Genes run just about everything that happens in our bodies, including how proteins behave in the brain. Too little or too much of a particular protein can cause some of the most devastating neurological diseases and disorders.
Dr. Brian Chen, a scientist from the Brain Repair and Integrative Neuroscience (BRaIN) Program at the Research Institute of the McGill University Health Centre (RI-MUHC), has been awarded the first Healthy Brains for Healthy Lives (HBHL) Neuro-Partnerships grant to develop a drug screening protocol designed to quickly identify promising treatments for patients with diseases caused by low gene expression.
The protocol will use a gene editing technique to insert a Protein Quantification Reporter (PQR) into a mutant gene to measure protein synthesis over time. Once the cells have been edited with the PQR, researchers will “wash” them with medical compounds, monitoring which ones boost protein synthesis.
Initially, Dr. Chen’s work will focus on three genes, including GBA1, which has been linked to Gaucher’s disease and Parkinson’s disease, but the drug-screening protocol could have broader applications.
“This approach will generate molecular treatments for any disease or disorder caused by insufficient gene expression,” said Dr. Chen, “and open up a new avenue for individualized and rapid drug discovery for these patients.”
Funds provided by HBHL and other partners will allow Dr. Chen’s lab to access libraries of FDA-approved compounds, and automate high-throughput cell assays that will accelerate the pace of research.
“Ideally, we will find compounds that will allow us to skip directly to Phase 2 clinical trials,” said Chen.
HBHL’s Neuro-Partnerships program co-funds projects with a high potential impact on biopharmaceutical research through existing programs offered by the Ministère de l'Économie et de l'Innovation (MEI). MEI co-funding is available through CQDM Quantum Leap and SynergiQc programs, which includes funding from Pfizer Canada. The Brain Canada Foundation has also contributed to Dr. Chen’s research.