Preliminary trial findings shared with FARA-USF research symposium participants
News kindly supplied by Juan Carlos Baiges
August 31, 2011
For the first time, an investigative drug has significantly improved the neurological function of patients with Friedreich’s ataxia.
The promising findings of a preliminary clinical trial testing the drug candidate known as EPI-A0001 was greeted with applause and cheers August 25 at the third annual scientific symposium hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the University of South Florida Ataxia Research Center directed by Theresa Zesiewicz, MD, USF Health professor of neurology.
FARA Executive Director Jennifer Farmer presented results of the double blind, placebo-controlled 28-day study conducted by Edison Pharmaceuticals, Inc., because the travel of speaker Guy Miller, MD, PhD, Edison’s CEO, was delayed by Hurricane Irene. The study found measures of balance and coordination were significantly better in the group of patients given high doses of EPI-A0001 as well as the group receiving lose doses of the drug in comparison to the placebo group. There were no differences in rates of adverse side effects between the placebo group and each of the drug-treated groups.
“This is the first clinical trial where patients were given a drug and they’ve shown neurological improvement,” Farmer said. “It’s incredibly hopeful… but there’s still a lot of work ahead. We need to show the drug really makes a difference over six months or a year.”