In a School of Medicine laboratory, skin cells obtained from a woman in the earliest stages of Alzheimer's disease are being cultivated in a test tube. The cells are being engineered to act as transport vehicles, carrying the gene that produces nerve growth factor (NGF), a naturally occurring protein that pro-motes nerve development.

When they are ready, the NGF-producing skin cells will be implanted into the woman's brain in a first-time effort to combat the devastating effects of Alzheimer's disease using gene therapy. This phase of the landmark study, directed by professor of neurosciences Mark Tuszynski, M.D., Ph.D., is designed to evaluate whether this procedure is safe and whether the genetically modifed implant will pump NGF directly into the surrounding brain tissue.

Once patient safety is assured, the study will move on to test whether the memory loss associated with Alzheimer's disease can be slowed, stopped, or even reversed by treating disease-damaged brain cells with NGF.

"Nerve growth factor is a very potent tool for reducing cell death and boosting function in atrophied or damaged nerve cells. We have proven this approach restores atrophied brain cells in aging monkeys," Tuszynski said. "Existing therapies for Alzheimer's disease aren't very good, so after we observed cell restoration in the primate brain, we felt it was important to devise a therapeutic approach for human patients."

This study is a dramatic example of translational research. When research results like Tuszynski's primate studies are heralded as potential medical breakthroughs, the excitement surrounding news of possible cures often overshadows the painstaking work that is necessary to develop practical human applications from laboratory and animal studies. Translating research findings into improvements in patient care involves developing the treatment technology, testing its safety, and finally measuring its effectiveness in carefully controlled studies.

UC San Diego's health sciences division, encompassing the academic programs and research laboratories of the School of Medicine, and the clinical excellence of the UCSD Healthcare system, is the ideal setting for this chain of events to take place. The ability of medical faculty to collaborate with UC San Diego campus colleagues and with the many private research institutes and biotechnology companies in close proximity to the campus further enhances and accelerates the pace of biomedical discovery. The academic as well as scientific value of collaboration is reflect-ed by the ranking of UC San Diego's graduate program in neurosciences, which has participants from several campus departments and neighboring institutions, as best in the nation by the National Research Council.

"The work of many laboratories has helped define the effects of NGF, and advances in molecular biology have given us the tools to practice gene therapy," Tuszynski said. ìI believe that this study is taking place at UC San Diego due to a serendipitous chain of events-UC San Diegoís early vision of promoting gene therapy as an important form of medicine, the expertise in Alzheimer's disease that has been established here, and the creation of an environment that encourages the bridging of science and clinical care. Having as colleagues physician-scientists who understand basic research and can help create and carry out a practical, clinical study is what makes this work possible."