Clinical Research at USF may develop treatment for difficult neuromuscular disease
Closer to a treatment for Friedreich's Ataxia
10:13 PM, Sep 4, 2013
2:33 AM, Sep 5, 2013
TAMPA - Lealan LaRoche is in the middle of an important six month check-up, she doesn't have the same reflexes you probably have.
That's because she has Friedreich's Ataxia, a devastating neuromuscular disease.
"Friedreich's Ataxia is a multi-system disease primarily affecting the neuromuscular system.
The symptoms can vary from poor coordination, fine motor skill impairment such as speech and handwriting, but it begins to affect other systems like the heart, the pancreas, and the spinal cord," Dr. Theresa Zesiewicz said.
Lealan says that right now there is no treatment or cure for her disease.
"The most serious thing would be heart failure because of this disease."
But Lealan is determined to turn her diagnosis into something positive by dedicating herself to fundraising for research - and even participating herself. She's enrolled in an on-going clinical trial at USF.
Dr. Zesiewicz is a Professor of Neurology and Director of USF Ataxia Center.
"This is a drug called epi-743, which is a vitamin E, Co Q 10 derivative. There were 60 spots open at three sites."
USF being one of them - the trial is full and already six months into the process. Lealan says it requires a lot from her.
"I'm given an antioxidant, three doses daily, and after every three, six, and 12 months, I'm reassessed to see where my progression point is and if the medicine or placebo is doing what it's supposed to be doing."
Dr. Zesiewicz said Friedreich's Ataxia is due to a problem with the mitochondria, which is the powerhouse of the cell.
"...So the thinking is this drug works on the mitochondria of the cell to improve function for these patients.
Our hope is that this will have systematic benefit for Friedreich's Ataxia patients, improve coordination, improve gait and balance and significantly improve the quality of life for the patient."
The doctor expects results in a year.
Events are being held tomorrow and this weekend to raise awareness and funds toward a cure. The details can be found below:
Understanding A Cure, Friedreich's Ataxia Scientific Symposium
Thursday, September 5
6:00 - 8:30 p.m.
USFHealth's Center for Advanced Medical Learning and Simulation