Antioxidant Treatment of Patients With FRDA Four-Year Follow-up CQ10+Vit.E

[Guest guest]

(No, I haven't lost my mind! I am posting this because Friedreich's

Ataxia is another inherited peripheral neuropathy, and often mimick's

CMT, as we have seen in the past. Also, some of us with CMT, myself

included, were at one time diagnosed with Friedreich's Ataxia. I also

feel the length of this study is very significant and I hope we see

something like this for CMT coming out. It is about CQ10 and Vitamin

E therapy. ~ Gretchen)

Arch Neurol. 2005;62:621-626.

Antioxidant Treatment of Patients With Friedreich Ataxia

Four-Year Follow-up

E. Hart, MRCP; Raffaele Lodi, MD; Bheeshma Rajagopalan, MD; Jane

L. Bradley, PhD; Jenifer G. Crilley, MD; , MRCP;

M. Blamire, PhD; Manners, DPhil; Styles, DPhil;

H. V. Schapira, MD, DSc; J. Mark , PhD

Background: Decreased mitochondrial respiratory chain function and

increased oxidative stress have been implicated in the pathogenesis

of Friedreich ataxia (FRDA), raising the possibility that energy

enhancement and antioxidant therapies may be an effective treatment.

Objective: To evaluate the long-term efficacy of a combined

antioxidant and mitochondrial enhancement therapy on the

bioenergetics and clinical course of FRDA.

Design: Open-labeled pilot trial over 47 months.

Patients: Seventy-seven patients with clinical and genetically

defined FRDA.

Intervention: A combined coenzyme Q10 (400 mg/d) and vitamin E (2100

IU/d) therapy of 10 patients with FRDA over 47 months.

Main Outcome Measures: Clinical assessment using echocardiography

and the International ative Ataxia Rating Scale and cardiac and

skeletal muscle bioenergetics as assessed using phosphorus P 31

magnetic resonance spectroscopy.

Results: There was a significant improvement in cardiac and skeletal

muscle bioenergetics that was maintained throughout the 47 months of

therapy. Echocardiographic data revealed significantly increased

fractional shortening at the 35- and 47-month time points. Comparison

with cross-sectional data from 77 patients with FRDA indicated the

changes in total International ative Ataxia Rating Scale and

kinetic scores over the trial period were better than predicted for 7

patients, but the posture and gait and hand dexterity scores

progressed as predicted.

Conclusion: This therapy resulted in sustained improvement in

mitochondrial energy synthesis that was associated with a slowing of

the progression of certain clinical features and a significant

improvement in cardiac function.

Author Affiliations: University Department of Clinical Neurosciences,

Royal Free and University College Medical School, London (Drs Hart,

Bradley, , Schapira, and ); Medical Research Council

Biochemical and Clinical Magnetic Resonance Unit, Department of

Biochemistry, University of Oxford and Oxford Radcliffe Hospital,

Oxford (Drs Lodi, Rajagopalan, Crilley, Blamire, Manners, and

Styles); and the Institute of Neurology, University College (Dr

Schapira), London, England; and the Dipartimento di Medicina Clinica

e Biotecnologia Applicata, Università di Bologna, Bologna, Italy (Dr

Lodi).