Repurposing a Blood Cancer Drug to Treat an Immune Disorder (HLH) in Children

Principal Investigator: Dr. Kim Nichols

Disease: Hemophagocytic Lymphohistiocytosis

Research Description: Hemophagocytic lymphohistiocytosis (HLH) is a rare, poorly understood disorder of the immune system characterized by recurrent episodes of hyperinflammation. Currently, the treatment for HLH is aimed at quieting the overactive immune system with drugs such as steroids and chemotherapy. While this approach is effective in some patients, these drugs can cause significant side effects with less than optimal results. In addition, with the high morbidity and mortality associated with HLH, there is a pressing need to develop more effective therapies. The research team has found that the FDA-approved blood cancer drug ruxolitinib decreases the signs of disease and enhances survival in mice, and they now want to determine whether ruxolitinib is safe and effective in human patients, specifically in children. The team will start with a 10-patient clinical trial, and if successful, the data provided may form the foundation for a larger pivotal Phase II clinical trial.

Funding Partners: Multiple Anonymous Funders

CWR funding role: Primary Funder