Neurodegenerative gene focus of new Open Science project

GBA1-Canada (G-Can) aims to develop new treatment clinical trial within five years

A new project led by scientists at The Neuro is zeroing in on a gene important to the study of neurological diseases, with the goal of testing a drug therapy within five years. The primary objective is to improve the quality of life for patients by reducing or preventing disease symptoms.

The project, called GBA1-Canada, or G-Can, is focusing on a gene called GBA1. It plays a pivotal role in the study of Lewy body dementia and Parkinson’s disease. Mutations in this gene are found in 5 to 30 per cent of patients with one of these diseases.

As a group, patients with GBA1mutations tend to experience faster progression, with both non-motor and motor symptoms deteriorating more rapidly compared to patients without GBA1mutations. This means that developing new treatments for GBA1 mutations could benefit millions of people worldwide.

The G-Can project will create an open science platform to share data on individuals who carry mutations in GBA1, including genetic, transcriptomic, biomarkers, epidemiologic, clinical and imaging data. It will also generate and share different human-based and animal models to study GBA1, and provide a platform to accelerate the development on new treatments and performance of clinical trials.

G-Can will do this by leveraging The Neuro’s existing infrastructure, especially its Open Biobank, the Early Drug Discovery Unit, and multiple research labs, along with partner organizations like Quebec Parkinson Network (QPN), the International RBD Genomics Consortium, the Canadian Open Parkinson Network (C-OPN), Cure Parkinson’s, Michael J Fox Foundation and others. The project will later expand sample collection internationally by collaborations with multiple research and clinical institutes worldwide.

The ultimate goal of G-Can is to develop a clinical trial for a GBA1-targeted therapy within the next five years, followed by an additional one or two treatments five years after that. The data G-Can generates will be made available to the greater scientific community and the pharmaceutical industry through The Neuro’s Open Science principles, increasing the likelihood of successful treatment development.

“This is a very exciting project, which will bring together the world’s leading scientists and clinicians who work on GBA1,” says Dr. Ziv Gan-Or, Co-Director of the Clinical Research Unit at The Neuro and the Principal Investigator of G-Can. “The open science environment at The Neuro will guarantee rapid dissemination of data and tools so that we can advance treatments targeting GBA1 in neurodegenerative disorders.”

G-Can has been made possible thanks to the visionary support of The Hilary and Galen Weston Foundation, as well as generous donations from J. Sebastian van Berkom and Ghislaine Saucier, and Jonathan Silverstein, founder of The Silverstein Foundation for Parkinson’s with GBA. Philanthropy plays a crucial role in advancing innovative research in neurological disorders. The Neuro is grateful to these donors for their invaluable support.

See the link: https://www.mcgill.ca/neuro/article/research/neurodegenerative-gene-focus-new-open-science-project