ANVIL BIOSCIENCES LLC — Department of Health and Human Services SBIR Phase I: DESCRIPTION (provided by applicant): Neuromyelitis Optica (NMO) is a rare autoimmune infla

Description

DESCRIPTION (provided by applicant): Neuromyelitis Optica (NMO) is a rare autoimmune inflammatory disease that affects the spinal cord and optical nerves leading to limb paralysis and loss of vision. Despite advances in the understanding of underlying disease mechanisms and patient diagnostic criteria there is a lack of treatment options. A specific cellular component of NMO disease was found to be neutrophils as they play a pathogenic role in causing the observed inflammation. We propose targeting a recently discovered pathogenic mediator secreted by neutrophils in NMO disease by using inhibitors of neutrophil elastase as a therapeutic approach. Various neutrophil elastase inhibitors have been used for other indications and our strategy seeks to re-purposethese drugs for NMO to find an optimal therapeutic. Our first aim will test the efficacy of two neutrophil elastase inhibitors using a Th1 mouse EAE transfer model that recapitulates the pathogenic features seen in NMO patients. We will measure thera