Novel drug molecule offers hope for early Parkinson's disease treatment

McGill UniversitySep 20 2024

A novel drug molecule could potentially lead to new treatments to prevent Parkinson's disease in younger patients, according to new research.

We are excited about this drug compound because we might have the possibility to develop the first cure for Parkinson's disease, at least for a subset of patients."

Kalle Gehring, lead author, Professor in the Department of Biochemistry at McGill University and Canada Research Chair in Structural Studies of Neurodegenerative Diseases

While Parkinson's symptoms -; slowed movements, tremors and balance problems -; often appear in people in their 60s, five to 10 per cent of people are diagnosed before they turn 40. The degenerative disorder affects over 100,000 Canadians, according to the researchers.

The study investigated how a molecule developed by the biotech company Biogen can reactivate a crucial protein called parkin. It normally plays a key role in maintaining healthy brain cells by clearing out damaged mitochondria, the energy powerhouses of cells. In some younger patients, mutations in parkin disrupt this process, leading to the buildup of damaged mitochondria that contributes to Parkinson's disease.

Using advanced technology at the Canadian Light Source (CLS) at the University of Saskatchewan, the researchers determined that the Biogen compound restores parkin's cleaning function by gluing together parkin and a natural activator found in cells.

The findings, published in Nature Communications, lay the foundation for the design of personalized treatments for younger patients with specific mutations, said the authors.

"The hope is that one day we'll find compounds that can treat Parkinson's disease in general," said Gehring, adding that it will be up to Biogen to apply the study's results in future drug development.

"As the population in Canada is aging and better treatments for other diseases are becoming available, neurodegenerative diseases such as Parkinson's will be a major health concern," he added.

McGill research groups led by Professor Jean-François Trempe in the Department of Pharmacology & Therapeutics and Professor Edward Fon at the Montreal Neurological Institute and Hospital collaborated on the study. It was funded by the Michael J. Fox Foundation, the Canadian Institutes of Health Research, and the Canada Research Chairs program.