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Biotech, Gene Therapy, Parkinson's Disease

Ceregene Awaits Parkinson’s Trial Results, In a Key Test for Gene Therapy

Luke Timmerman 10/31/08
http://www.xconomy.com/san-diego/200...-gene-therapy/

A critical turning point is coming for Ceregene by the end of the year. The San Diego-based biotech company expects to get results in the next couple months on whether its experimental gene therapy can effectively help treat Parkinson’s disease.

I got the overview of what this is all about a few weeks ago during a visit to the lab of Mark Tuszynski, a neuroscience professor at the University of California, San Diego, and Ceregene’s founder. He’s awaiting results of a trial that will have big implications for the much-maligned field of gene therapy, and for patients with Parkinson’s, who haven’t had a new advance to cheer about in decades. This could also be important news for Cambridge, MA-based Genzyme (NASDAQ: GENZ), which signed a partnership last year with Ceregene to get rights to this drug outside of North America.

Parkinson’s, a degenerative disease of the central nervous system, robs patients of their ability to control movement and speech. It is usually treated with generic L-dopa, which helps replenish the brain’s diminishing supply of dopamine, according to the National Institutes of Health. About 1.5 million people in the U.S. have the disease, which lasts for years, according to the National Parkinson Foundation.

Ceregene’s unusual idea is to use a single shot loaded with genetically modified viruses, called AAV, that shuttle copies of genes into the brain. Once the genes get there, they will churn out growth factor proteins that could prevent cell death in the brains of Parkinson’s patients, Tuszynski says. Biotech’s biggest companies, Amgen and Genentech, haven’t been able to get their proteins to do their thing with other delivery methods, so this could represent an important advance for Parkinson’s patients and gene therapy, he says.

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