A controlled trial of 5-hydroxy-L-tryptophan for ataxia in progressive myoclonus epilepsy

To study the role of serotonin in the ataxia of progressive myoclonus epilepsy, eight patients received oral 5-hydroxy-L-tryptophan (L-5-HTP) or placebo plus carbidopa for 1 month in a controlled, double-blinded, dose-ranging, cross-over, add-on study. Ataxia was evaluated using objective and subjective scales. All of the subjects had significantly slower motor performance on timed, repetitive tasks than controls and had moderately severe ataxia. L-5-HTP was not efficacious for ataxia or speed of motor performance. Serotonergic drugs that work through mechanisms different than L-5-HTP may be more effective in correcting the abnormal serotonergic neurotransmission suggested by reduction of serotonin metabolites in cerebrospinal fluid in patients with progressive myoclonus epilepsy.