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Northwestern researchers find way to turn off deadly brain cancer gene in mice

Chad Mirk |  Sun-Times file photo

Chad Mirkin | Sun-Times file photo

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Updated: November 1, 2013 10:08AM



Researchers at Northwestern University say they’ve found a way in mice to turn off a key gene linked to a type of brain cancer that claimed the lives of U.S. Sen. Edward Kennedy, Chicago sportscaster Tim Weigel and movie critic Gene Siskel.

They used nanotechnology to deliver a drug to the brain that turned off a cancer-causing gene involved in the development of glioblastoma multiforme, an incurable disease that kills about 13,000 people nationwide each year, typically within 14 to 16 months of diagnosis. It’s one of the deadliest cancers.

The Northwestern team, led by Chad Mirkin, a leading researcher on nanotechnology, and glioblastoma expert Alexander Stegh, said they were able to breech the big hurdle to such treatment — finding a way past the blood-brain barrier that’s the body’s defense to protect the brain.

The findings are preliminary, involving only mice, not humans. The mice were injected with the drug intravenously. The survival rate for the mice with glioblastoma rose by nearly 20 percent, and the size of their tumors was greatly reduced, the Northwestern team reported this week in the journal Science Translational Medicine.

More work is needed to see whether the treatment will work and improve survival rates in people. But Mirkin and Stegh are hopeful.

“This is a beautiful marriage of a new technology with the genes of a terrible disease,” said Mirkin. “Using highly adaptable spherical nucleic acids, we specifically targeted a gene associated with GBM and turned it off in vivo. This proof-of-concept further establishes a broad platform for treating a wide range of diseases, from lung and colon cancers to rheumatoid arthritis and psoriasis.”

Stegh added: “Glioblastoma is a very challenging cancer, and most chemo-therapeutic drugs fail in the clinic. The beauty of the gene we silenced in this study is that it plays many different roles in therapy resistance. Taking the gene out of the picture should allow conventional therapies to be more effective.”



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