MIAMI—The possibility that Parkinson’s dis- ease is a prion disorder has led investigators
to begin clinical trials of drugs that target misfolded
α-synuclein, according to an overview provided at the
First Pan American Parkinson’s Disease and Movement
Disorders Congress. Other potential therapies targeting the protein are in preclinical studies.
Researchers are exploring four main treatment approaches: immunization, enhancing protein clearance, knocking down host α-synuclein, and inhibiting the prion conformer reaction, said C. Warren
Olanow, MD, Professor and Chairman Emeritus of the
Department of Neurology and Professor Emeritus of
Neuroscience at the Mount Sinai School of Medicine in
New York City.
Inhibiting the prion conformer reaction is the approach that Dr. Olanow believes is most likely to be
effective. “But I also strongly believe every one of these
approaches needs to be tested,” he said.
The Prion Hypothesis
A prion is an infectious particle composed solely of misfolded protein. It replicates by a process whereby the
misfolded protein causes the native unfolded protein to
misfold through a process called the prion conformer reaction. Misfolded proteins form beta-rich sheets that contain
oligomers and rods, which are thought to be toxic, and
polymerize to form aggregates. Prions can cause neurodegeneration and be transmitted from one species to another.
Will Targeting Alpha-Synuclein Lead to
Effective Parkinson’s Disease Therapies?
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