1.2 cases per 1 million individuals per year. For many years, the mortality rate has been about 40%.
Jordan, has been working for years to find a treatment specific to HLH that can block the runaway inflammation. A key step in that effort occurred in 2004, when Jordan’s mouse study, published in the journal Blood, revealed that the HLH disease process relies on high levels of the protein interferon gamma (IFNg).
Now, emapalumab has shown its ability to block IFNg, which in turn offers improved odds for more children to live with HLH as more of a chronic disease instead of an early cause of death.
ENCOURAGING RESULTS
The phase 2–3 clinical trial was performed at 14 sites in Germany, Italy, Spain, the United Kingdom, and the United States. Jordan led the project in conjunction with Franco Locatelli, MD, at the University of Rome.
Overall, 34 patients received emapalumab, including 27 who had received other unsuccessful treatments and seven patients who had not been treated in any other way.
The eight-week treatment began with an emapalumab dose of 1 mg per kilogram of body weight every three days, which was increased over time up to as high as 10 mg per kilogram. Twenty-six patients completed the study.