The effect of 3,4-diaminopyridine on the patients with hereditary pure cerebellar ataxia

J Neurol Sci. 2010 May 15;292(1-2):81-4. doi: 10.1016/j.jns.2010.01.021. Epub 2010 Feb 23.

Abstract

Background: Downbeat nystagmus (DBN) is often seen in patients with pure cerebellar type of spinocerebellar ataxia (SCA) like spinocerebellar ataxia type 6 (SCA6). DBN frequently presents with other cerebellar symptoms such as postural imbalance or ataxia. A potassium channel blocker 3,4-diaminopyridine (3,4-DAP) has been reported to reduce DBN by increasing the excitability of Purkinje cells.

Objective: The objective of this study is to determine whether 3,4-DAP has a beneficial effect on DBN along with postural imbalance and ataxic symptoms in 10 patients with SCA6 and five patients with chromosome 16q22.1-linked autosomal dominant cerebellar ataxia (16q-ADCA).

Results: The patients took 20mg of 3,4-DAP twice a day for a week. DBN was observed in seven patients with SCA6 and two with 16q-ADCA. Although 3,4-DAP significantly reduced DBN (P<0.05), other ataxic symptoms did not improved. However, 3,4-DAP showed benefit in two patients with oscillopsia.

Conclusion: 3,4-DAP may be effective on DBN and oscillopsia, although it was not proved to be effective on other symptoms of ataxia in SCA patients.

MeSH terms

  • 4-Aminopyridine / analogs & derivatives*
  • 4-Aminopyridine / therapeutic use
  • Amifampridine
  • Ataxia / complications
  • Ataxia / drug therapy
  • Ataxia / genetics
  • Drug Administration Schedule
  • Female
  • Genetic Predisposition to Disease
  • Humans
  • Male
  • Nystagmus, Congenital / complications
  • Nystagmus, Congenital / drug therapy*
  • Nystagmus, Congenital / genetics
  • Posture
  • Potassium Channel Blockers / therapeutic use
  • Spinocerebellar Ataxias / complications
  • Spinocerebellar Ataxias / drug therapy*
  • Spinocerebellar Ataxias / genetics
  • Treatment Outcome

Substances

  • Potassium Channel Blockers
  • 4-Aminopyridine
  • Amifampridine