Dr. David Weinstein, a pediatrician at the University of Florida, now shares something with Pope John Paul II, Nelson Mandela and Oprah Winfrey. He joins these luminaries as a new recipient of the Order of the Smile Award, an international humanitarian award from an organization that goes by the award’s same name and is based in Poland.
“It’s a little bit of a shock considering I deal with a disease most people and even most doctors know nothing about,” Weinstein said. “This is the first time that the humanitarian part of our efforts has been recognized. Ultimately we’re in the business of helping kids.”
Notably, Weinstein was nominated for the award by his patients: children with a rare genetic condition called glycogen storage disease (GSD), in which they have missing or defective enzymes responsible for breaking down sugar in the body, causing it to build up in the liver and muscles.
Children that Weinstein has treated—from more than 30 countries—and their parents wrote letters to the Order of the Smile Foundation about how Weinstein had saved them. “It really shows the power of social media,” Weinstein said.
But the real power is his own single-minded devotion to a disease that no one else was touching when he came across it as a fellow at Harvard University 14 years ago.
“Somebody had to care about these kids. I decided that they needed a champion,” he said.
Weinstein moved to the University of Florida in 2005 in order to work with the Gene Therapy Institute on developing genetic therapies for the disease to ultimately find a cure. “We’ve progressed to the point of applying to the FDA to try to perform genetic therapy in humans since it’s worked well in dogs,” Weinstein said.
In moving south, Weinstein said he also hoped to broaden a clinical reference point for children with GSD. “One of the things I wanted to do was create a Disney World of health care for these kids,” he said.
That happened, and Weinstein has treated 425 children from all over the U.S. and 36 countries and Gainesville and helped advise hundreds more online. He also created a charity to help children who wouldn’t otherwise be able to travel to be seen by him. For example, the US Army met a child in Afghanistan who, because of the charity, was able to travel to Gainesville.
Weinstein also travels once a month to Europe and South America to educate physicians on this rare disease.
Most kids with GSD begin to show signs between four and eight months: they may not grow properly, or they have abdominal swelling. If the disease goes undiagnosed and untreated, children can die or suffer brain damage. The low blood sugar kids have during the night from fasting can cause sudden infant death syndrome (SIDs), Weinstein said, adding that in certain communities that tend to marry within the community, such as Jews and Latter Day Saints, the disease was once a major cause of SIDs.
The disease is primarily genetic and affects about one in every 100,000 babies.
Written by Kristine Crane. Read more at The Gainesville Sun.