Trinity Grad David Weinstein Tackles Glycogen Storage Disease

Weinstein tells Students to follow their Heart in Choosing Career
David Weinstein ’90, who has achieved great success as a medical researcher in the previously unheralded field of glycogen storage, attributed his job satisfaction and good fortune to the “amazing faculty” he encountered while an undergraduate at Trinity.
“I feel very fortunate to do something that I love,” he told a Common Hour audience in the Washington Room. “It was a wise decision to come to Trinity. I learned about collaboration from my time here and also that bigger [schools] aren’t necessarily better.”
Weinstein’s lecture was the fifth in a series, Science for the Common Good, in which Trinity alumni have returned to campus to discuss their careers. Weinstein began his talk with a few words of wisdom for the students: Take chances, don’t lose your idealism, follow your heart, and find something that you truly love, even if it means changing direction or career path.
Weinstein’s advice was based, in large measure, on his own experience. After graduating from Trinity, he attended Harvard Medical School, where he was determined to become an Alzheimer’s researcher. But he found that he was frustrated by the work and in 1991, he left the field of gerontology and reinvented himself as a pediatric endocrinologist at Children’s Hospital in Boston. After obtaining a master’s degree in clinical investigation from Harvard and MIT, Weinstein became director of the Glycogen Storage Disease (GSD) Program at the children’s hospital.
Today, Weinstein is a professor at the University of Florida, where he directs the Glycogen Storage Disease Program, following the largest cohort of hepatic GSD patients in the world.
GSD is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially the liver, kidneys, and small intestines, impairs their ability to function normally.
The symptoms, which typically appear in children at the age of 3 or 4 months, include the inability of babies to sleep through the night, seizures, thin arms and legs, short stature, an enlarged liver and kidneys, a protruding abdomen, diarrhea and deposits of cholesterol in the skin. Until the 1970s, the disorder was almost always fatal, said Weinstein. “There was no hope.”
But it was discovered that cornstarch therapy was a life-sustaining remedy for this rare disorder, which particularly afflicts Ashkenazi Jews, and the treatment was used until the late 1990s.
Weinstein said there was relatively little research being done because so few children were inflicted with GSD, but he decided that it was what he longed to do. “I wanted to be the champion. Somebody had to care about” these children, he said.
He said he had two dreams. The first was to create a research program to improve care, and the second was to make sure that no child suffered. Because GSD was considered a rare disease, there was virtually no research money available.
At the University of Florida, which is the largest clinical and research facility in the world for the study of hepatic glycogen storage diseases, Weinstein and his team of researchers began to use gene therapy to see if they could devise a cure.
At first, they experimented on mice and almost all of the symptoms disappeared. But, as Weinstein noted, “people are not mice.” So he and his team began using the therapy on dogs, which are clinically identical to people. Weinstein said the results of the therapy have been very encouraging, providing evidence that gene therapy does indeed work. His hope is to next apply the therapy to people, although he acknowledged that “gene therapy is not going to be available for everybody.”
Weinstein made special mention of Alyssa’s Angel Fund (AAF), which was founded by Gayle and Steve Temkin in honor of their daughter, Alyssa, who has GSD. The Temkins founded AAF to give people the ability to travel to the University of Florida to meet with Weinstein and receive the medical attention they need. The fund, which is affiliated with the Mandell Jewish Community Center in West Hartford, covers all expenses for families, including medical care, cornstarch dietary needs and supplies.
Weinstein said the fund has helped children in countries all over the world, among them Sri Lanka, Afghanistan, China, Israel, Australia, Brazil and the United Kingdom.
Weinstein ended his presentation on an upbeat note. Referring to Alyssa while looking at the students he said, “If a 6-year-old can change the world, you guys can change the world.”
The author of more than 50 articles and 25 textbook chapters on GSD, Weinstein won the Jan Albrecht Award from the American Association for the Study of Liver Diseases. He also received the George Sacher Award from the Gerontological Society of America; a United Nations humanitarian award for his efforts to help children from around the world; and the Order of the Smile, an international award given by children in Poland to adults for their love, care and aid of children.