New Stroke Drug Shows Promise in Animal Trials

GAI-17 Reduces Brain Damage and Paralysis, Offers Hope for Broader Neurological Treatments

A novel drug, GAI-17, developed by Japanese researchers, has demonstrated significant potential in protecting brain cells and mitigating paralysis in stroke models. This breakthrough offers a glimmer of hope for patients and could pave the way for treatments for other brain disorders.

Targeting a Key Protein

Researchers at Osaka Metropolitan University have engineered a compound that inhibits the aggregation of glyceraldehyde-3-phosphate dehydrogenase (GAPDH). This multi-functional protein has been implicated in the progression of several neurological conditions. The findings were published in the journal iScience.

Extended Treatment Window Achieved

In studies involving mice engineered to experience acute strokes, GAI-17 administration led to a marked decrease in both brain cell death and paralysis. Crucially, the drug proved effective even when administered up to six hours after stroke onset, a significant improvement over the much tighter windows typically associated with existing stroke therapies. For context, the American Stroke Association notes that fast treatment is critical, as brain cells can begin to die within minutes.

Broad Neurological Potential

Associate Professor **Hidemitsu Nakajima**, who led the research, expressed optimism about the drug’s wider applications.

“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.”

—Hidemitsu Nakajima, Associate Professor

Favorable Safety Profile

Early tests indicate that GAI-17 exhibits no significant adverse effects, showing no negative impact on cardiac or cerebrovascular systems. This promising safety profile is a key factor for future clinical development.

The development of GAI-17 represents a substantial advancement in stroke research. By targeting a common mechanism across multiple neurological diseases with an apparently favorable safety record, this compound could usher in a new era of treatments for individuals affected by strokes and other neurodegenerative conditions.