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

eriments showed gene activity was limited to pancreatic cancer cells and was minimal or absent from other cancers and three lines of normal pancreatic, lung and breast cells.

The next step was to load the vector with a weapon. The team packaged its mutant version of the Bik gene along with C-VISA in a liposome. For comparison, the liposomal gene also was attached to the CMV vector.

They tested both systems in the lab against all 13 available human cell lines of pancreatic cancer available worldwide. BikDD killed cells in all lines, with the VISA-delivered gene destroying more cells in all but one case. The C-VISA system spared healthy tissue while CMV-BikDD destroyed healthy cells. Subsequent tests compared the two methods in the mouse models.

Hung's team continues research on gene expression vehicle. "VISA is versatile enough that if you change the promoter, you could target other cancers or even other diseases," Hung says.

Research was funded by Abbruzzese's pancreatic cancer SPORE grant from the NCI, M. D. Anderson's NCI center support grant, a grant from the Topfer Research Funds, and an AstraZeneca Fellowship.

M. D. Anderson has filed for a patent on VISA-BikDD, which has been licensed to Alchemgen Therapeutics Inc. (ATI). ATI partially supports Hung's research. Hung and M. D. Anderson own stock in the company. These arrangements are managed by M. D. Anderson in accordance with its conflict of interest policies.

Co-authors of the Cancer Cell paper with Hung, Abbruzzese and Xie are: Weiya Xia, Hsu-Ping Kuo, Yuanfang Liu, Zheng Li, Qingqing Ding, Su Zhang, Bill Spohn, Yan Yang, Yongkun Wei, and Jing-Yu Lang, all of the Department of Molecular and Cellular Oncology; and Zhongkui Li, Douglas B. Evans, and Paul J. Chiao, all of the Department of Surgical Oncology. Kuo and Hung also are affiliated with the Graduate School of Biomedical Sciences, operated jointly by M. D. Anderson and The University of Texas Health Science Cen
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