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Promising results in stem cell research
Study yields helpful disease treatments
Monday, March 12, 2007
BY KITTA MacPHERSON
Star-Ledger Staff
Scientists have come up with what could someday be one of the first workable
treatments using embryonic stem cells to thwart a disease.
"Everybody is always saying to us, 'Well, you guys studying the human
embryonic stem cells, you haven't benefited anyone yet,'" said Evan Snyder,
a neuroscientist who has published breakthrough papers on both embryonic and
adult stem cells. "Well, this is it."
The international team, headed by Snyder, a physician-scientist at the
Burnham Institute in California, also compared the effectiveness of
embryonic stem cells versus the "adult" variety and found them to be equally
effective.
The data is so strong, Snyder said, he plans to call upon the federal Food
and Drug Administration to allow him to launch clinical trials immediately
to test the effectiveness of the treatment against Tay-Sachs disease, a
genetic disorder affecting Ashkenazi Jews.
The research was posted online yesterday on the Web site of the journal
Nature Medicine. Snyder, along with colleagues at Burnham and the University
of Oxford in England, studied mice with a mutation that impedes the normal
processing of fatty proteins for brain development.
The mice develop Sandhoff disease, a fatal neurodegenerative disorder
closely related to Tay-Sachs disease and part of a larger class of brain
diseases like Parkinson's, Alzheimer's and autism.
In the experiment, the scientists started by injecting mouse neural stem
cells into the brains of newborn Sandhoff mice. They found the new cells
replaced brain tissue damaged by the disease and did even more, repairing
nerve cells and even transmitting nerve impulses. This was evidence, the
scientists said, stem cells may integrate electrically and functionally into
a diseased brain.
"It shows that stem cells are not merely replacement parts -- it's not like
swapping out spark plugs," Snyder said. "There's a whole complex network of
cross-talk and that's how they exert their effects."
For the next step, the scientists conducted a set of parallel experiments,
injecting Sandhoff mice in one group with embryonic stem cells and the other
with "adult" cells that had been isolated from human fetal brain cells.

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