UF Receives Private Gift To Establish Premier Research Site, Professorship For Underdiagnosed Lung-liver Disease

August 27, 1998

GAINESVILLE—Fueled by a generous gift from the Miami-based Alpha One Foundation, the University of Florida Gene Therapy Center is slated to become the premier research site for an underdiagnosed, potentially fatal lung-liver disease.

The grant is a magnet for additional funds that will enable UF to develop what is thought to be the first academic research endeavor of its kind devoted exclusively to the study of alpha-1-antitrypsin deficiency, which causes early emphysema and severe liver disease. The disease, considered the second most-common genetic disorder among Caucasians, affects an estimated 100,000 Americans. One in 50 carries the gene for it.

Capping off the unique program is the foundation’s establishment of a UF College of Medicine professorship that will be filled this fall by noted National Institutes of Health scientist Dr. Mark L. Brantly.

The Alpha One Foundation is providing a $600,000 endowment for the professorship — to be augmented by $420,000 in state matching funds — and a $200,000 supplement for the research program. The college and other funding sources will add to those monies with up to $1.3 million over the next three years. In addition, Brantly will bring with him another $200,000 in support from the pharmaceutical industry, as well as up to three members of his current research team.

The research program will feature a basic science laboratory focused on the disorder’s molecular mechanisms; a clinical research unit directed toward developing new therapies for alpha-1-antitrypsin deficiency, including gene therapy; and a diagnostic laboratory for the identification of individuals with the disorder. An added plus: a tissue bank will stock lung and liver tissue samples as well as plasma and serum from individuals with the disease.

“The UF College of Medicine is an ideal setting for an Alpha One Research Program since it has national leaders in gene therapy research, world-renowned clinical pulmonologists and hepatologists, experts in solid organ transplants and an NIH-sponsored Gene Therapy Center,” said John Walsh, the Alpha One Foundation’s chief executive officer. “The major impediment to a coordinated research effort on alpha-1-antitrypsin deficiency has been the lack of a dedicated research program at an academic university with excellence in medical research.”