Abstract
Treatment with 6R-L-erythro-5,6,7,8-tetrahydrobiopterin (R-THBP) has been suggested to improve autistic behavior. Cerebrospinal fluid (CSF) levels of total biopterin, oxidized and reduced forms of biopterin, homovanillic acid, and 5-hydroxyindoleacetic acid were measured in 14 autistic 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 other 7 patients did not (nonresponders). There were no significant differences between responders, nonresponders, and controls in the CSF levels of the metabolites before R- THBP administration. When lumbar puncture was repeated in 6 autistic children in the 24th week of R-THBP therapy, there was no significant change in the CSF levels of any metabolites.
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We thank Edward M. Ornitz for his helpful advice. Our work has partly supported by Grant 2A-5B-32 from the National Center of Neurology and Psychiatry of the Japanese Ministry of Health and Welfare. We also thank Suntory Ltd. for supporting our work.
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Komori, H., Matsuishi, T., Yamada, S. et al. Cerebrospinal fluid biopterin and biogenic amine metabolites during oral R-THBP therapy for infantile autism. J Autism Dev Disord 25, 183–193 (1995). https://doi.org/10.1007/BF02178503
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DOI: https://doi.org/10.1007/BF02178503