Neuromyelitis Optica Treatment with Targeted Molecular Therapy

Neuromyelitis optica (NMO) is a distinct autoimmune disease, typically characterized by aquaporin-4 (AQP4) antibodies, which are associated with complement-mediated attack against astrocytes. The disease may result in devastating visual loss and spinal cord injury. It is currently treated with generalized immunosuppression.

In the present study, investigators developed a modified antibody against AQP4, which they engineered to not fix complement and to compete with the pathogenic AQP4 antibodies (which do fix complement). They engineered the inactive antibody by introducing point mutations designed to make the antibody deficient in initiating complement-dependent cytotoxicity and antibody-dependent cell-mediated cytotoxicity. (The institutio…