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Three-year Follow-up Shows Antioxidant Treatment Improves Friedreich's Ataxia: Presented at MDS

By Bruce Sylvester
Special to DG News

MIAMI, FL -- November 15, 2002 -- Antioxidant therapy appears to improve mitochondrial energy synthesis over time, slowing the clinical progression of Friedreich's ataxia and providing a significant improvement in heart function, researchers report.

"This [finding] supports the establishment of a double blind, placebo controlled trial which is currently underway," said Paul Hart MD, research clinician at the department of neuroscience, Royal Free and University Medical College, in London, England.

"This investigative use of a background of pathogenic information is now moving into the area of potential treatment options," he told Doctor's Guide, during his presentation here November 14 at the Movement Disorders Society's (MDS) 7^th International Congress of Parkinson's Disease and Movement Disorders.

Previous research has indicated that decreased mitochondrial respiratory chain function and increased oxidative stress is part of the pathogenesis of Friedreich's ataxia. In vivo phosphorus magnetic resonance spectroscopy (31P-MRS) has confirmed decreased mitochondrial energy metabolism in heart and skeletal muscle of patients with Friedreich's ataxia.

The purpose of the study was to investigate whether patients with Friedreich's ataxia might benefit from long-term vitamin E and coenzyme Q10 therapy.

The investigators followed 10 patients with genetically defined Friedreich's ataxia for three years. They used ICARS and echocardiography to evaluate each subject at years 1, 2 and 3 on the same therapy to determine effects on the clinical progression of the disease. They also used 31P-MRS to evaluate the effect on mitochondrial bioenergetics of the heart and skeletal muscle.

After three and six months antioxidant treatment (coenzyme Q10, 400 mg/day and vitamin E, 2100 IU/day) cardiac and skeletal muscle MRS bioenergetic parameters were significantly improved in a small cohort of patients, Dr. Hart said. The influence upon clinical progression was not detectable after such a short period of time, he reported.

Heart and skeletal muscle MRS improvements were maintained at each yearly evaluation. Echocardiography data indicated that the fraction shortening at 36 months for the subjects as a group was significantly increased relative to the pre-therapy data.

The investigators compared the progression of patients' clinical scores with cross-sectional data. They found that clinical scores for eight patients were better than predicted, while two patients' scores declined as expected.

"We can now look at rational therapies focusing on mitochondrial disorders and see if they benefit these patients both clinically and by the surrogate markers," Dr. Hart said.

The Royal Free and University Medical College in London supported the research.

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