Football player turned philanthropist Jim Kelly was at Tulane University Medical Center July 23 to announce a $199,000 grant to the Tulane Regional Primate Research Center from Hunter's Hope Foundation. The grant will fund the study of gene therapy as a treatment for Krabbe's disease -- a rare, degenerative, inherited condition that is usually fatal by age two -- and other leukodystrophies and brain diseases. About 40 babies are born with Krabbe's disease each year in the United States.
At the news conference, Kelly -- the retired quarterback of the Buffalo Bills with four Super Bowls to his credit -- spoke to the press and medical community as a father and as a funder. Kelly's son, Hunter James, in whose name the foundation was established, was born with the disease in 1997 on Valentine's day, a birth date he shares with his father. With a large portrait of his son as a backdrop, Kelly described Hunter as "the real fighter in the family."
"Many families like ours were told that a cure was not available for their children," Kelly said. "Hunter's Hope gives children and families affected by Krabbe's disease hope for the future."
Tulane was one of the first recipients of grants made by the foundation; a total of $830,575 was awarded to eight universities across the United States and Australia. "People from around the country have been extremely generous and supportive in helping the Hunter's Hope Foundation raise more than $2.5 million in less than two years," said Jill Kelly, Jim's wife and chief executive officer of the foundation, who concurrently announced recipients in Buffalo, N.Y.
The grant is the first funded project of the new Louisiana gene therapy research initiative -- a partnership between Tulane and Louisiana State University (LSU) -- as well as the first gene therapy project to receive outside funding to an investigator at the primate center. According to Director Peter J. Gerone, ScD, the primate center is ideally positioned for conducting this research ‹ with the only breeding group of monkeys that carry the gene that causes Krabbe's disease; a talented team of investigators; and a new building for gene therapy research. "The unique resources available at the primate center offer an unprecedented opportunity to develop genetic therapy for Krabbe's disease and other leukodystrophies," he said.
Gary B. Baskin, DVM, principal investigator of the project, agrees. "We have an excellent model for Krabbe's disease in the rhesus monkey," he says. Krabbe's disease, destroys the myelin sheath that protects nerve fibers, explains Baskin, who likens it to "electrical wires without the protective rubber coating."
"Since Krabbe's disease is caused by a defective gene, the ideal treatment would be to give the patient a gene that works properly," continues Baskin. "We can use our colony of monkeys that carry the genetic defect that causes Krabbe's disease to study whether gene therapy may cure Krabbe's disease in humans."
Aiding this effort is a three-year, $1.6 million grant from the National Institutes of Health's National Center for Research Resources to support and increase the population of monkeys used for this research. "This grant will allow us to use modern, assisted reproductive technology to ensure that there will be an adequate supply of monkeys showing this genetic defect," says Baskin, principal investigator.
Working with Baskin are co-investigators Krishna J. Fisher, PhD, director of research at Tulane's Center for Gene Therapy; Jay Kolls, MD, director of LSU's gene therapy program; and Billie Davison, DVM, research veterinarian at the primate center -- a group Gerone calls "the team necessary to do the job."