Partnership helps pets and people
By Sarah Carey
A five-year collaboration between the Powell Gene Therapy Center, the UF College of Medicine department of pediatrics and the UF College of Veterinary Medicine has strengthened professional ties between the colleges and led to research that may improve both animal and human health.
“We have worked together on projects that use gene therapy as a possible means for treating congestive heart failure and glycogen storage disease, or GSD, in people, as well as two types of cardiac problems experienced in dogs,” said Thomas Conlon, Ph.D., an assistant professor in the department of pediatrics and director of the Powell Gene Therapy Center’s Toxicology Core. His team performs preclinical studies on disease models, many of which have led to successful drug trials in people.
“We have a lot of collaborations with other researchers, but in this case there is a real dedication to the idea of making sure that both sides benefit from the collaboration,” said Andrew Specht, D.V.M., an assistant professor of small animal medicine at UF who has worked with a research colony of dogs used as a model in GSD studies. “Dr. Conlon’s idea of collaboration is that we should constantly be talking with each other, not only about the current project, but also about other potential new ideas. He has embraced that concept.”
Specht has co-authored two publications relating to glycogen storage disease with Conlon and David Weinstein, M.D., M.P.H., the principal investigator on the study and an expert on GSD, a rare disease that prevents the body from properly storing and using sugar. About one in 100,000 children have the disease. In 2009, College of Medicine and College of Veterinary Medicine researchers reported breakthrough gene therapy findings in a dog born with the disease.
“This particular form of metabolic disease is a natural mutation, but you’d never see it clinically (in dogs),” Specht said. “Dogs that get it naturally die shortly after birth or even before birth, so almost none would ever make it to the vet. The only reason ours survive is that we know they will have it, so we can be prepared to care for them.”
Conlon also has worked with Amara Estrada, D.V.M., an associate professor in the department of small animal clinical sciences and chief of the cardiology service, on two studies, one relating to heart attack repair using pigs as an animal model. The other is an ongoing study involving the use of adipose-derived mesenchymal stem cells — donated by other dogs and enhanced with gene therapy — to treat dilated cardiomyopathy, or DCM, in Dobermans at the UF Small Animal Hospital.
“Our basic research relates to population-specific diseases,” Conlon said. “GSD is a human disease, whereas DCM-related therapies are geared toward helping dogs. In these studies, which are in what we call the proof-of-concept stage, we are still trying to determine the best therapeutic plan, but the knowledge gained will eventually help the human and veterinary communities.”
Conlon and colleagues in the College of Veterinary Medicine hope to collaborate on hemophilia and glaucoma studies, as well.