In a groundbreaking advancement for neurodegenerative disease therapy, a Canadian-led international research consortium has unveiled a novel compound showing unprecedented promise in the treatment of multiple sclerosis (MS). Spearheaded by Dr. Fang Liu, a Senior Scientist at the Centre for Addiction and Mental Health (CAMH) and a professor in the Department of Psychiatry at the University of Toronto, the team has secured nearly $800,000 US (approximately $1.14 million CAD) in funding to push this innovative therapy closer to human clinical trials. This research represents a critical stride toward regenerative medicine for MS, potentially not only halting disease progression but also reversing neurological damage.
Multiple sclerosis afflicts an estimated 2.8 million individuals worldwide and is characterized by the immune system’s misguided attack on myelin—the insulating sheath enveloping nerve fibers. This demyelination disrupts electrical signal transmission between the brain and the body, leading to an array of incapacitating symptoms such as profound fatigue, impaired vision, and loss of mobility. Although existing treatment modalities can attenuate the frequency and severity of flare-ups, none sufficiently address the repair of existing myelin damage or restore lost neurological function.
The paradigm-shifting therapy under investigation confronts a complex pathological mechanism known as excitotoxicity. This process involves excessive stimulation of glutamate receptors on neurons leading to ionic imbalance, cellular damage, and eventual neuronal death. In the context of MS, excitotoxicity exacerbates nerve cell injury, compounding the demyelination process. The novel compound developed by Dr. Liu’s team specifically targets and inhibits excitotoxic pathways without impairing normal synaptic transmission, thereby offering protective effects while maintaining essential brain functions.
Preclinical animal models have demonstrated compelling efficacy for this compound. When administered following the onset of MS-like symptoms, treated animals exhibited restoration in motor performance alongside histological evidence of myelin regeneration. These findings are particularly significant because they suggest the possibility of repairing existing neurological injury, a feat current clinical options cannot achieve. The capability to regenerate myelin and restore function introduces a fundamentally new therapeutic strategy that tackles neurodegeneration at its core rather than merely modulating immune response.
Dr. Liu, collaborating closely with Dr. Iain Greig, a Reader in Medicinal Chemistry at the University of Aberdeen, has propelled this research to the final stages of preclinical validation. The multi-disciplinary team combines expertise in neuropharmacology, medicinal chemistry, and neurobiology to optimize the compound’s efficacy and safety profiles. Their collaborative efforts have culminated in patent protection filed jointly by CAMH and the University of Aberdeen, underscoring the translational potential of their innovation.
Canada’s urgent need for breakthrough MS therapy is underscored by its disproportionately high national prevalence; approximately 90,000 Canadians currently live with MS, one of the highest rates globally. Dr. Liu emphasizes the transformative impact the compound could have, asserting that this innovation transcends symptom management to empower patients to reclaim neurological functions lost to the disease. This sentiment echoes throughout the research community involved in the project, highlighting the hope that regenerative treatments represent for this challenging condition.
Dr. Greig underscores the therapy’s broad implications beyond MS. Given that excitotoxicity is a pathological feature common to multiple neurodegenerative diseases, this compound’s mode of action could be adapted to combat conditions like Parkinson’s, Alzheimer’s, and amyotrophic lateral sclerosis (ALS). Such versatility enhances the compound’s value and fuels urgency for advancing clinical development, potentially revolutionizing neurodegenerative disease treatment at large.
The funding consortium backing this research includes prestigious bodies such as Brain Canada, the National MS Society’s Fast Forward program, and Health Canada via the Canada Brain Research Fund. Their financial support enables the team to refine pharmacodynamics, optimize dosing schedules, and conduct rigorous toxicology assessments essential for eventual human trials. Prior grants from the CAMH Discovery Fund Accelerator, the Canadian Institutes of Health Research (CIHR), and the National MS Society have laid vital groundwork leading to this pivotal stage of investigation.
As the project moves closer to clinical application, CAMH and the University of Aberdeen actively seek industry partners and investors to facilitate the transition from laboratory success to patient treatment. This phase involves navigating complex regulatory landscapes, scaling production, and designing human trials that will rigorously test safety and efficacy in individuals living with MS. The partnership aims not only to bring this therapy to market but also to establish a framework for rapid translation of regenerative neurological treatments.
CAMH, as a global leader in mental health and neuroscience research, continues to champion innovations that integrate scientific discovery with clinical impact. Dr. Aristotle Voineskos, CAMH’s Senior Vice President for Research and Science, expressed pride in leading these pioneering efforts, emphasizing the institution’s commitment to accelerating the translation of cutting-edge science into therapies that profoundly improve patient lives. This mission is vital as the neurodegenerative disease burden grows worldwide.
Representing Brain Canada, Dr. Viviane Poupon highlighted the significance of this development within Canada’s broader neuroscience ecosystem. By fostering collaborative research endeavors and public-private partnerships, Brain Canada accelerates breakthrough innovations like Dr. Liu’s compound. This synergy between funding, research institutions, and industry is critical for pushing the envelope of medical technology, bringing hope to millions affected by debilitating neurological diseases.
The National MS Society’s perspective, voiced by Dr. Walter Kostich, reinforced the therapy’s promise of not only halting MS progression but restoring neurological functions diminished by disease. The society invests heavily in research aimed at combating MS’s root causes, and this compound epitomizes the hopeful direction of such research. This alignment between patient advocacy and scientific advancement exemplifies the ecosystem needed to make substantial headway against complex, chronic diseases.
In sum, this therapeutic innovation, by selectively inhibiting excitotoxicity and fostering myelin repair, marks a revolutionary departure from traditional MS treatments. The ongoing efforts to transition from successful animal studies to human clinical evaluation represent a beacon of hope for the millions suffering from MS and related neurodegenerative disorders. Should this approach succeed, it could inaugurate an era of regenerative neurology, fundamentally changing the paradigm of treatment from management to cure.
Subject of Research: People
Article Title: Canada Leads Development of First Potentially Regenerative Multiple Sclerosis Therapy Targeting Excitotoxicity
News Publication Date: September 15, 2025
Web References:
https://www.camh.ca/en/camh-news-and-stories/new-funding-fuels-development-of-first-potentially-regenerative-treatment-for-multiple-sclerosis?sc_itemid=%7BC85C1CC3-58BB-4DCA-A816-B3F3F4694DB7%7D&item=web%3a%7bADAA849E-6081-449D-99C1-7EE5FD40088B%7d%40en
Keywords: Health and medicine, Diseases and disorders, Health care, Human health, Medical specialties, Clinical medicine, Neuroscience
Tags: Dr. Fang Liu multiple sclerosis studyexcitotoxicity in multiple sclerosisfunding for MS researchhuman clinical trials for MS therapyinnovative treatments for demyelinationinternational research consortium for MSmultiple sclerosis treatmentmyelin repair strategiesneurological damage reversalnovel compound for MSregenerative therapy for neurodegenerative diseasessymptoms of multiple sclerosis
