Branched-chain amino acid therapy for spinocerebellar degeneration: a pilot clinical crossover trial.
OBJECTIVE
The potential effects of branched-chain amino acids (BCAAs) on spinocerebellar degeneration (SCD) were explored in eleven patients.


METHODS
The patients received 200 ml of BCAA-rich solution, 2 mg of thyrotropin-releasing hormone (TRH; protirelin), or a placebo daily for 7 days each in a random order. An SCD score was used to quantify the severity of symptoms.


METHODS
Eleven patients with SCD (7 male, 4 female; mean age 60+/-11; mean disease duration 5.5 years) participated in this study.


RESULTS
The mean SCD 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 manifested predominantly cerebellar symptoms, but no prominent parkinsonian symptoms. Two patients with marked rigidity and akinesia did not respond to the treatment.


CONCLUSIONS
We concluded that BCAAs do have a beneficial effect on functional improvement in patients with SCD, and that further large scale studies are needed.
Mori
N
N
Division of Neurology, Institute of Neurological Sciences, Tottori University, Faculty of Medicine, Yonago.
Adachi
Y
Y
Takeshima
T
T
Kashiwaya
Y
Y
Okada
A
A
Nakashima
K
K
D000328:Adult; D000368:Aged; D000597:Amino Acids, Branched-Chain / Q000627:therapeutic use*; D018592:Cross-Over Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D012720:Severity of Illness Index; D016037:Single-Blind Method; D013132:Spinocerebellar Degenerations / Q000188:drug therapy* / Q000503:physiopathology; D013973:Thyrotropin-Releasing Hormone / Q000627:therapeutic use; D016896:Treatment Outcome
D000597:Amino Acids, Branched-Chain; D013973:Thyrotropin-Releasing Hormone