Top ▲

Neuromyelitis optica

Disease ID:1266
Name:Neuromyelitis optica
Associated with:1 target
1 immuno-relevant ligand
Devic's disease/syndrome | Neuromyelitis optica spectrum disorder | NMOSD
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


C3a receptor
Role:  Astrocyte-microglia interaction drives evolving neuromyelitis optica lesion. This involves C3a receptor signaling.
References:  1


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


Show »

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]