Date:
October 8, 2014
Source:
University of California, Los
Angeles (UCLA), Health Sciences
Summary:
A
drug being evaluated to treat an entirely different disorder helped slow the
progression of Parkinson’s disease in mice, a team of researchers has reported.
Their study found that the drug, AT2101, which has also been studied for
Gaucher disease, improved motor function, stopped inflammation in the brain and
reduced levels of alpha-synuclein, a protein critically involved in
Parkinson's.
A
new study from UCLA found that a drug being evaluated to treat an entirely
different disorder helped slow the progression of Parkinson's disease in mice.
The
study, published in the October edition of the journal Neurotherapeutics,
found that the drug, AT2101, which has also been studied for Gaucher disease,
improved motor function, stopped inflammation in the brain and reduced levels
of alpha-synuclein, a protein critically involved in Parkinson's.
though
the exact cause of Parkinson's is unknown, evidence points to an accumulation
of alpha-synuclein, which has been found to be common to all people with the
disorder. The protein is thought to destroy the neurons in the brain that make
dopamine, a neurotransmitter that helps regulate a number of functions,
including movement and coordination. Dopamine deficiency is associated with
Parkinson's disease.
Gaucher
disease is a rare genetic disorder in which the body cannot produce enough of
an enzyme called β-glucocerebrosidase, or GCase. Researchers seeking genetic
factors that increase people's risk for developing Parkinson's have determined
that there may be a close relationship between Gaucher and Parkinson's due to a
GCase gene. Mutation of this gene, which leads to decreased GCase activity in
the brain, has been found to be a genetic risk factor for Parkinson's, although
the majority of patients with Parkinson's do not carry mutations in the Gaucher
gene.
"This
is the first time a compound targeting Gaucher disease has been tested in a
mouse model of Parkinson's disease and was shown to be effective," said
the study's senior author, Marie-Francoise Chesselet, the Charles H. Markham
Professor of Neurology at UCLA and director of the UCLA Center for the Study of
Parkinson's Disease. "The promising findings in this study suggest that
further investigation of this compound in Parkinson's disease is
warranted."
In
the study, the researchers used mice that were genetically engineered to make
too much alpha-synuclein which, over time, led the animals to develop deficits
similar to those observed in humans with Parkinson's. The researchers found
that the mice's symptoms improved after they received AT2101 for four months.
The
researchers also observed that AT2101 was effective in treating Parkinson's in
mice even though they did not carry a mutant version of the Gaucher gene,
suggesting that the compound may have a clinical effect in the broader
Parkinson's population.
AT2101
is a first-generation "pharmacological chaperone" -- a drug that can
bind malfunctioning, mutated enzymes and lead them through the cell to their
normal location, which allows the enzymes to carry on with their normal work.
This was the first time that a pharmacological chaperone showed promise in a
model of Parkinson's, according to Chesselet.
Parkinson's
disease affects as many as 1 million Americans, and 60,000 new cases are
diagnosed each year. The disorder continues to puzzle scientists. There is no
cure and researchers have been unable to pin down its cause and no drug has
been proven to stop the progression of the disease, which causes tremors,
stiffness and other debilitating symptoms. Current Parkinson's treatments only
address its symptoms.
Story Source:
The
above story is based on materials
provided by University of
California, Los Angeles (UCLA), Health Sciences. Note:
Materials may be edited for content and length.
end
story_source
Journal Reference:
1 Franziska Richter,
Sheila M. Fleming, Melanie Watson, Vincent Lemesre, Lee Pellegrino, Brian
Ranes, Chunni Zhu, Farzad Mortazavi, Caitlin K. Mulligan, Pedrom C. Sioshansi,
Sindalana Hean, Krystal De La Rosa, Richie Khanna, John Flanagan, David J.
Lockhart, Brandon A. Wustman, Sean W. Clark, Marie-Françoise Chesselet. A
GCase Chaperone Improves Motor Function in a Mouse Model of Synucleinopathy.
Neurotherapeutics, 2014; DOI: 10.1007/s13311-014-0294-x
end
journal_references
Cite This Page:
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University of
California, Los Angeles (UCLA), Health Sciences. "Drug used for another
disease slows progression of Parkinson's." ScienceDaily. ScienceDaily, 8
October 2014. <www.sciencedaily.com/releases/2014/10/141008153507.htm
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