New Drug Molecule Offers Hope for Treating Early-Onset Parkinson’s Disease
A novel drug molecule may reactivate parkin, offering potential treatments for younger patients with Parkinson’s disease.
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Summary
New research suggests that a novel drug molecule could lead to treatments preventing Parkinson’s disease in younger patients. The study, led by Kalle Gehring at McGill University, focuses on a Biogen-developed compound that reactivates the parkin protein, crucial for clearing damaged mitochondria. This approach could pave the way for personalized treatments.
Key Takeaways
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,” said lead author Kalle Gehring, a 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.
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Subscribe for FREEThe 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.
Reference: Sauvé V, Stefan E, Croteau N, et al. Activation of parkin by a molecular glue. Nat Commun. 2024;15(1):7707. doi: 10.1038/s41467-024-51889-3
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