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. Author manuscript; available in PMC: 2019 Apr 17.
Published in final edited form as: Nat Rev Neurol. 2018 Oct;14(10):590–605. doi: 10.1038/s41582-018-0051-6

Table 4 |.

Candidate symptomatic drugs for treatment of SCAs

SCA type Candidate drug Scientific premise Preclinical study Comments
SCA6 and other SCAs 4-Aminopyridine Restoration of pacemaker activities of Purkinje cells by blocking the voltage- dependent family of potassium channels87 Alleviated motor coordination deficits and restored Purkinje cell firing precision in SCA6 mice homozygous for 84 polyQ repeats87 Approved by the FDA for symptomatic improvement of walking in multiple sclerosis. A randomized, double- blind, placebo- controlled study showed a decreased number of attacks in patients with episodic ataxia 2 (reF.213). A phase I study214 and a small open- label study215 have been done in patients with SCAs
SCA2 and other SCAs Chlorzoxazone KCNN1 channel activator. Normalization of the Purkinje cell spontaneous firing rate in SCA2 transgenic mice with 58 polyQ repeats100 and in Cacna1a- mutant mice93 Not yet done Chlorzoxazone is approved by the FDA and has been used extensively as a muscle relaxant
SCA44 Nitazoxanide Negative allosteric modulator of metabotropic glutamate receptor 1 and 5 with potent inhibition of mutant forms of these receptors in transfected cells214 Not done in SCA44 models, but similar modulators alleviated ataxia in an SCA1 mouse model216,217 Nitazoxanide is approved by the FDA and is used for various helminthic, protozoal and viral infections218

KCNN1, small-conductance calcium-activated potassium channel protein 1; polyQ, polyglutamine; SCA, spinocerebellar ataxia.

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