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NMOSD treatments vary in efficacy, but there are not many highly effective options. Novel therapies provide greater diversity of mechanisms and targets for a more well-rounded approach, but require additional research to determine their efficacy.

Neuromyelitis optica spectrum disorder (NMOSD) is characterized by demyelination in the central nervous system and the presence of the aquaporin-4 immunoglobulin G (AQP4-IgG) antibody. It involves repeated acute attacks that manifest as optc neuritis, myelitis, and certain brain and brainstem syndromes that can result in disability, devastating sequelae, or death.

Phase 3 trials of newly developed therapies have shown some effectiveness and improved tolerance when compared to traditional therapies and previous drugs used to deal with this condition, but there remains a lack of large sample, double-blind, randomized clinical studies to confirm the efficacy, tolerability, and safety of these drugs. This article, published in CNS Neuroscience & Therapeutics, analyzed the current situation with respect to these novel therapeutic options, including the effect on patients without AQP4-IgG and refractory patients. 

Current therapies for NMOSD vary between drugs with a variety of mechanisms and targets. These include first-line therapies such as glucocorticoids, azathioprine (AZA), and rituximab (RTX), and second-line therapies with other immunosuppressive treatments such as methotrexate, mycophenolate mofetil (MMF), and mitoxantrone. Some of the novel biological therapies for NMOSD include inebilizumab and ocrelizumab targeting B cells,  tocilizumab and satralizumab to target anti-interleukin-6 receptor antibodies, and eculizumab targeting complement inhibitor, although none of these treatments qualify as curative, approved treatments for NMOSD. The authors note that due to various difficulties in treating NMOSD, alleviating acute symptoms and preventing relapses are the key therapeutic objectives for NMOSD.

The effectiveness of these novel therapies and their mechanisms and targets includes varied approaches such as stem cell therapy, targeting Th17 cells, and blocking IL23/IL17/Th17 pathway, all of which are promising but require additional research to determine their safety and efficacy. The authors conclude that the diversity of curative targets and mechanisms provides promise that patients with NMOSD in a variety of conditions could have access to effective therapeutic interventions in the near future.

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Shi, M., Chu, F., Jin, T., & Zhu, J. (2022). Progress in treatment of neuromyelitis optica spectrum disorders (NMOSD): Novel insights into therapeutic possibilities in NMOSD. CNS Neuroscience & Therapeutics, 28(7), 981-991. https://doi.org/10.1111/cns.13836