Idebenone, a compound similar to coenzyme Q10, continues to show promise in treating the heart complications of Friedreich's ataxia (FA), notes correspondence published in the Feb. 10 issue of Neurology.
Researchers had previously reported significant benefits for the FA-affected heart after short-term treatment with the compound, which has been approved for use by European regulators but not by the U.S. Food and Drug Administration.
The new report says that a French patient who took 5 milligrams per kilogram of body weight of idebenone per day for five years had significant reduction of abnormal heart enlargement and complete resolution of heart dysfunction. Cardiac enzymes were largely restored to normal. The researchers describe the drug as having had a “spectacular effect.”
Idebenone is being tested at the National Institutes of Health in Bethesda, Md., under the direction of neurologist Kenneth Fischbeck. (See Idebenone -- Phase 1 Clinical Trial for more information.)
“A dose-escalation tolerability study is nearing completion, and a longer-term trial of high-dose treatment is planned,” Fischbeck said. “The hope is that high-dose treatment will prove to be safe and may be more effective than the doses used in other studies to date.”