title: Update on the Pharmacotherapy of Cerebellar Ataxia and Nystagmus.
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).
authors.affiliation: Department of Neurology, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany. katharina.feil@med.uni-muenchen.de.|German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany. katharina.feil@med.uni-muenchen.de.
authors.forename: Katharina
authors.initials: K
authors.lastname: Feil
authors.affiliation: German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.|Graduate School of Systemic Neurosciences, Ludwig-Maximilians University, Munich, Germany.
authors.forename: Tatiana
authors.initials: T
authors.lastname: Bremova
authors.affiliation: German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.
authors.forename: Carolin
authors.initials: C
authors.lastname: Muth
authors.affiliation: Department of Neurology, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.|German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.
authors.forename: Roman
authors.initials: R
authors.lastname: Schniepp
authors.affiliation: Department of Neurology, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.|German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.
authors.forename: Julian
authors.initials: J
authors.lastname: Teufel
authors.affiliation: Department of Neurology, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.|German Center for Vertigo and Balance Disorders, University Hospital, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Strupp
mesh_terms: D015761:4-Aminopyridine / Q000627:therapeutic use; D002524:Cerebellar Ataxia / Q000150:complications / Q000188:drug therapy*; D002753:Chlorzoxazone / Q000627:therapeutic use; D004358:Drug Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans; D007930:Leucine / Q000031:analogs & derivatives / Q000627:therapeutic use; D009125:Muscle Relaxants, Central / Q000627:therapeutic use; D009759:Nystagmus, Pathologic / Q000188:drug therapy* / Q000209:etiology; D026902:Potassium Channel Blockers / Q000627:therapeutic use
chemical_list: D009125:Muscle Relaxants, Central; D026902:Potassium Channel Blockers; D015761:4-Aminopyridine; D007930:Leucine; D002753:Chlorzoxazone; C088117:acetylleucine
keywords: 4-Aminopyridine; Acetyl-DL-leucine; Chlorzoxazone; Downbeat nystagmus; Episodic ataxia type 2; Niemann-Pick type C