Cancer gene therapy news backgrounder: New ideas fuel next generation gene therapy research

us is disabled, but is considered a short-term therapy that must be constantly repeated.

Adenovirus-based therapies, which are the most commonly studied cancer gene therapy vehicles, work well when they are injected directly into a tumor. But because they are by their nature an infectious agent, these vectors can produce systemic immune reactions in patients when delivered throughout the body by intravenous injection.

It was a fatal immune reaction to an experimental adenovirus-based therapy that abruptly halted gene therapy research nationwide in 1999, when 18-year-old patient Jesse Gelsinger died while participating in a University of Pennsylvania clinical trial. The study wasn't about cancer, but was designed to treat a rare metabolic disorder. Another experiment in France using a virus vector succeeded beautifully in curing young patients of a critical immune deficiency disease, but also caused leukemia in two patients. The gene that was delivered settled itself next to an oncogene, and turned it on.

After each incident, the federal government shut down related gene therapy clinical trials for a period of time and has increased oversight of all clinical trials. While there have been no similar serious reactions to adenovirus-based cancer gene therapies, the major hurdle any such therapy faces is treating cancer that has spread. "The obstacle in dealing with malignancy is facing the dissemination of disease - its spread," says Gary Clayman, M.D., another gene therapy researcher at M. D. Anderson. "Therefore, a clear barrier to gene therapy is addressing that spread in an effective way."

Fat bubbles

Bubbles of fat may offer an answer. The newest strategy to emerge out of Roth's lab is a blob of lipid, a type of fat that holds therapeutic genes. Developed by Nancy Templeton, Ph.D., of Baylor College of Medicine, the special "liposome" is of a size that is easily absorbed into cells. "Dr. Templeton hit upon a lipo

Contact: Nancy Jensen
University of Texas M. D. Anderson Cancer Center

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