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Nat Rev Neurol. 2014 Sep;10(9):493-506. doi: 10.1038/nrneurol.2014.141. Epub 2014 Aug 12.

Treatment of neuromyelitis optica: state-of-the-art and emerging therapies.

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Academic Neurosurgery Unit, St George's, University of London, Room 0.136 Jenner Wing, Cranmer Terrace, Tooting, London SW17 0RE, UK.
Departments of Neurology and Ophthalmology, University of Colorado School of Medicine, Research Complex 2, Mail stop B-182, 12700 East 19th Avenue, Aurora, CO 80045, USA.
Department of Medicine, University of California, San Francisco, Health Science East Tower Room 1246, 513 Parnassus Avenue, San Francisco, CA 94143, USA.


Neuromyelitis optica (NMO) is an autoimmune disease of the CNS that is characterized by inflammatory demyelinating lesions in the spinal cord and optic nerve, potentially leading to paralysis and blindness. NMO can usually be distinguished from multiple sclerosis (MS) on the basis of seropositivity for IgG antibodies against the astrocytic water channel aquaporin-4 (AQP4). Differentiation from MS is crucial, because some MS treatments can exacerbate NMO. NMO pathogenesis involves AQP4-IgG antibody binding to astrocytic AQP4, which causes complement-dependent cytotoxicity and secondary inflammation with granulocyte and macrophage infiltration, blood-brain barrier disruption and oligodendrocyte injury. Current NMO treatments include general immunosuppressive agents, B-cell depletion, and plasma exchange. Therapeutic strategies targeting complement proteins, the IL-6 receptor, neutrophils, eosinophils and CD19--all initially developed for other indications--are under clinical evaluation for repurposing for NMO. Therapies in the preclinical phase include AQP4-blocking antibodies and AQP4-IgG enzymatic inactivation. Additional, albeit currently theoretical, treatment options include reduction of AQP4 expression, disruption of AQP4 orthogonal arrays, enhancement of complement inhibitor expression, restoration of the blood-brain barrier, and induction of immune tolerance. Despite the many therapeutic options in NMO, no controlled clinical trials in patients with this condition have been conducted to date.

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