USF Ataxia Research Center Leads Clinical Trial of Possible Friedreich's Drug

For the last few years, the USF Ataxia Research Center and the Friedreich's Ataxia Research Alliance (FARA) have hosted a pair of events in Tampa: a national symposium that brings together Friedreich ataxia experts from around the world, and a gala ball that raises hundreds of thousands of dollars for research.

While they're preparing for this year's events, there's a bit of extra pressure -- the USF Ataxia Research Center is also the lead site on a national study of a potent antioxidant treatment for the life-shortening, degenerative neuromuscular disorder.

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Mark Schreiner's University Beat report on the USF Ataxia Research Center

Dr. Theresa Zesiewicz said Friedreich's ataxia normally strikes the very young.

"Typically, these are kids who all of a sudden have problems with running, it’s a little bit difficult," she said. "Mom and dad carry the gene but neither one knows that they carry the gene, so it’s a big surprise to the parents."

From there, Zesiewicz added, Friedreich's ataxia "progresses relentlessly to cause gait imbalance, talking difficulties, speech problems, hearing problems, and eventually causes diabetes in many cases, and also heart failure.”

Currently, doctors use physical therapy to work on a patient’s core strength and balance because there’s no approved pharmaceutical treatment for Friedreich’s. That may soon change, as USF is leading a national study of EPI-743.

The drug, developed by Edison Pharmaceuticals, Inc., protects the mitochondria in nerve cells in the spinal cord that Friedreich’s attacks.

“It's like an antioxidant, but an extremely strong antioxidant, much stronger than the CoQ10 or Vitamin E that you see in your drugstore, for example,” Zesiewicz says.

The Children's Hospital of Philadelphia and UCLA are the two other sites in the placebo-controlled study of EPI-743. Zesiewicz hopes that the drug might not be limited to just being the first real treatment for Friedreich’s.

“It may have other implications for diseases like Parkinson’s disease, Alzheimer’s disease, and other neuro-degenerative diseases,” she said.

Natchez Hanson, 24, has been taking part in the trial for about eight months. She doesn’t know if she’s receiving the actual drug or a placebo, but is apparently seeing some benefits.

“I kind of feel like I’m the same, but people tell me that I’m standing better, I’m walking better, I sound better," Hanson said.

The Polk County math teacher, who was formally diagnosed with Friedreich's ataxia at age 17, now uses a walker to get around.

"That just has opened so many doors," she said, with a smile, "considering that people mostly open the doors for me, but I mean, it carries all my stuff, I get front row parking!"

In September, for the third straight year, Hanson will speak at the USF-FARA symposium and share her rather upbeat story of living with the disease.

“Well you only have one life to live, and everyone has a burden to bear," Hanson said. "I always say either you laugh about it or you cry about it, and I’m done crying. I’ve moved on from that, so now I’m just trying to be as happy as I possibly can be.” (Hear more from Hanson here.)

"Understanding A Cure: The Friedreich's Ataxia Scientific Symposium" will be held at 5 p.m. Thursday, Sept. 5 at USF Health’s Center for Advanced Medical Learning and Simulation (CAMLS) in downtown Tampa. The event is free and open to the public (click here to RSVP) and will also be live-streamed by FARA.

Edison Pharmaceuticals Chairman/CEO, Dr. Guy Miller, will update the status of the EPI-743 clinical trial, while Dr. Mirella Dottori of the University of Melbourne, Australia, will talk about treating Friedreich's ataxia with stem cells.

Then on Saturday, Sept. 7, it's the 2013 FARA Energy Ball, a fund-raiser for USF and FARA that's being held at the A La Carte Event Pavilion in Tampa. Click here for ticket info.

http://wusfnews.wusf.usf.edu/post/usf-ataxia-research-center-leads-clinical-trial-possible-friedreichs-drug

Really hope this is a breakthrough for Friedreich's, Thanks again Alan

Very hopeful. Thanks Alan. xB