A new drug, GAI-17, has reduced brain cell death and paralysis in mice after stroke, even when given six hours after onset.
Researchers found the compound blocks a protein linked to cell death, suggesting it may extend the treatment window for stroke and reduce long-term damage.
The team, based at Osaka Metropolitan University in Japan, developed GAI-17 to inhibit glyceraldehyde-3-phosphate dehydrogenase (GAPDH), a protein involved in various cellular functions and associated with neurological disorders.
Mice given the drug after induced strokes showed significantly less brain damage and paralysis than those that did not receive treatment.
Stroke causes rapid brain cell death due to a lack of oxygen. Most therapies must be delivered quickly to be effective. GAI-17’s effectiveness at six hours post-stroke could represent a potential advance.
The compound showed no major side effects in tests, including no adverse impact on the heart or cerebrovascular system.
Associate professor Hidemitsu Nakajima, from the Graduate School of Veterinary Science, led the study.
He said: “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.
“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.”
Stroke is the second leading cause of death globally, after heart disease.
Without swift intervention, blocked blood flow can result in permanent brain damage.
While these results are from animal models, researchers say GAI-17 could help pave the way for a new class of treatments targeting mechanisms common to several brain disorders.
Human trials would be needed to confirm its safety and effectiveness.
