Testing theories in a new experimental model
Development of a synuclein-based model of Parkinson’s disease
Before researchers can test drugs or other therapies intended to treat or cure Parkinson’s disease on people, they must first be sure the medication or treatment is both safe and effective. To do that, they need models of the disease.
At McGill University, Dr. Philippe Huot, a neurologist and an assistant professor, is developing one such experimental model. He’s using it to study the behavior of a protein called alpha-synuclein, which researchers have identified as a key player in Parkinson’s disease.
Large accumulations, or clumps, of alpha-synuclein have been found in the brain cells of people with Parkinson’s. The prevailing theory is that these clumps prevent the regulatory process within dopamine-producing cells that would normally clean those cells of damaged proteins. Instead, they clog the process and the dopamine-producing cells die.
Huot and his team are going to inject alpha-synuclein into their experimental model and use imaging and observation to monitor the way the protein spreads within brain cells. They hope to discover whether alpha-synuclein interferes with the regular processes in the cells that produce dopamine. They’ll also study the resulting changes in movement that may occur, to see if the prevailing theory of how the disease progresses is correct.
For Huot, who spends a day a week treating people with Parkinson’s at a movement disorders clinic and the rest of his time in the lab, this project is particularly exciting.
“This project will open a whole new area of research, which is testing drugs in proper experimental models of Parkinson’s disease that can actually reverse the disease process and hopefully open the way to finding cures,” says Huot. “That’s what motivates me.”
The marriage of both research and clinical work is important for Huot because it allows him to learn from his patients and to share his progress with them.
“They are very inspirational, because they are suffering, but they are so resilient and hopeful,” he says. “I hope my work is also a source of hope for them.”