Riluzole therapy in Huntington's disease (HD).
We conducted a 6-week open-label trial of riluzole (50 mg twice a day) in eight subjects with Huntington's disease. Subjects were evaluated before riluzole treatment, on treatment, and off treatment with the chorea, dystonia, and total functional capacity (TFC) scores from the Unified Huntington's Disease Rating Scale and magnetic resonance spectroscopy measurements of occipital cortex and basal ganglia lactate levels. Adverse events and safety blood and urine tests were assessed throughout the study. All subjects completed the study and riluzole was well tolerated. The age was 45+/-10.2 years (mean +/- standard deviation) and the disease duration was 6.1+/-4.1 years. The chorea rating score improved by 35% on treatment (p = 0.013) and worsened after discontinuation of treatment (p = 0.026). There were no significant treatment effects on the dystonia or TFC scores. The baseline occipital and basal ganglia lactate levels were elevated in all subjects; there was a trend toward lower lactate/creatine ratios during riluzole treatment in the basal ganglia spectra but not in occipital cortex spectra. Additional clinical studies of riluzole for both symptomatic and neuroprotective benefit in Huntington's disease are warranted.
Rosas
H D
HD
Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston 02114, USA.
Koroshetz
W J
WJ
Jenkins
B G
BG
Chen
Y I
YI
Hayden
D L
DL
Beal
M F
MF
Cudkowicz
M E
ME
D000328:Adult; D001479:Basal Ganglia / Q000187:drug effects / Q000378:metabolism; D002819:Chorea / Q000188:drug therapy; D018691:Excitatory Amino Acid Antagonists / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy* / Q000378:metabolism; D019344:Lactic Acid / Q000378:metabolism; D009682:Magnetic Resonance Spectroscopy; D008297:Male; D008875:Middle Aged; D018696:Neuroprotective Agents / Q000009:adverse effects / Q000627:therapeutic use*; D009778:Occipital Lobe / Q000187:drug effects / Q000378:metabolism; D010865:Pilot Projects; D019782:Riluzole / Q000009:adverse effects / Q000627:therapeutic use*; D012720:Severity of Illness Index; D016037:Single-Blind Method; D016896:Treatment Outcome
D018691:Excitatory Amino Acid Antagonists; D018696:Neuroprotective Agents; D019344:Lactic Acid; D019782:Riluzole