Branched-chain amino acid therapy for spinocerebellar degeneration: A pilot clinical crossover trial

Object: The potential effects of branched-chain amino acids (BCAAs) on spin ocerebellar degeneration (SCD) were explored in eleven patients. Methods: T he patients received 200 mi of BCAA-rich solution, 2 mg of thyrotropin-rele asing hormone (TRH; protirelin), or a placebo daily for 7 days each in a ra ndom order. An SCD score was used to quantify the severity of symptoms. Pat ients: Eleven patients with SCD (7 male, 4 female; mean age 60+/-11; mean d isease duration 5.5 years) participated in this study. Results: The mean SC D score of the eleven patients improved significantly by the BCAA treatment compared with the baseline. The conditions of five of the eleven patients (45%) were clearly improved by the BCAA treatment. All of the responders ma nifested predominantly cerebellar symptoms, but no prominent parkinsonian s ymptoms. Two patients with marked rigidity and akinesia did not respond to the treatment. Conclusion: We concluded that BCAAs do have a beneficial eff ect on functional improvement in patients with SCD, and that further large scale studies are needed.