CEREBROSPINAL-FLUID BIOPTERIN AND BIOGENIC-AMINE METABOLITES DURING ORAL R-THBP THERAPY FOR INFANTILE-AUTISM

Treatment with 6R-L-erythro-5,6,7,8-tetrahydrobiopterin (R-THBP) has b een suggested to improve autistic behavior. Cerebrospinal fluid (CSF) levels of total biopterin, oxidized and reduced forms of biopterin, ho movanillic acid, and 5-hydroxyindoleacetic acid were measured in 14 au tistic children and 18 controls to clarify the mechanism of action of R-THBP. The 14 autistic children received R-THBP orally at 1 mg/kg per day; 7 children showed clinical improvement (responders) and the othe r 7 patients did not (nonresponders). There were no significant differ ences between responders, nonresponders, and controls in the CSF level s of the metabolites before R-THBP administration. When lumbar punctur e was repeated in 6 autistic children in the 24th week of R-THBP thera py, there was no significant change in the CSF levels of any metabolit es.