Pharmacotherapy of vestibular and cerebellar disorders and downbeat nystagmus: translational and back-translational research

Ann N Y Acad Sci. 2015 Apr:1343:27-36. doi: 10.1111/nyas.12774.


There are currently eight groups of drugs for the pharmacotherapy of vertigo, nystagmus, and cerebellar disorders: antiemetics; anti-inflammatories, antimenieres, and antimigraineous medications; antidepressants, anticonvulsants, aminopyridines, and acetyl-DL-leucine ("the eight A's"). In acute unilateral vestibulopathy, corticosteroids improve the recovery of peripheral vestibular function, but there is not sufficient current evidence for a general recommendation. There is also insufficient evidence that 48 or 144 mg/day betahistine has an effect in Ménière's disease. Therefore, higher dosages are currently recommended; in animal studies, it was shown that betahistine increases cochlear blood flow. In vestibular paroxysmia, oxcarbazepine was effective (one yet not randomized controlled trial (RCT)). Aminopyridines are recommended for the treatment of downbeat nystagmus (two RCTs) and episodic ataxia type 2 (EA2, one RCT). There are so far no RCTs on vestibular migraine, so currently no treatment can be recommended. Acetyl-dl-leucine improves cerebellar ataxia (three observational studies); it also accelerates central compensation in an animal model of acute unilateral lesion, but RCTs were negative. There are ongoing RCTs on vestibular paroxysmia with carbamazepine (VESPA), acute unilateral vestibulopathy with betahistine (BETAVEST), vestibular migraine with metoprolol (PROVEMIG), benign paroxysmal positional vertigo with vitamin D (VitD@BPPV), EA2 with 4-aminopyridine versus acetazolamide (EAT-2-TREAT), and cerebellar ataxias with acetyl-DL-leucine (ALCAT).

Keywords: Ménière's disease; acute unilateral vestibulopathy; aminopyridines; central vestibular disorders; cerebellar ataxias; downbeat nystagmus; episodic ataxia type 2; vestibular migraine; vestibular paroxysmia.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aminopyridines / therapeutic use
  • Animals
  • Betahistine / therapeutic use
  • Humans
  • Nystagmus, Pathologic / drug therapy*
  • Postural Balance / drug effects
  • Translational Research, Biomedical
  • Vestibular Diseases / drug therapy*
  • Vestibule, Labyrinth / physiopathology


  • Aminopyridines
  • Betahistine