Short-term effects of coenzyme Q10 in progressive supranuclear palsy: a randomized, placebo-controlled trial.
Mitochondrial complex I appears to be dysfunctional in progressive supranuclear palsy (PSP). Coenzyme Q(10) (CoQ(10)) is a physiological cofactor of complex I. Therefore, we evaluated the short-term effects of CoQ(10) in PSP. We performed a double-blind, randomized, placebo-controlled, phase II trial, including 21 clinically probable PSP patients (stage < or = III) to receive a liquid nanodispersion of CoQ(10) (5 mg/kg/day) or matching placebo. Over a 6-week period, we determined the change in CoQ(10) serum concentration, cerebral energy metabolites (by (31)P- and (1)H-magnetic resonance spectroscopy), motor and neuropsychological dysfunction (PSP rating scale, UPDRS III, Hoehn and Yahr stage, Frontal Assessment Battery, Mini Mental Status Examination, Montgomery Asberg Depression Scale). CoQ(10) was safe and well tolerated. In patients receiving CoQ(10) compared to placebo, the concentration of low-energy phosphates (adenosine-diphosphate, unphosphorylated creatine) decreased. Consequently, the ratio of high-energy phosphates to low-energy phosphates (adenosine-triphosphate to adenosine-diphosphate, phospho-creatine to unphosphorylated creatine) increased. These changes were significant in the occipital lobe and showed a consistent trend in the basal ganglia. Clinically, the PSP rating scale and the Frontal Assessment Battery improved slightly, but significantly, upon CoQ(10) treatment compared to placebo. Since CoQ(10) appears to improve cerebral energy metabolism in PSP, long-term treatment might have a disease-modifying, neuroprotective effect.
Stamelou
Maria
M
Department of Neurology, Philipps University, Marburg, Germany.
Reuss
Alexander
A
Center for Clinical Trials, Philipps University, Marburg, Germany.
Pilatus
Ulrich
U
Brain Imaging Center and Institute of Neuroradiology, J.W. Goethe University, Frankfurt, Germany.
Magerkurth
Jörg
J
Brain Imaging Center and Institute of Neuroradiology, J.W. Goethe University, Frankfurt, Germany.
Niklowitz
Petra
P
Children's Hospital Datteln, Witten-Herdecke University, Germany.
Eggert
Karla M
KM
Department of Neurology, Philipps University, Marburg, Germany.
Krisp
Andrea
A
Department of Neurology, Philipps University, Marburg, Germany.
Menke
Thomas
T
Children's Hospital Datteln, Witten-Herdecke University, Germany.
Schade-Brittinger
Carmen
C
Center for Clinical Trials, Philipps University, Marburg, Germany.
Oertel
Wolfgang H
WH
Department of Neurology, Philipps University, Marburg, Germany.
Höglinger
Günter U
GU
Department of Neurology, Philipps University, Marburg, Germany.
D000328:Adult; D000368:Aged; D001479:Basal Ganglia / Q000187:drug effects / Q000378:metabolism; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D009682:Magnetic Resonance Spectroscopy; D008297:Male; D008875:Middle Aged; D018696:Neuroprotective Agents / Q000494:pharmacology* / Q000627:therapeutic use*; D013494:Supranuclear Palsy, Progressive / Q000188:drug therapy*; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use
D018696:Neuroprotective Agents; D014451:Ubiquinone; C024989:coenzyme Q10