UCSD Prepares for Big Step Into Gene Therapy : Medicine: School prepares to apply gene transplant research at the bedside when its new hospital opens in 1993.

UC San Diego’s medical school is organizing a human gene therapy program, to move its gene transplant research from the laboratory to the hospital bed by the time the university’s new La Jolla hospital opens in 1993.

A search committee is seeking a director for the human genetics program, who could be hired within the next few months, said Dr. Gerard Burrow, medical school dean.

In addition, a leading UCSD gene therapy researcher and advocate, Dr. Theodore Friedman, is putting together a proposal for Burrow on how the gene therapy program should be organized.

“We have about 40 people on the campus who are interested in different aspects of human genetics. It just hasn’t been put together in a program,” Burrow said Monday.

Although human genetics programs have been established at other universities, they have emphasized the basic research that needs to be done before gene therapy can actually be tried in patients.

UCSD’s planned program is unusual because, with the university’s basic research in the field already well-established, it instead emphasizes turning gene therapy ideas into actual experiments in humans.

“The goal of course is to bring (gene therapy) to the bedside, and to do that as expeditiously and well as we can. And in order to do that we need more than a collection of individuals,” Friedman said.

Because of rapidly accumulating knowledge about the genetic basis for human disease, gene therapy has been advocated for conditions ranging from cystic fibrosis to atherosclerosis and cancer. Working with Friedman, neuroscientist Fred Gage also is studying ways to use gene therapy against Alzheimer’s and Parkinson’s diseases.

UCSD’s plan is the most ambitious in a San Diego field that is expected to include non-academic efforts as well.

A La Jolla biotechnology firm, Viagene, hopes to use gene therapy techniques to treat AIDS patients next year. And the newly established San Diego Regional Cancer Center, led by former UCSD researcher Dr. Ivor Royston, plans to try gene therapy against cancer.

The only clinical gene therapy experiments so far in the United States have been developed since August of this year, by doctors at the National Institutes of Health.

In one, doctors are genetically altering skin cancer patients’ own immune system cells to try to stimulate them to more effectively battle the cancer. In the other, children with crippled immune systems are being provided with cells that can produce an essential immune protein they are lacking.

These experiments have followed a decade in which human gene therapy has moved very slowly despite early, glowing forecasts of its promise.

The scientists who remain skeptical about how soon gene therapy may bear clinical results include Paul Berg, director of the Center for Molecular and Genetic Medicine at Stanford University. Berg said centers such as UCSD’s are inevitable, however, because the ideas raised by the genetic revolution need to be tested.

Friedman, widely recognized as a leader among the handful of U.S. researchers racing towards gene therapy, sees the new unit an administrative structure to support research.

“Its function will be informational and sort of catalytic--putting an administrative face on much of the work that’s already going on here,” Friedman said.

He noted that a number of organizational questions will need to be answered before the UCSD human genetics program can begin. Among them:

* How will the Center for Molecular Genetics, in which Friedmann and other molecular biologists hold their academic appointments, interact with the new program?

* Will researchers at the Salk Institute and Scripps Research Institute be included in the efforts?

* Since cancer is a key target of gene therapy techniques, will the program require a rethinking of UCSD’s cancer programs?

Friedman said he hopes to get a proposal for the genetics program’s organization to Burrow within the next few days.

Inder Verma, a noted Salk Institute molecular biologist, said he doesn’t expect UCSD’s planned program to produce quick results in treating human disease.

“Unless they have something up their sleeve that I don’t know about, I don’t think it will produce anything imminent,” Verma said. “But I think it will focus things. The biggest advantage I can see is that it might attract new people to come and join this group.”