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HealthWatch: UCSF Researchers Make Brain Discovery

Dr. Kim Mulvihill

UCSF researchers may have discovered a new approach for treating brain
disorders such as Alzheimer's and Parkinson's through efforts to treat a childhood
disease.

Niemann Pick Type C usually strikes very young children, and causes a progressive
deterioration of the nervous system. It interferes with the child's ability to metabolize
cholesterol, and brain cells accumulate fat and die.

"It's a 100% fatal disease," said Dr. Synthia Mellon, a UCSF researcher. "All
children and adults who have this disease will die from it, and there is no treatment
for this disease."

Researchers looked at mice from a family that naturally develops the disease. The
animals at first couldn't perform the simplest task of crawling across a piece of
string. But with a single dose of an important hormone, a mouse could complete the
task with no apparent difficulty.

The hormone is a neurosteroid called allopregnanolone. The researchers
discovered that mice with Niemann Pick Type 3 made less and less of it, and it
appears to play a role in how our nervous system develops.

A single treatment of the hormone provided dramatic results, and researchers found
that the earlier they administered the treatment the longer the mouse lived. It's not a
cure, but the treatment did delay the onset of neurological problems.

The researchers caution that their findings must still be evaluated in humans, but
their research could have implications for treating other neurodegenerative
diseases like Parkinson's and Alzheimer's.

"I think it opens up a huge area of investigation that has previously been untapped,"
Mellon said.

» 07-21-2004

SOURCE: KPIX-TV 5, CA
http://tinyurl.com/4qoqg

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