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Gene Therapy Eradicates Pancreatic Cancer in Preclinical Trial,Targeting System's Success in Mouse Model Reported in Cancer Cell

HOUSTON, July 9, 2007 - A molecularly engineered therapy selectively embeds a gene in pancreatic cancer that shrinks or eradicates tumors, inhibits metastasis, and prolongs survival with virtually no toxicity, researchers from The University of Texas M. D. Anderson Cancer Center report in the July 9 edition of Cancer Cell.

"This vehicle, or vector, is so targeted and robust in its cancer-specific expression that it can be used for therapy and perhaps for imaging," notes senior author Mien-Chie Hung, Ph.D., professor and chair of M. D. Anderson's Department of Molecular and Cellular Oncology.

The researchers call the system a versatile expression vector - nicknamed VISA. It includes a targeting agent, also called a promoter, two components that boost gene expression in the target tissue, and a payload - in this case a gene known to kill cancer cells. It's all packaged in a fatty ball called a liposome and delivered intravenously.

Researchers are working with M. D. Anderson clinicians to move the system, developed and tested in mouse models of pancreatic cancer, to a Phase I clinical trial.

"This looks like a promising approach to gene therapy for pancreatic cancer and we are working to bring it to a clinical trial," says James Abbruzzese, M.D., professor and chair of the M. D. Anderson Department of Gastrointestinal Oncology.

He estimates that it will take between one and two years to complete U.S. Food and Drug Administration requirements for a Phase I trial. Abbruzzese's clinicians are working with Hung's group to compile preclinical information for FDA review.

About 37,000 cases of pancreatic cancer are diagnosed annually in the United States. Early diagnosis is extremely difficult, so the disease is often discovered at a late stage after it already has spread, or metastasized. Fewer than 4 percent of pancreatic cancer patients survive five years after diagnosis, one
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