Abstract
Options for drug treatment are very limited in patients with degenerative forms of ataxias. Drug treatments are available in a few metabolic disorders and vitamin deficiencies that can cause ataxia. These diseases have to be excluded in all cases of ataxias of unknown origin. Despite the huge increase in knowledge in the underlying genetics and pathophysiology, however, causal treatments are currently not available for the large majority of hereditary and non-hereditary degenerative ataxias. Most preclinical and clinical studies have been done in Friedreich’s ataxia, and findings will be briefly summarized. Likewise, no symptomatic drug treatment is available which ameliorates the clinical signs and symptoms of ataxia. The only exceptions are aminopyridines for treatment of downbeat nystagmus, and acetazolamide and aminopyridines for treatment of episodic ataxias. A number of other drugs have been tried for symptomatic treatment of ataxias, but without provable effects.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Ilg W, Bastian AJ, Boesch S, Burciu RG, Celnik P, Claaßen J, Feil K, Kalla R, Miyai I, Nachbauer W, Schöls L, Strupp M, Synofzik M, Teufel J, Timmann D (2014) Consensus paper: management of degenerative cerebellar disorders. Cerebellum 13:248–268
Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR (2011) Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J 161:639–645
Libri V, Yandim C, Athanasopoulos S, Loyse N, Natisvili T, Law PP, Chan PK, Mohammad T, Mauri M, Tam KT, Leiper J, Piper S, Ramesh A, Parkinson MH, Huson L, Giunti P, Festenstein R (2014) Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich’s ataxia: an exploratory, open-label, dose-escalation study. Lancet 384:504–513
Mariotti C, Fancellu R, Caldarazzo S, Nanetti L, Di Bella D, Plumari M, Lauria G, Cappellini MD, Duca L, Solari A, Taroni F (2012) Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial. Mov Disord 27:446–449
Pandolfo M (2012) Friedreich ataxia. Handb Clin Neurol 103:275–294
Pandolfo M, Arpa J, Delatycki MB, Le Quan Sang KH, Mariotti C, Munnich A, Sanz-Gallego I, Tai G, Tarnopolsky MA, Taroni F, Spino M, Tricta F (2014) Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. Ann Neurol 76:509–521
Ramirez-Zamora A, Zeigler W, Desai N, Biller J (2015) Treatable causes of cerebellar ataxia. Mov Disord 30(5):614–623
Ristori G, Romano S, Visconti A, Cannoni S, Spadaro M, Frontali M, Pontieri FE, Vanacore N, Salvetti M (2010) Riluzole in cerebellar ataxia: a randomized, double-blind, placebo-controlled pilot trial. Neurology 74:839–845
Saiz A, Blanco Y, Sabater L, González F, Bataller L, Casamitjana R, Ramió-Torrentà L, Graus F (2008) Spectrum of neurological syndromes associated with glutamic acid decarboxylase antibodies: diagnostic clues for this association. Brain 131:2553–2563
Strupp M, Schüler O, Krafczyk S, Jahn K, Schautzer F, Büttner U, Brandt T (2003) Treatment of downbeat nystagmus with 3,4-diaminopyridine: a placebo-controlled study. Neurology 61:165–170
Strupp M, Kalla R, Claassen J, Adrion C, Mansmann U, Klopstock T, Freilinger T, Neugebauer H, Spiegel R, Dichgans M, Lehmann-Horn F, Jurkat-Rott K, Brandt T, Jen JC, Jahn K (2011) A randomized trial of 4-aminopyridine in EA2 and related familial episodic ataxias. Neurology 77:269–275
Strupp M, Teufel J, Habs M, Feuerecker R, Muth C, van de Warrenburg BP, Klopstock T, Feil K (2013) Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series. J Neurol 260:2556–2561
Velázquez-Pérez L, Rodríguez-Chanfrau J, García-Rodríguez JC, Sánchez-Cruz G, Aguilera-Rodríguez R, Rodríguez-Labrada R, Rodríguez-Díaz JC, Canales-Ochoa N, Gotay DA, Almaguer Mederos LE, Laffita Mesa JM, Porto-Verdecia M, Triana CG, Pupo NR, Batista IH, López-Hernandez OD, Polanco ID, Novas AJ (2011) Oral zinc sulphate supplementation for six months in SCA2 patients: a randomized, double-blind, placebo-controlled trial. Neurochem Res 36:1793–1800
Zesiewicz TA, Greenstein PE, Sullivan KL, Wecker L, Miller A, Jahan I, Chen R, Perlman SL (2012) A randomized trial of varenicline (Chantix) for the treatment of spinocerebellar ataxia type 3. Neurology 78:545–550
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2016 Springer International Publishing Switzerland
About this chapter
Cite this chapter
Timmann, D., Ilg, W. (2016). Drugs in Selected Ataxias. In: Gruol, D., Koibuchi, N., Manto, M., Molinari, M., Schmahmann, J., Shen, Y. (eds) Essentials of Cerebellum and Cerebellar Disorders. Springer, Cham. https://doi.org/10.1007/978-3-319-24551-5_82
Download citation
DOI: https://doi.org/10.1007/978-3-319-24551-5_82
Published:
Publisher Name: Springer, Cham
Print ISBN: 978-3-319-24549-2
Online ISBN: 978-3-319-24551-5
eBook Packages: Biomedical and Life SciencesBiomedical and Life Sciences (R0)