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Update on the Pharmacotherapy of Cerebellar Ataxia and Nystagmus

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Abstract

Pharmacological treatment of cerebellar ataxias and cerebellar nystagmus still remains difficult. The efficacy of most of the agents recommended in the past for symptomatic or even causative therapy could not be proven in larger state-of-the art clinical trials. Exceptions are (a) 4-aminopyridine (4-AP) for episodic ataxia type 2 (EA2): one observational and one randomized controlled trial showed a significant effect on the number of attacks of ataxia and quality of life; (b) aminopyridines in cerebellar downbeat nystagmus (DBN): two randomized controlled trials and several observational studies demonstrate a significant improvement of the intensity of DBN, visual acuity, and postural imbalance. In both diseases the sustained-release form is evidently also efficient; (c) 4-AP in cerebellar gait ataxia: evidence comes from two observational studies. (d) chlorzoxazone in DBN which, however, was so far demonstrated in only one observational study; (e) the modified amino acid acetyl-DL-leucine: evidently effective in cerebellar ataxias, shown in three observational studies, one on patients with Niemann-Pick type C; its mode of action has to be evaluated in animal models and on a cellular/electrophysiological level. There are ongoing randomized placebo-controlled trials on EA2 with 4-AP versus acetazolamide (EAT-2-TREAT), cerebellar gait ataxia with 4-AP (FACEG), and a multinational trial on cerebellar ataxia with acetyl-DL-leucine (ALCAT).

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Acknowledgments

We thank Katie Ogston for copy-editing the manuscript.

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Correspondence to Katharina Feil.

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Some of the studies described are supported by a grant from the German Federal Ministry of Education and Research (BMBF) to the German Center for Vertigo and Balance Disorders (grant code 01 EO 0901).

Conflict of Interest

K. Feil, C. Muth, R. Schniepp, and J. Teufel report no disclosures. T. Bremova received speaker’s honoraria from Actelion. M. Strupp is Joint Chief Editor of the Journal of Neurology, editor in chief of Frontiers of Neuro-otology and section editor of F1000. He has received speaker’s honoraria from Abbott, UCB, GSK, TEVA, MSD, Heel, Biogen Idec, Pierre-Fabre, Eisai, and Hennig Pharma. He works as a consultant for Abbott.

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Feil, K., Bremova, T., Muth, C. et al. Update on the Pharmacotherapy of Cerebellar Ataxia and Nystagmus. Cerebellum 15, 38–42 (2016). https://doi.org/10.1007/s12311-015-0733-1

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