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Cerebellar ataxia

Disease ID:199
Name:Cerebellar ataxia
Associated with:1 target
Database Links
Disease Ontology: DOID:0050753


Role:  KCa2.2 suppression in deep cerebellar neurons in transgenic mice induces severe ataxia.
Drugs:  Riluzole showed positive effects in patients with hereditary cerebellar ataxia (SCA) in a randomised, double-blind, placebo-controlled trial; NS13001 alleviates behavioural and neuropathological phenotypes of aging SCA2 transgenic mice; SKA-31 corrects abnormal Purkinje cell firing and reduces motor deficits in SCA3 transgenic mice; Perfusion of EBIO into the cerebellum or systemic administration of chlorzoxazone to mice with episodic ataxia (EA2) significantly improves motor performance in model.
Side effects:  Sedation at higher doses. KCa2.2 activation could potentially impair memory.
Therapeutic use:  KCa activators have been proposed for the treatment of cerebellar ataxia.
References:  1-6


No ligand related data available for Cerebellar ataxia


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1. Alviña K, Khodakhah K. (2010) KCa channels as therapeutic targets in episodic ataxia type-2. J Neurosci, 30 (21): 7249-57. [PMID:20505091]

2. Kasumu AW, Hougaard C, Rode F, Jacobsen TA, Sabatier JM, Eriksen BL, Strøbæk D, Liang X, Egorova P, Vorontsova D et al.. (2012) Selective positive modulator of calcium-activated potassium channels exerts beneficial effects in a mouse model of spinocerebellar ataxia type 2. Chem Biol, 19 (10): 1340-53. [PMID:23102227]

3. Lam J, Coleman N, Garing AL, Wulff H. (2013) The therapeutic potential of small-conductance KCa2 channels in neurodegenerative and psychiatric diseases. Expert Opin Ther Targets, 17 (10): 1203-20. [PMID:23883298]

4. Romano S, Coarelli G, Marcotulli C, Leonardi L, Piccolo F, Spadaro M, Frontali M, Ferraldeschi M, Vulpiani MC, Ponzelli F et al.. (2015) Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial. Lancet Neurol, 14 (10): 985-91. [PMID:26321318]

5. Shakkottai VG, Chou CH, Oddo S, Sailer CA, Knaus HG, Gutman GA, Barish ME, LaFerla FM, Chandy KG. (2004) Enhanced neuronal excitability in the absence of neurodegeneration induces cerebellar ataxia. J Clin Invest, 113 (4): 582-90. [PMID:14966567]

6. Shakkottai VG, do Carmo Costa M, Dell'Orco JM, Sankaranarayanan A, Wulff H, Paulson HL. (2011) Early changes in cerebellar physiology accompany motor dysfunction in the polyglutamine disease spinocerebellar ataxia type 3. J Neurosci, 31 (36): 13002-14. [PMID:21900579]