Riluzole improves symptoms of cerebellar ataxia, according to a new placebo-controlled study. Riluzole is an approved treatment for ALS that may reduce hyperexcitability of neurons in the deep cerebellar nuclei.
Forty patients with cerebellar ataxia of different etiologies (including hereditary ataxias, MSA-C, FXTAS, and Friedreich’s ataxia) were randomized to placebo or 50 mg riluzole twice daily for 8 weeks. Baseline scores on the International Cooperative Ataxia Rating Scale ranged from 12 to 70 out of 100.
The number of patients with at least a 5-point improvement in the ICARS was greater in the riluzole group than in the placebo group at both 4 weeks (9/19 vs. 1/19, odds ratio=16.2) and 8 weeks (13/19 vs. 1/19, OR=39). Riluzole improved the mean ICARS score by 7 points versus a 0.2 point worsening for placebo. Improvements were seen on the subscores for static function, kinetic function, and dysarthria, but not for oculomotor function. One patient experienced a slight increase in alanine aminotransferase.
The results, the authors conclude, “may warrant the long-term use of riluzole in chronic cerebellar ataxia.”
Riluzole in cerebellar ataxia: A randomized, placebo-controlled pilot trial G Ristori, S Romano, A Visconti, S Cannoni, M Spadaro, M Frontali, FE Pontieri, N Vanacore, M Salvetti Neurology 2010;74:839-845