Major pediatrics research award goes to UF gene therapy pioneer Flotte

University of Florida pediatrics chairman and gene therapy expert Dr. Terence R. Flotte, is being recognized for his research efforts with one of the most prestigious awards in pediatrics.

The Society for Pediatric Research has named Flotte, a UF Nemours eminent scholar professor of pediatrics and a professor of molecular genetics and microbiology, a 2005 recipient of the prestigious E. Mead Johnson Award for Research in Pediatrics.

He will receive the award at the Pediatric Academic Societies’ annual meeting in Washington, D.C., May 16.

The Johnson award is one of the most competitive and prestigious academic honors, said Dr. Douglas Barrett, UF senior vice president for health affairs and immediate past chairman of the UF pediatrics department.

“It’s an outstanding recognition of the impact of Dr. Flotte’s work by the nation’s academic pediatric community,” said Barrett, who also is a professor of pediatrics. “He has made major contributions to the fields of genetics, gene therapy and childhood lung diseases such as cystic fibrosis. We are fortunate indeed to have Dr. Flotte as a leader within UF’s College of Medicine.”

Flotte is perhaps best known for his pioneering work on gene therapies for cystic fibrosis and alpha-1 antitrypsin deficiency, which were the first to use the apparently harmless adeno-associated virus, also known as AAV, as a vehicle to transport missing genes to cells.

Cystic fibrosis, which affects about 30,000 U.S. children and adults, is a lung disorder in which bronchial tubes produce excessive amounts of thick mucus, interfering with respiration. Patients with alpha-1 antitrypsin deficiency lack a lung-protective protein normally made in the liver and can suffer serious lung infections. One of the most underdiagnosed conditions, it strikes about 100,000 Americans.

Clinical trials currently are under way on gene therapy treatments for both conditions. Ongoing efforts are pushing AAV-based gene therapies for other genetic defects and for diabetes closer to clinical application, Flotte said.

“To me, this award is a recognition of our team’s efforts to bring the power of molecular biology directly to bear on the health and well-being of children and adults with genetic diseases,” Flotte said.

Presented annually since 1939, the Johnson award recognizes individuals whose recent work has contributed significantly to the overall field of pediatrics, according to the Society for Pediatric Research. The award is presented to two researchers each year.

Flotte joined the UF College of Medicine faculty in July 1996 as an assistant professor of pediatrics and molecular genetics and microbiology. Promoted to associate professor in August 1998 and professor in July 2001, he has served as pediatrics department chairman since July 2002.

“He’s had remarkable success at both bench and bedside,” said diabetes expert Dr. Desmond Schatz, a UF professor of pediatrics. “Dr. Flotte has brought national and international recognition to the gene therapy program and the department of pediatrics at the University of Florida.”

Flotte is an assistant program director for the UF General Clinical Research Center and is affiliated with UF’s Evelyn F. and William L. McKnight Brain Institute, and previously directed both the UF Genetics Institute and the UF Powell Gene Therapy Center.

Since arriving at UF, Flotte has secured research grants totaling more than $11.5 million in funding. He holds four gene therapy-related patents, and during his career has published about 100 research studies and 11 book chapters.

“The work which is being recognized (by the Johnson award) would not have been possible without the ongoing support of a number of key leaders and groups within our UF community,” Flotte said. “Foremost among those have been the past and present Powell Gene Therapy Center Directors Drs. Nick Muzyczka and Barry Byrne, the UF Genetics Institute Director Dr. Kenneth Berns, our dean, Dr. Craig Tisher, and Senior Vice President for Health Affairs Dr. Douglas Barrett.

“I have also had numerous other mentors and collaborators who have made our success possible, including Drs. Barrie Carter, Bill Guggino, Mark Atkinson, Bill Hauswirth, Richard Snyder, Mark Brantly and many others,” Flotte added. “Also, I have to recognize the tremendous efforts of the graduate students, technicians, postdoctoral fellows and research nurses in my group who have performed most of this work.”

Flotte is the second UF pediatrician to receive the award. The first was Dr. Richard T. Smith, the College of Medicine’s founding chairman of pediatrics, who received it in 1963. Smith was honored for his research in pediatric immunity, notably his demonstration that newborn babies are able to produce disease-fighting antibodies.