A new stroke treatment, GAI-17, developed by researchers in Japan, has shown encouraging results in reducing brain cell death and paralysis in animal models – potentially allowing for future development of treatments for other brain disorders.
Stroke is currently the second leading cause of death worldwide – following heart disease. In a new development, researchers from Osaka Metropolitan University have created a drug that could significantly reduce the brain damage typically caused by strokes.
Led by Associate Professor Hidemitsu Nakajima from the Graduate School of Veterinary Science, the team developed a novel compound aimed at protecting neurons by inhibiting a key protein involved in cell death.
The research findings have been published in iScience.
Targeting a multifunctional protein
The drug, named GAI-17, works by inhibiting the aggregation of glyceraldehyde-3-phosphate dehydrogenase (GAPDH) – a multifunctional protein that has been linked to the progression of various neurological disorders.
When GAI-17 was administered to mice modelled to have acute strokes, researchers observed a significant reduction in brain cell death and paralysis compared to mice that did not receive the treatment.
A potential treatment window of six hours
Crucially, GAI-17 demonstrated its effectiveness even when administered six hours after the onset of stroke, an important advance considering the typically narrow treatment window associated with most traditional therapies.
Additionally, the compound showed no major side effects, including no adverse impact on the heart or cerebrovascular system – a positive sign for future clinical development.
Hope for broader neurological applications
Professor Nakajima emphasised the broader potential of the new therapy:
“The GAPDH aggregation inhibitor we have developed is expected to be a single drug that can treat many intractable neurological diseases, including Alzheimer’s disease,” he said. “Going forward, we will verify the effectiveness of this approach in disease models other than stroke and promote further practical research toward the realisation of a healthy and long-lived society.”
Looking forward
The development of GAI-17 signifies a major step in stroke research – offering a potential therapy that not only limits brain damage but may also extend treatment windows well beyond current standards.
With its ability to target a common mechanism underlying multiple neurological diseases with a strong safety profile in early tests, GAI-17 could start the development of a new class of treatments for patients affected by strokes and other neurodegenerative disorders.