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Dana-Farber Cancer Institute Released:
Scientists ID Switch for Brain’s Natural Anti-Oxidant Defense

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Scientists at Dana-Farber Cancer Institute report they have found how 
the brain turns on a system designed to protect its nerve cells from 
toxic “free radicals,” a waste product of cell metabolism that has 
been implicated in some degenerative brain diseases, heart attacks, 
strokes, cancer, and aging.

Newswise — Scientists at Dana-Farber Cancer Institute report they 
have found how the brain turns on a system designed to protect its 
nerve cells from toxic “free radicals,” a waste product of cell 
metabolism that has been implicated in some degenerative brain 
diseases, heart attacks, strokes, cancer, and aging.

Potentially, the researchers say, it may be possible to use drugs to 
strengthen the anti-oxidant system in the brain as a treatment for 
presently incurable diseases like Parkinson’s, Huntington’s, and 
Alzheimer’s and possibly other maladies.

Dana-Farber’s Bruce Spiegelman, PhD, and colleagues, using a mouse 
model, discovered that a regulatory protein, PGC-1a, switches on the 
anti-oxidant system when free radicals, or reactive oxygen species, 
begin to accumulate. It’s believed that some brain diseases involve a 
failure of the protective system, and the authors report that turning 
on PGC-1a to high levels in cultured cells protected them against 
nerve toxins. The findings will be reported in the Oct. 20 issue of 
the journal Cell.

“This could have broad implications for the many diseases in which 
reactive oxygen species are implicated,” said Spiegelman. Anti-
oxidant supplements have been used with some success in patients with 
neurodegenerative diseases, but Spiegelman noted that the process 
sparked by PGC-1a “is how nature does it.”

Researchers currently are screening drugs in search of compounds that 
could spur PGC-1a expression in brain cells, as well as exploring 
whether any harmful side effects might result. PGC-1a is a 
transcriptional co-activator discovered in Spiegelman’s Dana-Farber 
laboratory in 1998. It has subsequently been found to play a master 
regulatory role in metabolic processes and muscle function, as well 
as being a culprit in diabetes.

The report establishes for the first time that PGC-1a both drives the 
mitochondria to make energy and triggers the cleanup of toxic free 
radicals that accumulate in the cell as byproducts. As excess free 
radicals build up, their toxicity places the cell under “oxidative 
stress,” which prompts the cell to produce more PGC-1a, which in turn 
spurs the anti-oxidant defenses into action.

“With this mechanism, the body can speed up mitochondrial formation 
and at the same time suppress the creation of reactive oxygen 
species, which are known to be terribly damaging to the cell,” 
explains Spiegelman, who is also a professor of cell biology at 
Harvard Medical School. In this respect, the cell could be compared 
to a self-cleaning oven — but one that becomes less efficient with 
age and in certain diseases.

Therefore, the new finding of a specific regulator of the body’s own 
anti-oxidant system could lead to more-effective treatments for a 
number of diseases, and might even retard some of the effects of 
aging, the researchers say.

In previous experiments, Spiegelman and others had bred mice that 
lacked the PCG-1a gene. As would be expected, the absence of PCG-1a 
caused the mice to have abnormalities in their metabolism — they had 
less exercise capacity and were extremely sensitive to cold. But what 
the scientists hadn’t predicted was that the mice had 
neurodegenerative lesions in their brains and behaved abnormally: 
This was a clue that without PGC-1a, the cells’ “self-cleaning” 
mechanism wasn’t activated properly, leaving the mice more vulnerable 
to brain damage from renegade free radicals.

In the current research, Spiegelman and his colleagues exposed normal 
mice and rodents lacking PGC-1a to a nerve toxin that accelerates the 
production of free radicals. Mice without PGC-1a suffered more brain 
damage because they couldn’t turn on their anti-oxidant defenses.

Finally, to investigate whether increasing PGC-1a activity in the 
brain would protect against oxidative stress, the scientists caused 
mouse brain cells and human brain cells in the laboratory to make 40 
times as much PCG-1a as normal. They exposed the cells to increasing 
amounts of paraquat or hydrogen peroxide, chemicals that cause 
oxidative stress and cell damage. The result: many more brain cells 
survived the assault than did cells without the extra PGC-1a activity 
to augment their defenses.

Because PGC-1a has now been shown both to rev up energy production in 
the mitochondria and to suppress the resulting free radicals, “this 
is an almost ideal protein to control or limit the damage seen in 
neurodegenerative diseases that have been associated with defective 
mitochondrial function,” the authors wrote. As a result, finding 
drugs that increase PGC-1a in the brain “could represent a new mode 
of therapy for a set of diseases that are both common and have only 
marginal therapies at this moment.”

Lead authors of the report are Julie St-Pierre, PhD, and Stavit 
Drori, PhD, formerly of Dana-Farber and Harvard Medical School. The 
paper’s co-authors are based at Dana-Farber, Beth Israel Deaconess 
Medical Center, and Harvard Medical School.

The research was funded in part by the National Institutes of Health.

Dana-Farber Cancer Institute (http://www.dana-farber.org) is a 
principal teaching affiliate of the Harvard Medical School and is 
among the leading cancer research and care centers in the United 
States. It is a founding member of the Dana-Farber/Harvard Cancer 
Center (DF/HCC), designated a comprehensive cancer center by the 
National Cancer Institute.

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