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Altered cells deliver Parkinson's therapy to brain
Tue Dec 27, 2005 7:17 PM GMT9



   NEW YORK (Reuters Health) - Genetically modified nerve 'progenitor' cells
can be used as mini-pumps to deliver nerve growth factor to the brain, a new
study in animals shows.
 The results suggest such an approach could be used to treat Parkinson's
disease and other brain diseases in humans, Dr. Clive D. Svendsen of the
University of Wisconsin-Madison and colleagues report.
 A nerve growth factor called "glial cell line-derived neurotrophic
factor" (abbreviated to GDNF) has been shown to protect dopamine-producing
neurons, which are lost in Parkinson's disease, Svendsen and his team note in
the research journal Gene Therapy.
 In fact, it's safe to infuse GDNF into brain regions of patients with
Parkinson's disease, according to some studies, and it seems effective.
However, delivering the drug in this fashion is complex and only reaches a
single point in the brain.
 In the current study, using rats with symptoms akin to Parkinson's disease,
the researchers investigated the effect of human neural progenitor cells
engineered to produce GDNF.
 The rats were transplanted with the modified cells, and after two weeks these
were seen to have migrated to affected areas and to be secreting enough GDNF
to extend the survival of dopamine neurons and promote outgrowth of nerve
fibers.
 By five weeks post-transplant, the animals showed a "strong trend toward
functional improvement," and at eight weeks the cells were still releasing
the growth factor.
 Tests in elderly monkeys showed the cells survived and continued to release
GDNF for three months after transplant. None of the animals in the studies
developed brain tumors.
 Svendsen and his colleagues conclude that their results "show that combining
human progenitor cell therapy with ... gene therapy is a powerful approach to
the future treatment of Parkinson's disease and other neurological
conditions."
 SOURCE: Gene Therapy, online December 15, 2005.

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