Researchers highlighted the need to perform clinical trials for novel drugs to treat neuromyelitis optica spectrum disorders (NMOSD) in a timely manner and published their findings in CNS Neuroscience & Therapeutics.

The review conducted by Shi et al referenced 107 publications regarding updates on NMOSD treatment. Although visible progress has been made to better understand the physiopathological processes of the disease, many proposals for new therapeutic targets have arisen; in daily clinical practice, not much has changed for these patients.

Drugs currently approved for NMOSD include glucocorticoids, azathioprine, rituximab, methotrexate, mycophenolate mofetil, and mitoxantrone. These have been most useful to maintain remission and prevent acute attacks, and these agents have important limitations such as considerable side effects and ineffectiveness in some cases. New biological agents have been under scientific interest in the past years; nevertheless, most clinical trials compared their results with a placebo control group, making unclear the benefits of these drugs vs the traditional schemes.

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“These new biologicals are promising and have emerged in the form of targeting B-cell (inebilizumab and ocrelizumab, the anti-CD19 and CD20 antibodies, respectively), anti-IL-6 receptor antibodies (tocilizumab and satralizumab), complement inhibitors (eculizumab), etc., which have been started to investigate their effectiveness and advent events in NMOSD via clinical trials in the randomized large samples,” the authors said.

“The clinical trial results evidenced that these new biological therapies successfully improved clinic symptoms, reduced the risk of disease relapse, and had good tolerance in most NMOSD patients, especially in the patients with AQP4-IgG positivity.”

Recent researchers have proposed different approaches, such as targeting T-helper cell 17 (Th17) to block the interleukin 23(IL23)/interleukin 17 (IL17)/Th17 pathway, and stem cell therapy that aims to improve tolerance to autoantigens and promote seroconversion to negative AQP4-immunoglobulin G. These options have showcased promising results during experimental studies, but no clinical trials have proven their efficacy in preventing attacks and diminishing symptoms, let alone assessing recommended schemes and possible side effects.

Moreover, regardless of the treatment of choice, a shift towards personalized therapy has been generally preferred. The authors suggest basing this choice upon the patient’s response, tolerability, and underlying AQP4-immunoglobulin G status, as well as whether to implement a combination therapy or initiate with a single drug.

“The amazing novel therapeutic biologicals with diverse curative targets and mechanisms have started to be applied in order to treat NMOSD patients and finally emerge as effective and promising therapeutic interventions,” the authors concluded.


Shi M, Chu F, Jin T, Zhu J. Progress in treatment of neuromyelitis optica spectrum disorders (NMOSD): novel insights into therapeutic possibilities in NMOSD. CNS Neurosci Ther. Published online April 15, 2022. doi:10.1111/cns.13836