Journal of Neural Transmission

, Volume 112, Issue 6, pp 789–796 | Cite as

L-carnitine and creatine in Friedreich’s ataxia. A randomized, placebo-controlled crossover trial

  • L. Schöls
  • J. Zange
  • M. Abele
  • M. Schillings
  • G. Skipka
  • S. Kuntz-Hehner
  • M. C. P. van Beekvelt
  • W. N. J. M. Colier
  • K. Müller
  • T. Klockgether
  • H. Przuntek
  • M. Vorgerd
Article

Summary.

Impaired oxidative phosphorylation is a crucial factor in the pathogenesis of Friedreich’s ataxia (FA). L-carnitine and creatine are natural compounds that can enhance cellular energy transduction. We performed a placebo-controlled triple-phase crossover trial of L-carnitine (3 g/d) and creatine (6.75 g/d) in 16 patients with genetically confirmed FA. Primary outcome measures were mitochondrial ATP production measured as phosphocreatine recovery by 31Phosphorus magnetic resonance spectroscopy, neurological deficits assessed by the international co-operative ataxia rating scale and cardiac hypertrophy in echocardiography. After 4 months on L-carnitine phosphocreatine recovery was improved compared to baseline (p < 0.03, t-test) but comparison to placebo and creatine effects did not reach significance (p = 0.06, F-test). Ataxia rating scale and echocardiographic parameters remained unchanged. Creatine had no effect in FA patients. L-carnitine is a promising substance for the treatment of FA patients, and larger trials are warranted.

Keywords: Friedreich’s ataxia, therapy, L-carnitine, oxidative phosphorylation, magnetic resonance spectroscopy. 

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Copyright information

© Springer-Verlag/Wien 2004

Authors and Affiliations

  • L. Schöls
    • 1
  • J. Zange
    • 2
  • M. Abele
    • 3
  • M. Schillings
    • 4
  • G. Skipka
    • 5
  • S. Kuntz-Hehner
    • 6
  • M. C. P. van Beekvelt
    • 7
  • W. N. J. M. Colier
    • 7
  • K. Müller
    • 2
  • T. Klockgether
    • 3
  • H. Przuntek
    • 8
  • M. Vorgerd
    • 9
  1. 1.Centre of Neurology and Hertie-Institute for Clinical Brain Research, University of TübingenGermany
  2. 2.Institute of Aerospace Medicine, German Aerospace CentreCologneGermany
  3. 3.Department of NeurologyUniversity of BonnGermany
  4. 4.Department of CardiologySt. Josef HospitalGermany
  5. 5.Institute of Medical Informatics, Ruhr-University BochumGermany
  6. 6.Department of CardiologyUniversity of BonnGermany
  7. 7.Departments of Geriatric Medicine and PhysiologyUniversity Medical CentreNijmegenThe Netherlands
  8. 8.Department of NeurologySt. Josef HospitalGermany
  9. 9.Department of Neurology, Kliniken BergmannsheilRuhr-University BochumGermany

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