David Weinstein '90, M.D., M.M.

 DEGREE: B.S. in biology; M.D., Harvard University; M.M.Sc., Harvard University and Massachusetts Institute of Technology 
JOB TITLE: Professor of endocrinology, Director of the Glycogen Storage Disease Program, and Associate Program Director for Research, University of Florida College of Medicine; recent recipient of the Order of the Smile award.

REPORTER: What is glycogen storage disease? 
WEINSTEIN: Normally when we eat a meal our bodies store sugar and use it during times of fasting. For children affected by glycogen storage disease, sugar is stored but cannot  get out. Any time they do not eat, their blood sugar falls dangerously low. The disease is genetic and is relatively rare—it affects approximately one in 100,000 children worldwide. When I first started working with GSD, patients were not expected to live past childhood without a liver transplant. Administration of regular doses of cornstarch, which metabolizes slower than other sugars, can help to treat symptoms of GSD. It has gotten to the point now that, with proper care, patents are expected to do well. Still in many places outside of the U.S. the prognosis is very poor.  

REPORTER: What drove you to study GSD? 
WEINSTEIN: I first heard of it through Trinity Professor Emeritus of Biology John Simmons,who referenced GSD when he taught courses in endocrinology. I was medically exposed to it between 1994 and 1998 when I was working at Children’s Hospital Boston. In 1998, I was invited to a national conference on the subject, and I was shocked by the state of care for people with this disorder. There had not been any clinical advances for 16 years because there was just was no financial support or glamor in studying it. As a result of the lack of research and understanding, many children were suffering or even dying. At that conference, I decided that these children needed an advocate, and I have been dedicating myself to this condition for the past 14 years. 

REPORTER: How did your program at the University of Florida develop?
WEINSTEIN: My ultimate goal is to cure glycogen storage disease, and we are working on gene therapy. Before we can try this in humans, testing in animals with the disease must  occur. I was interested in pursuing gene therapy on dogs that naturally have glycogen storage disease. In order to do this, I needed to find an institution that also had a veterinary school, since the support did not exist in Boston. In 2005 I moved from Boston to the University of Florida, where I had the opportunity to create a research program from scratch. The move to Florida allowed me to try to create the Disney World of health care for children and adults with GSD. We have built the largest clinical and research program for GSD, and the program follows children and adults from 36 counties. I also wanted to create a charitable arm for the program to make sure that every child with this disease has access to quality medical care. This became the Alyssa’s Angel Fund, named for one of my patients who lives in Hartford and whose family helped establish the charity. We receive a great deal of support from the Hartford community, particularly the Mandell Jewish Community Center of Greater Hartford. Through this generosity, we have been able to care for children on nearly every continent, and no one gets turned away. 

REPORTER: What was the path that led you to a career in medical research? 
WEINSTEIN: I was interested in medicine when I was young, and at the age of 12 I became a routine volunteer for a group in Miami that was studying pulmonary medicine. I asked a lot of questions, and the researchers liked my suggestions. I was offered a job and became published at the age of 14. By the time I entered Trinity, I had developed an interest in studying Alzheimer’s disease. I eventually went to study at Harvard University, where I decided that I could do more by studying pediatrics. I really believe that the success I had is due to my experience at Trinity.

REPORTER: What did you find outstanding about the Trinity experience? 
WEINSTEIN: One of the reasons I chose Trinity was the small environment, which afforded a lot of opportunities. I started doing research in the first week of my freshman year in Professor Simmons’s lab, and I continued to do so during my entire undergraduate career. As a senior I received funding from the office of then-President Tom Gerety, which gave me a budget and lab space to conduct independent research. The work that resulted was recognized by the Gerontological Society of America with an award for outstanding research in the field of gerontology by an undergraduate or graduate student. The work was also recognized with a Barry Goldwater Scholarship, and the success allowed me to get lab space at Massachusetts General Hospital when I moved to Boston. None of that would have been possible if I didn’t receive the support and encouragement at Trinity. 

REPORTER: What is the Order of the Smile award and how were you nominated? 
WEINSTEIN: It is an international humanitarian award given by children to adults in recognition of their service to children. Prior winners include Pope John Paul II, Mother Teresa, and Nelson Mandela. I will be traveling to Poland to receive the award in the summer of 2013. I was nominated due to the efforts of a girl who lives in Poland that I treated. She went on to medical school, where she realized how ill she had been and how far she had come. She wanted to do something to thank me for helping her. She collected letters from over 250 people from 30 countries—all patients who had been helped by our services—and submitted the letters to the award coordinators. 
REPORTER: How does it feel to be recognized in this way by your patients? 
WEINSTEIN: It means a lot that my patients would go out of their way to do this. It is special to be recognized for clinical work. People praise research all the time, but very few organizations recognize people for taking care of other people. I am extremely honored. To learn more about David’s research program and the Alyssa’s Angel Fund, visit www.Glycogenstoragedisease.com.​​