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Study identifies biomarker that safely monitors tumor response to new brain cancer treatment
Date:7/1/2009

LOS ANGELES (STRICTLY EMBARGOED UNTIL 12:01 A.M. EDT on JULY 1, 2009) A specific biomarker, a protein released by dying tumor cells, has been identified as an effective tool in an animal model to gauge the response to a novel gene therapy treatment for glioblastoma mulitforme. The finding, reported in the July 1 issue of Clinical Cancer Research, paves the way for a Phase 1 clinical trial expected to begin in late 2009.

The gene therapy is a two-pronged strategy devised by scientists at the Cedars-Sinai Board of Governors Gene Therapeutics Research Institute. It uses a genetically engineered, harmless virus to deliver a combination of proteins and a drug to kill tumor cells, which triggers an ongoing immune response against malignant brain tumors cells.

The Cedars-Sinai team led by Pedro R. Lowenstein, M.D., Ph.D., director of the Board of Governors Gene Therapeutics Research Institute, and Maria G. Castro, Ph.D., co-director of the Institute, developed this gene therapy strategy during 10 years of laboratory research.

"Using this therapy, we have shrunk and completely eliminated very large brain tumors in animals and have trained their immune systems to develop memory so that recurrent tumors are also destroyed," said Castro, principal investigator of the study. "The biomarker identified in this study will help us determine the effectiveness of the therapy in patients with glioblastoma multiforme."

In this study, the researchers identified the most effective and least toxic combination of therapeutic agents that would offer the best results. They found that a protein released by dying tumor cells may be used as a "biomarker" to gauge the effectiveness of treatment. This protein, called "high mobility group box 1" (HMGB1), regulates gene expression in healthy cells by binding to the cells' DNA. When cancerous cells are killed, however, HMGB1 is released into the general blood circulation. This research shows t
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Contact: Simi Singer
simi.singer@cshs.org
310-423-7798
Cedars-Sinai Medical Center
Source:Eurekalert

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