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’Engineered’ Stem Cells May Help Treat Parkinson’s

Thursday December 22, 2005

12/15/05(Forbes) - Engineered human progenitor brain stem cells are able to produce and deliver into the brain a growth factor that shows promise in treating Parkinson’s disease, say researchers at the University of Wisconsin-Madison.

A major challenge in treating Parkinson’s and other neurodegenerative brain disorders is getting drugs to the desired areas of the brain. The brain’s structure effectively blocks the delivery of most drugs via the bloodstream. The engineered progenitor brain cells are designed to sneak drugs past the blood-brain barrier.

The University of Wisconsin team obtained and grew large numbers of progenitor cells from human fetal brain tissue and engineered those cells to produce the growth factor called glial cell line-derived neurotrophic factor (GDNF).

Some small clinical trials showed that GDNF provide relief from the debilitating symptoms of Parkinson’s disease. However, GDNF could not cross the blood-brain barrier and had to be pumped directly into the brains of Parkinson’s patients in order to work.

In this latest study, researchers transplanted the engineered progenitor cells into the brains of rats and monkeys. The cells effectively integrated into the brains of the animals and delivered GDNF to the targeted brain areas.

The findings were published in the Dec. 15 issue of the journal Gene Therapy.

"This work shows that stem cells can be used as drug delivery vehicles in the brain," University of Wisconsin-Madison neuroscientist Clive Svendson said in a prepared statement.

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