For Dulce, 14 months without cornstarch is a very good thing.
As a Maltese puppy, Dulce suffers from glycogen storage disease type 1A, a disease found in both dogs and humans that prevents the body from converting glycogen to glucose, the essential fuel to keep bodies in motion.
The current treatment for the disease includes frequently administering corn starch in order to keep blood sugar in check. Before 1971, the disease was considered fatal.
The UF Glycogen Storage Disease Program is hoping to transform current treatment methods after its recent and successful attempt at gene therapy in dogs.
Maltese dogs, the only known animal to suffer from the naturally occurring disease, are more prone to the disease because they have been inbred for years, said Dr. David Weinstein, the director of the program.
Though Maltese are no strangers to international dog shows like Westminster Kennel Club, puppies with the disease are not among the prize-winning show dogs.
"Without treatment, the dogs with the disease could die within an hour of birth," Weinstein said.
Gene therapy has helped Maltese puppies live longer by removing the original genetic material from a liver-and-kidney-attacking virus and inserting a desired gene sequence. The altered virus is injected into the body, where it attacks the liver and inserts the desired gene, Weinstein said.
Prior to gene therapy, no Maltese with this disease had survived for more than 28 days.
Dulce is now 20 months old and has been off all treatment for 18 months since her last gene therapy session in January 2008, he said.
While helping puppies is a worthy cause, Weinstein said the ultimate goal is to find a viable, effective treatment for humans who suffer from the disease.
Of the 12 types of glycogen storage disease, type 1A is the most severe and causes low blood sugar, or hypoglycemia.
Even with cornstarch, patients with type 1A can suffer from liver cancer, severe kidney disease, osteoporosis and other complications, Weinstein said.
"Children and adults with glycogen storage disease live by the clock," he said.
Precise amounts of cornstarch are consumed about every four hours, and a delayed dose will result in severe hypoglycemia, which can lead to seizures or even death, he said.
"One missed or delayed dose can be disastrous, and this causes tremendous anxiety for patients and families," Weinstein said.
Because safe gene therapy in humans is a long way off, Weinstein said, UF researchers are working on an extended-release cornstarch that could be available as soon as 2010.
"(The cornstarch) may finally allow people with (the disease) to sleep through the night," he said.
Weinstein said UF has been the exclusive site for these studies since the program moved from a branch of Harvard University to UF in 2005.
Despite groundbreaking work and more than 300 patients from 18 countries, budget cuts are threatening to end the program and any hopes for new treatments, according to Weinstein.
"We must be careful not to destroy great programs here at UF in an effort to save a little money," he said.
