Gene therapy takes off

April 11, 2000

Judith Zwolak

Ending years of searching for the right person to head its new Center for Gene Therapy, Tulane has hired Darwin Prockop, a renowned researcher in the field. Prockop, director of gene therapy at the MCP Hahnemann University School of Medicine in Philadelphia, also will bring as many as a dozen key members of his research team to Tulane when he starts this summer.

"We have searched for some time and Dr. Prockop was identified as an ideal candidate," says Paul Whelton, senior vice president for the health sciences. "We worked very hard to recruit him to Tulane and we're very pleased we were able to do it."

In its initial phase, the center will focus primarily on laboratory research in gene therapya treatment that replaces absent or faulty genes with working ones.

"At this stage it will be a research and development center for what we hope will be clinical applications eventually," Whelton says.

Of the $15 million Tulane plans to invest in the center, $8 million will come from Columbia/HCA Corp., the majority owner of Tulane's hospital and clinic. "It's a lot of money and it's money for which Columbia has no direct expectation of seeing a return," Whelton says. "We're very appreciative of their investment."

Prockop, who will also bring a number of federal research grants with him to Tulane, says the commitment from Columbia/HCA and the university to invest in gene therapy attracted him here.

"Research is a delicate flower," Prockop says. "It needs funding but it also needs emotional and intellectual commitment."

Another key asset to gene-therapy research, he says, is the Tulane Regional Primate Research Center, where researchers can test new therapies on primates before performing human clinical trials. Prockop is currently involved in research trials on children who have osteogenesis imperfecta, a disorder that causes extremely brittle bones.

Prockop discovered the genetic defect that causes the disorder, and researchers at St. Jude's Children's Research Hospital in Memphis are using gene therapy to treat the disease in five children. In this study, the technique involves using stem cells to introduce good genes into a cell with defective genes. Stem cells are "blank" cells that reside in bone marrow and have the ability to divide without limit and give rise to specialized tissue cells.

Previously, researchers have focused on using partially inactivated viruses to invade a defective cell and release the new gene. Stem cells offer a safer way of delivering therapeutic genes because researchers can use the patient's own cells and genetically alter them without infecting the patient with a virus, he adds.

"If you use non-viral techniques, you have to grow cells rapidly and increase the numbers quickly," Prockop says.
Growing great numbers of stem cells rapidly was a challenge for researchers until Prockop discovered a new way to culture the cells. By placing only 1,000 stem cells together in a petri dish, Prockop and his research team found the cells grew 100 times faster than in the one-million-cell cultures commonly used by researchers.

Prockop says his discovery will help his team grow stem cells for eventual gene-therapy treatments for other severe bone diseases as well as other genetic disease such as Parkinson's Disease and Alzheimer's Disease. Although the possibilities for these treatments are exciting, he says, the process of developing therapies must proceed slowly and with caution.

"Moving to real therapy is a very expensive process and you can't avoid the costs," he says. For his part, Whelton says he envisions the gene-therapy center as an integral part of Tulane's research, education and clinical activities. The center faculty will interact not only with other basic-science faculty, he says, but also with engineering researchers on the uptown campus and faculty members and students in the School of Public Health and Tropical Medicine.

"Certainly I'd like to have Dr. Prockop involved in health administration in our school of public health," Whelton says. "We need to think about how we apply gene therapy to biotechnology applications. How do we train the managers to manage biotech companies?"

Creating companies that develop and market gene-therapy treatments is a primary goal of the Louisiana Genetics Research Consortium, a statewide partnership set up last year between academic, governmental and business entities. Whelton, who is president of the consortium, says applications resulting from the research at Tulane's new gene-therapy center should help the local and state economies.

"We see this as an important investment that hopefully will improve the health of the community we serve and be an important economic contributor to the metropolitan area and the state," he says.

Prockop and his laboratory will eventually reside on the sixth floor of the J. Bennett Johnston Building on the downtown campus. While the new space should be ready by early fall, Whelton says he is currently looking for temporary space to house Prockop and his staff during the summer. Prockop, who received his medical degree from the University of Pennsylvania and his doctorate in biochemistry from George Washington University, is a member of the National Academy of Sciences and the National Institute of Medicine.

Tulane University, New Orleans, LA 70118 504-865-5000