Idebenone may improve patients with Friedreich’s ataxia in increasing lipid peroxydation

P. Arnold, T. Kuntzer, O. Boulat, M. Markert, J. Bogousslavsky (CHUV, 1011 Lausanne, Switzerland)

Mise en ligne le 11 / 07 / 02

Friedreich’s ataxia (FA), the most common form of degenerative ataxia, is thought to be caused by respiratory chain deficiency, with mitochondrial iron accumulation and oxidative stress. Idebenone, a free-radical scavenger, protects mitochondrial function in in vitro FA models. A recent study has reported improvement of FA-associated cardiomyopathy with idebenone, but its effect on neurological dysfunctions remains unknown.

To collect clinical and biochemical data in order to measure neurological effect of idebenone.

19 FA patients (mean age 33.6 years, range 13 to 68) agreed to take idebenone and were prospectively followed. The international cooperative ataxia rating scale (ICARS) was used to quantify ataxia. Blood samples were collected to measure malondialdehyde (MDA) levels, a product of lipid peroxydation.


Results (cnt)
• MDA has been measured in whole blood on EDTA, and levels were increased in FA patients treated with idebenone, compared with those without treatment (p= 0.04) (Fig. 2)

Fig. 2 MDA level with and without idebenone
• There was a clear increase of MDA levels after introduction of treatment with idebenone (Fig. 3).

Fig3 MDAlevels in three FA patients before and after treatment with idebenone