Focused Screening of Novel Therapies for HLH Using an Animal Model
Michael Jordan MD
Cincinnati Children’s Hospital Medical Center – Cincinnati, Ohio USA
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In recent years significant progress has been made in the treatment of hemophagocytic lymphohistiocytosis (HLH). However, outcomes for children with HLH are often still quite poor. Many children fail to respond completely or even adequately to the current standard treatments. We developed the first animal model of HLH and have learned much about how HLH develops and how current therapies work. We are now proposing to use this model as a screening tool, to identify new drugs which act in new or complimentary fashions compared to current approaches. This screening process is directed by 2 factors: first, we have identified particular targets within the immune response that are particularly promising for stopping HLH, and second, new drugs have become available (or will be soon) which can target these critical connections. This screen is a crucial first step. If we find particularly promising drug candidates in these preclinical studies, then we would propose to conduct a clinical trial to test these in patients. Through this effort we intend to improve the long term survival of children with HLH and develop significant new insights into HLH and human immune function.