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Drug hope for Friedreich's ataxia
An antioxidant drug that is hoped to slow the progression of the neurological genetic disease Friedreich ataxia is about to enter trials in Melbourne and the United States.
Associate Professor Martin Delatycki, head of the Bruce Lefroy Centre at the Murdoch Childrens Research Institute, said the drug called mitoquinone, harbours a chemical that targets mitochondria, the energy source of cells.
The disease is usually diagnosed in children and teenagers, with early symptoms including unsteady gait, jerky eye movements and loss of reflexes. Despite existing treatments, which include physiotherapy, speech therapy and some orthopaedic surgery, most people are confined to a wheelchair within 15 years of disease onset.
Carrie Beetham, 28, was diagnosed with Friedreich ataxia 12 years ago and intends to participate in the mitoquinone trial.
"There has never been a controlled drug trial for Friedreich's Ataxia and this is the first one to show real potential benefit," Carrie said. "No one knows what the results will be, but we can hope for the absolute best. This is our biggest source of hope."