title: Double-blind crossover study of branched-chain amino acid therapy in patients with spinocerebellar degeneration.
abstract: To determine whether treatment with branched-chain amino acids (BCAA) can improve the condition of patients with ataxia, a double-blind crossover study of BCAA therapy was performed in 16 patients with spinocerebellar degeneration (SCD). The patients were treated with BCAA in oral doses of 1.5, 3.0, or 6.0 g or with placebo daily for 4 weeks in each study phase. The order of treatment phases (placebo or BCAA) was assigned randomly. An International Cooperative Ataxia Rating Scale (ICARS) was used to quantify the severity of symptoms of SCD. The mean ICARS score improved significantly with BCAA treatment compared with the mean pretreatment score (p<0.01). In addition, the improvement in the mean global ICARS score was significant in the middle-dose group compared with that in the placebo group (p<0.02). The estimated improvement in kinetic functions compared with pretreatment (p<0.01) was significant after treatment with BCAA, 1.5 and 3.0 g. All of the responders manifested predominantly cerebellar symptoms, especially those with spinocerebellar ataxia type 6 (SCA6). Thus, treatment with BCAA may be effective in patients with the cerebellar form of SCD.
authors.affiliation: Division of Neurology, Institute of Neurological Sciences, Faculty of Medicine, Tottori University, 36-1 Nishimachi, Tottori 683-8504, Yonago, Japan.
authors.forename: Masatada
authors.initials: M
authors.lastname: Mori
authors.forename: Yoshiki
authors.initials: Y
authors.lastname: Adachi
authors.forename: Nozomi
authors.initials: N
authors.lastname: Mori
authors.forename: Saiko
authors.initials: S
authors.lastname: Kurihara
authors.forename: Yoshihiro
authors.initials: Y
authors.lastname: Kashiwaya
authors.forename: Masayoshi
authors.initials: M
authors.lastname: Kusumi
authors.forename: Takao
authors.initials: T
authors.lastname: Takeshima
authors.forename: Kenji
authors.initials: K
authors.lastname: Nakashima
mesh_terms: D000368:Aged; D000597:Amino Acids, Branched-Chain / Q000008:administration & dosage* / Q000009:adverse effects; D002531:Cerebellum / Q000187:drug effects* / Q000378:metabolism / Q000503:physiopathology; D018592:Cross-Over Studies; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D018698:Glutamic Acid / Q000172:deficiency* / Q000235:genetics; D006801:Humans; D008297:Male; D008875:Middle Aged; D020127:Recovery of Function / Q000187:drug effects / Q000502:physiology; D013132:Spinocerebellar Degenerations / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D013569:Synapses / Q000187:drug effects* / Q000378:metabolism; D016896:Treatment Outcome
chemical_list: D000597:Amino Acids, Branched-Chain; D018698:Glutamic Acid