New Gene Therapy Shows Promise for Neurodegenerative Diseases, Including Stroke and Dementia
Researchers at MacKay Memorial Hospital have announced a breakthrough in gene therapy that offers potential for repairing damage caused by neurodegenerative diseases like stroke, dementia, and amyotrophic lateral sclerosis (ALS). The innovative approach, shared at the hospital’s recent “International Symposium on Innovative Treatment Strategies for Neurodegenerative Diseases,” focuses on restoring neurological function through gene delivery.
The research team, led by Dr. Lin Da- Xiong, Director of the Department of Translational Medicine at MacKay Memorial Hospital and Professor at MacKay Medical College, has developed a new gene therapy strategy. This involves using an AAV9 viral vector to deliver normal genes into the nervous system, successfully restoring nerve function in animal models. This development represents a significant step forward in the search for effective treatments for conditions that currently lack curative options and often lead to long-term disability.
The team initially focused on Krabbe disease, a rare genetic disorder, as a model for their research. This disease results from a deficiency in the GALC enzyme, leading to damage to the myelin sheath surrounding nerves, causing neurological deterioration, seizures, and paralysis. Infants with the most severe form of Krabbe disease typically have a life expectancy of around 13 months.
According to the study, the precise gene delivery technology allows the normal GALC gene to enter both the central and peripheral nervous systems. This enables cells to continuously produce active enzymes, reducing neurotoxic metabolites and decreasing nerve inflammation. Mice treated with this gene therapy experienced a significant extension in survival time and maintained their nerve structure and motor function.
“Conceptually estimating based on lifespan ratios, this outcome suggests that a single gene therapy could potentially extend the lives of patients who would otherwise face death in early childhood and improve their quality of life,” Dr. Lin stated. The research too establishes a new benchmark for gene therapy studies targeting neurodegenerative and rare white matter diseases.
The findings offer hope for new treatments beyond Krabbe disease, potentially impacting the care of patients with stroke, dementia, and other debilitating neurological conditions. Further research will be needed to determine the safety and efficacy of this approach in human clinical trials.
