Treating Dogs, Curing Humans?

GAINSVILLE, Fla. On the outside, she's any other 5 year old. But on the inside, Haylee Fiske's body can't keep up. Haylee suffers from glycogen storage disease. Her liver can't release sugar fast enough.

"Their brains end up with hypoglycemia," David Weinstein, MD, MSC director of the glycogen storage disease program, at the University of Florida, Gainesville, told Ivanhoe. "And they can either end up developmentally delayed or unfortunately even dying."

The only treatment for Haylee's condition is a strict meal schedule. Dr. Weinstein at the University of Florida is pioneering a treatment. After two injections of genes designed to restore a faulty enzyme, a dog with the same condition can now eat a normal diet.

"The disease is so mild now that we have not had to do any medical intervention,"Dr. Weinstein explained.

Another treatment in dogs brings hope to patients with muscular dystrophy. A new drug cocktail helped this dog go from struggling to walk, to running. Experts say the injection works like a band-aid that covers up the genetic mutation linked to muscle weakness.

"It's almost if you were reading a recipe, and instead of putting salt, your mutation said put in lots of hot pepper instead," Eric, Hoffman, Ph.D., professor of pediatrics at Children's National Medical Center, Washington D.C. said. "Well, your cake wouldn't be so great. This band-aid lets it skip over that wrong pepper instruction."

Trials of the treatment have begun in humans and could eventually help people like Drew Donner.

"How's life any fun if you're just going to be a downer? Why not just live it and be happy," Donner said.

For now, Drew takes on his obstacles with a smile and waits for medicine's next solution.

Dr. Weinstein says without treatment, dogs with Haylee's condition die within an hour of birth. Before Dr. Weinstein's treatment, the longest a dog had lived with the disease was 28 days. The treated dog is now two and a half years old.

FOR MORE INFORMATION, CONTACT:
David Weinstein, MD, MSc
University of Florida
Gainesville, FL
weinsda@peds.ufl.edu

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