UPEI researchers collaborating with partners to develop novel drug therapy to potentially slow ALS
Two researchers from UPEI are collaborating with colleagues at the Research Institute of the McGill University Health Centre (The Institute) and Neurodyn Life Sciences Inc., a PEI-based biotechnology company, on a project to develop a novel therapy to slow, or potentially functionally stabilize, the progression of amyotrophic lateral sclerosis (ALS).
A news release recently issued by the Quebec Consortium for Drug Discovery (CQDM) and Brain Canada says that the project, led by Dr. Andrew Bateman at The Institute, in partnership with UPEI and Neurodyn Life Sciences Inc., has been awarded $881,122. The funding partners for the project are the Quebec government via CQDM, Brain Canada, and Neurodyn Life Sciences Inc.
Working with Dr. Bateman are Dr. Andrew Tasker, Professor Emeritus, Neuropharmacology, and Dr. Jay Penney, Canada Research Chair in Biomedical Genetics, both with UPEI’s Faculty of Veterinary Medicine.
“The project explores the neuroprotective properties of granulins to counteract the toxic effects of the TDP-43 protein involved in disease progression,” said the news release. “Two therapeutic formulations will be evaluated to support future clinical trials. By combining expertise across disciplines, this initiative seeks to improve the quality of life of patients while strengthening Canada’s leadership in neuroscience.”
Granulins are active fragments of a larger protein called progranulin that plays important roles in maintaining healthy brain function but is not suitable as a therapy for various reasons. Because different granulins have different effects on cells, the researchers from The Institute and UPEI will use different granulin combinations and two different methods to develop the therapeutic formulations.
Dr. Bateman’s lab will test granulin formulations using motor neurons, which are cells in the brain and the spinal cord that send commands to the muscles regulating movement, speech, swallowing, and breathing. Dr. Penney’s lab will test the same formulations using microglia, which are key cells in brain development, maintenance of neuronal networks, and injury repair. Dr. Tasker will oversee testing of the most promising formulations in an animal model of ALS.
“The most effective formulation will hopefully move on to further trials,” said Dr. Penney.
Dr. Tasker said that ALS is primarily treated with therapeutic measures such as physiotherapy and language training as well as surgical techniques to aid swallowing and breathing that are mainly designed to keep patients comfortable as much as possible. Current drug therapy is limited in effectiveness.
“ALS is a progressive and inevitably fatal disease,” said Dr. Tasker. “As ALS progresses, patients lose use of their voluntary muscles first and ultimately their involuntary muscles, which control functions such as swallowing and breathing. The goal of this project is to find a way to slow, or in an ideal world, stop the progression of the disease.”
Dr. Greg Naterer, Vice-President, Academic and Research, congratulated Drs. Penney and Tasker on their collaboration in the project.
“This project has promising potential to improve the lives of people who have ALS,” he said. “It is also a prime example of the excellent collaborative research that is happening at UPEI—between faculty members on campus and with external partners.”
The project was one of five large-scale collaborative research projects that received funding totalling $5.4 million from CQDM and Brain Canada. The five projects are funded through grants from the Government of Québec, administered by CQDM, totaling $2,452,745, with additional support from several industry partners, matched by a $2,961,693 contribution from Brain Canada, through the Canada Brain Research Fund (CBRF).
To learn more, read the complete news release issued by CQDM and Brain Canada.