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Gene therapy 'trains' immune system to destroy brain cancer cells and reverses behavioral deficits
Date:2/19/2008

LOS ANGELES (EMBARGOED UNTIL: 4:30 p.m. EST /February 19, 2008) A new gene therapy approach that attracts and trains immune system cells to destroy deadly brain cancer cells also provides long-term immunity, produces no significant adverse effects and -- in the process of destroying the tumor -- promotes the return of normal brain function and behavioral skills, according to a study conducted by researchers at Cedars-Sinai Medical Centers Board of Governors Gene Therapeutics Research Institute.

The study was conducted in a recently developed laboratory rat model of glioblastoma multiforme (GBM) that closely simulates outcomes in humans and supports the translation of this procedure to human clinical trials later this year. Results of the study are described in the Feb. 19 issue of Molecular Therapy, the journal of the American Society for Gene Therapy.

These findings appear to be a significant milestone in creating an effective treatment for glioblastoma multiforme, said Maria Castro, Ph.D., co-director of the Board of Governors Gene Therapeutic Research Institute and principal investigator of the study. This therapy significantly improved survival rate, induced long-lasting systemic anti-tumor immunity, and resolved the neuropathological abnormalities caused by the tumors, which has been a stumbling block to many promising treatments.

The article documents the gene therapys effectiveness in killing GBM cells and launching an effective and sustained systemic immune response. It also focuses on the safety of the therapy as well as its ability to restore normal brain function and reverse behavioral deficits caused by large tumors.

Humans with GBM often suffer behavioral abnormalities that affect concentration, memory and balance. In the animal studies, rats exhibited abnormal rotational movements. The research team found that as the tumors grew, they displaced and compressed nerve terminals and impulse-conducting axons. But
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Contact: Simi Singer
simi.singer@cshs.org
310-423-7798
Cedars-Sinai Medical Center
Source:Eurekalert

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