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Neuromyelitis optica

GtoPdb Disease Summaries

This section gives an overview of the disease, and where available shows the following:

  • Synonyms: Shows known synonyms for the disease.
  • Description: Gives a basic description/definition of the disease.
  • Database Link: External links to the same disease at the Disease Ontology, OMIM or Orphanet sites.
  • Immunopharmacology comments: General comments about the target's role in immunopharmacology, provided by GtoImmuPdb curators.
  • Associated with: Counts are displayed for the total targets the disease is associated with in GtoPdb. The counts of targets and ligands of immunological relevance associated to the disease are also shown.

More information can be found in the help pages.

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Disease ID:1266
Name:Neuromyelitis optica
Associated with:1 target
1 immuno-relevant ligand
Synonyms
Devic's disease/syndrome | Neuromyelitis optica spectrum disorder | NMOSD
Description
Neuromyelitis optica (NMO) is an inflammatory demyelinating disease. It selectively causes inflammation in the optic nerves (optic neuritis) and spinal cord (myelitis). Some neuromyelitis optica sufferers are seropositive for IgG autoantibodies against the aquaporin 4 (AQP4) water channel that is expressed in the brain. Patients with NMO frequently have other systemic autoimmune disorders (e.g. systemic lupus erythematosus, Sjögren's syndrome or myasthenia gravis).
Database Links
Disease Ontology: DOID:8869
OMIM: 600308
Orphanet: ORPHA71211

Targets

GtoPdb Disease Summaries - Targets

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Where available, information is display on the role of the target in the disease; drugs which target the disease and their therapeutic use and side-effects.

If there is mutation data curated in GtoPdb this is indicated, with a link back to the appropriate section on the target detailed view page

Immuno ligand interactions - If available, a table of immuno-relevant ligands is shown. These ligands have been curated as having an association to the disease and possess interaction data with the target in GtoPdb. The approval status of the ligand is shown, along with curator comments and an indication of whether the target is considered the primary target of the ligand.

More information can be found in the help pages.

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Key to terms and symbols
C3a receptor
Role:  Astrocyte-microglia interaction drives evolving neuromyelitis optica lesion. This involves C3a receptor signaling.
References:  1

Ligands

GtoPdb Disease Summaries - Ligands

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  • Approved: If the ligand is an approved drug this is indicated, along with approval bodies.
  • Immuno: Immuno icon indicates the ligand is immuno-relevant

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  • Immuno Disease Comments: Curatorial comments specifically added as part of GtoImmuPdb. They give more information on the association between the ligand and disease in the context of immunopharmacology.
  • Clinical Use: General clinical comments relating to the ligand and may not necessarily be specific to the disease in question. With hyperlink to more details on the ligand summary pages.
  • Bioactivty Comments: Curatorial comments specifically about the compounds biological activity - with hyperlink to more details on the ligand summary pages.

More information can be found in the help pages.

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Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
eculizumab 2
Immuno Disease Comments: FDA approved drug for NMO patients with anti-AQP4 autoantibodies (granted in June 2019).
Clinical Use: Used to treat paroxysmal nocturnal hemoglobinuria (PNH). In the UK, NICE (National Institute for Health and Care Excellence) has approved eculizumab as a treatment for the rare, but fatal blood disorder, atypical haemolytic uraemic syndrome (aHUS). This decision was reached because eculizumab represents a significant innovation for a disease with a high unmet clinical need. Eculizumab treatment is expected to increase life expectancy of aHUS patients by decades. The FDA expanded approval in October 2017, to include treatment of patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.

SARS-C0V-2 and COVID-19: Eculizumab has been entered into clinical trials that aim to determine its ability to combat the dysregulated immune response that drives organ damage (lung and other organs) in patients with severe COVID-19. | View clinical data
Bioactivity Comments: Eculizumab binds to and blocks the cleavage of the complement protein C5, thereby preventing the formation of . The terminal half-life of eculizumab is 11.3 ± 3.4 days [3]. | View biological activity

References

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1. Chen T, Lennon VA, Liu YU, Bosco DB, Li Y, Yi MH, Zhu J, Wei S, Wu LJ. (2020) Astrocyte-microglia interaction drives evolving neuromyelitis optica lesion. J Clin Invest, 130 (8): 4025-4038. [PMID:32568214]

2. Pittock SJ, Berthele A, Fujihara K, Kim HJ, Levy M, Palace J, Nakashima I, Terzi M, Totolyan N, Viswanathan S et al.. (2019) Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med, 381 (7): 614-625. [PMID:31050279]

3. Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. (2007) Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol, 25 (11): 1256-64. [PMID:17989688]