Scientist’s encouragement enhances CVM’s collaborations with Powell Gene Therapy Center

Dr. Amara Estrada, Dr. Brandon Pogue and Dr. Thomas Conlon are shown outside of the UF Small Animal Hospital with two Dobermans that were part of a collaborative stem cell study.

 By Sarah Carey

A five-year collaboration between the Powell Gene Therapy Center, the University of Florida College of Medicine’s 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, 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.

UF veterinary researchers say Conlon’s interest in translational possibilities, as well as in providing unique educational possibilities to veterinary students, has positioned him as a respected colleague and peer whose connections and expertise are extremely valuable to the college.

“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 Dr. Andrew Specht, 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 coauthored two publications relating to GSD with Conlon and Dr. David Weinstein,  a principal investigator on the study who treats people with GSDs. Specht said he became interested in studying the disease because it was an opportunity to help people, but that the work was also rewarding because of the opportunity to help improve the lives of the affected dogs.

In addition, Specht hopes that some of what is learned from treating these dogs might also help dogs with other metabolic or genetic diseases.

“This particular form of metabolic disease is a natural mutation, but you’d never see it clinically,” 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.”

He added that dogs that have the disease remain at UF and are treated for it, and those that do not have it are made available for adoption, frequently to families with children who have the disease.

“These kids then get to see a dog with the same disease that they have, so it’s kind of a fun part of the project,” Specht said.

Added Dr. Erin Runcan,  a resident in the UF Veterinary Hospital’s large animal theriogenology service, “This project has given our students a wonderful opportunity for hands-on learning experiences.”

She said students are involved with every step of the process, from breeding management to C-section planning and neonatal resuscitation.

“The students are very excited to be involved with these unique cases,” Runcan said.

Brooke Minton, a senior UF veterinary student, has worked in with the GSD colony of dogs for the past three years.

“The project has been a very positive experience from the start,” she said. “I applied for the job looking for hands-on experience in the veterinary field. Over the last three years, I have gained so much from this opportunity.”

Minton added that she had improved her clinical skills by working firsthand with clinically ill dogs and puppies, while simultaneously getting an inside look at the research side of veterinary medicine.

“I couldn’t have asked for a more rewarding job,” she said.

Conlon also has worked with Dr. Amara Estrada, associate professor in the department of small animal clinical sciences and chief of the cardiology service, on two studies, one relating to myocardial infarction repair using pigs as an animal model, and an ongoing study involving the use of adipose derived mesenchymal stem cells cells donated by other dogs and enhanced with gene therapy — to treat dilated cardiomyopathy, or DCM, in Dobermans.

“We grow the stem cells, which takes about two weeks for each patient,” Conlon said. “The morning of the injection, we prepare the cells and deliver the gene therapy to those cells using an adeno associated virus. The virus infects the cells, which, when combined with the animal’s other cells, causes the gene we put in to be overexpressed.”

Conlon, or a member of his research lab, personally delivers the stem cells to Estrada’s team whenever a Doberman in the DCM study is ready to receive them at the UF Small Animal Hospital and during a procedure which takes place in the catheterization laboratory.

“Our basic research, the DCM and GSD work, relates to population-specific diseases,” Conlon said. “GSD is strictly a human disease, whereas DCM related therapies are geared towards 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.”

Once a drug has been tentatively approved at the federal level for use in people, a toxicology study must be performed, he said, adding that both the AAV myoblast and congestive heart failure studies, which used adeno virus as a gene therapy vehicle, were formal toxicology studies that were reviewed by the FDA to determine if they were safe to use in people.

“Until we pick one therapy that is the best, a study is still in that proof-of-concept stage while we try to prove that we can help,” Conlon said.

He added that potential future gene therapy collaborations with the CVM, including studies that relate to hemophilia and glaucoma could benefit both animals and people, are on the horizon as well.

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March 2011

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Small animal surgery residents excel at recent Veterinary Orthopedic Society meeting in Snowmass, Colo.

Researcher’s work at UF CVM strengthens interdisciplinary ties

Thanks to support from a leading College of Medicine scientist, UF CVM’s collaborations with the Powell Gene Therapy Center have thrived in recent years.

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