UCSD to Speed Gene Findings to Human Trials : Medicine: The school's goal is to transfer research results to therapy for patients more swiftly. - Los Angeles Times
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UCSD to Speed Gene Findings to Human Trials : Medicine: The school’s goal is to transfer research results to therapy for patients more swiftly.

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UC San Diego’s School of Medicine, a leading center for research into human gene therapy, has created a program designed to speed the transfer of its most advanced research into human clinical trials.

“This is where we wanted to be 20 years ago when we began (gene therapy) research,” Medical School Dean John Alksne said Thursday. “We’re making rapid headway in research. . . . What we need to do (now) is transfer the technology to patients.”

Besides identifying which gene therapy research projects should enter human trials, the Program in Gene Therapy will develop guidelines covering how those trials will be conducted.

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And, it will start educating doctors and patients about the new world promised by researchers who are manipulating genes to uncover treatments for cancer and other life-threatening diseases.

“This is a very positive thing,” said Terry Gach, a vice president at the La Jolla Cancer Research Foundation. “The end result is that therapies will be taken to the bedside much quicker . . . and that’s to the advantage of the patient.”

UCSD’s program will include both laboratory researchers and clinical doctors “so the transfer from (research) bench to bedside can get under way,” said Fred Gage, a program co-director. “When we get these groups together, we can begin putting pieces together.”

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The program will help to create guidelines governing initial human clinical testing of gene therapies that might one day result in effective treatment of cancer, Alzheimer’s, Parkinson’s and other diseases.

Trials will “move forward cautiously and safely,” Alksne said. “The health of our patients is our primary concern.”

The technology-transfer program is an important development for UCSD because, although its researchers win grants to conduct research, they generally lack the capital needed to move basic research from the laboratory into clinical trials.

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Because of funding shortfalls, many universities have stuck to basic research and licensed promising leads to for-profit companies that conduct clinical trials to determine if therapies are safe and effective.

UCSD will use university funds, federal grants and private donations to cover the costs of setting up the program and conducting trials.

UCSD’s strategy “makes sense” because the university’s continued involvement with promising research leads should help to “speed potential treatments into clinical use,” said Michael J. Dunn, an executive at Sibia, a company that is owned partly by the Salk Institute for Biological Studies. Sibia develops diagnostic and therapeutic products that rely upon the Salk Institute’s research.

In the long term, the program also could increase the value of UCSD’s research, Dunn said. “With clinical trials, you’re adding value to your research,” Dunn said. “You’re convincing (for-profit) companies that there is something of more value there.”

Gage and co-director Stephen Wasserman will lead the program until a nationwide search for a director ends, Alksne said.

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