open access

Vol 53, No 5 (2019)
Invited review article
Published online: 2019-10-30
Submitted: 2019-10-11
Accepted: 2019-10-20
Get Citation

Novel emerging treatments for NMOSD

Krzysztof Selmaj, Igor Selmaj
DOI: 10.5603/PJNNS.a2019.0049
·
Pubmed: 31664710
·
Neurol Neurochir Pol 2019;53(5):317-326.

open access

Vol 53, No 5 (2019)
Invited review article
Published online: 2019-10-30
Submitted: 2019-10-11
Accepted: 2019-10-20

Abstract

Neuromyelitis optica spectrum disorders (NMOSD) are inflammatory demyelinating diseases of the central nervous system (CNS) that cause optic neuritis, transverse myelitis, and some other CNS syndromes.
Recently, diagnosis and understanding of these diseases has been markedly enhanced by the discovery that serum autoantibodies that target aquaporin-4 (AQP4) are strongly associated with the disease. This spectrum includes also a potential subset of patients with a phenotype of NMOSD who have anti-myelin oligodendrocyte glycoprotein (MOG) antibody. Although steroids and immunosuppressive drugs have been widely used for NMOSD treatment, until recently there was no approved therapy for these diseases. With improved understanding of the pathophysiology of NMOSD, numerous new therapeutic strategies have recently been evaluated. The results of these studies, involving monoclonal antibodies (mAbs) inhibiting terminal complement protein cleavage interfering with interleukin-6 receptor (IL-6 R) signaling and depleting CD19-positive B cells, have been published in recent months. All of these new therapeutics have shown a high degree of efficacy in diminishing NMOSD activity and inhibiting disability progression. At the same time, all these mAbs have demonstrated favorable safety and tolerability profiles, with a limited rate of adverse events.
The first of these new drugs, eculizumab, have been approved in USA and Europe for NMOSD treatment within the last couple of months and it is expected that the other novel, effective and safe treatments for NMOSD will be approved in the near future.

Abstract

Neuromyelitis optica spectrum disorders (NMOSD) are inflammatory demyelinating diseases of the central nervous system (CNS) that cause optic neuritis, transverse myelitis, and some other CNS syndromes.
Recently, diagnosis and understanding of these diseases has been markedly enhanced by the discovery that serum autoantibodies that target aquaporin-4 (AQP4) are strongly associated with the disease. This spectrum includes also a potential subset of patients with a phenotype of NMOSD who have anti-myelin oligodendrocyte glycoprotein (MOG) antibody. Although steroids and immunosuppressive drugs have been widely used for NMOSD treatment, until recently there was no approved therapy for these diseases. With improved understanding of the pathophysiology of NMOSD, numerous new therapeutic strategies have recently been evaluated. The results of these studies, involving monoclonal antibodies (mAbs) inhibiting terminal complement protein cleavage interfering with interleukin-6 receptor (IL-6 R) signaling and depleting CD19-positive B cells, have been published in recent months. All of these new therapeutics have shown a high degree of efficacy in diminishing NMOSD activity and inhibiting disability progression. At the same time, all these mAbs have demonstrated favorable safety and tolerability profiles, with a limited rate of adverse events.
The first of these new drugs, eculizumab, have been approved in USA and Europe for NMOSD treatment within the last couple of months and it is expected that the other novel, effective and safe treatments for NMOSD will be approved in the near future.

Get Citation

Keywords

neuromyelitis optica spectrum disorders, monoclonal antibodies, demyelinating diseases

About this article
Title

Novel emerging treatments for NMOSD

Journal

Neurologia i Neurochirurgia Polska

Issue

Vol 53, No 5 (2019)

Pages

317-326

Published online

2019-10-30

DOI

10.5603/PJNNS.a2019.0049

Pubmed

31664710

Bibliographic record

Neurol Neurochir Pol 2019;53(5):317-326.

Keywords

neuromyelitis optica spectrum disorders
monoclonal antibodies
demyelinating diseases

Authors

Krzysztof Selmaj
Igor Selmaj

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