By: Dr Avi Verma

In what could be a game-changing development for millions affected by stroke and neurodegenerative diseases, Japanese scientists at Osaka Metropolitan University have unveiled a new drug, GAI-17, that appears to significantly reverse stroke-induced brain damage—even when administered six hours after onset. Even more exciting? It may also hold potential to fight Alzheimer’s disease and other neurological disorders.

What is GAI-17?

GAI-17 is a protein aggregation inhibitor, developed by a research team led by Associate Professor Hidemitsu Nakajima from the Graduate School of Veterinary Science at Osaka Metropolitan University. The drug targets GAPDH (glyceraldehyde-3-phosphate dehydrogenase), a key enzyme with multiple roles in both metabolism and the progression of brain diseases.

In recent years, researchers have found that GAPDH, when it clumps together abnormally, plays a role in brain cell death after stroke and in chronic disorders like Alzheimer’s and Parkinson’s. GAI-17 works by preventing this protein from aggregating, thus safeguarding neurons from the damaging cascade that follows a stroke.

How does it work?

When mice were subjected to acute ischemic strokes in a controlled setting, those treated with GAI-17 demonstrated:

• Significantly reduced neuronal death
• Lower rates of paralysis
• Improved motor function
• Sustained neurological recovery

What’s particularly remarkable is that these improvements occurred even when the drug was given six hours after the stroke event. This is a far wider treatment window than current therapies like tPA (tissue plasminogen activator), which is only effective within 3–4.5 hours post-stroke and comes with risks of hemorrhage.

No major side effects reported

Another promising aspect of GAI-17 is its favorable safety profile. In early trials on animal models, the drug:

• Did not impact cardiovascular or cerebrovascular health
• Showed no toxicity to heart tissue
• Did not affect blood pressure or liver enzyme levels

While these are preclinical results, they represent a major step forward in stroke and Alzheimer’s research, where treatment options are severely limited and often come with serious side effects.

GAI-17 vs. Alzheimer’s and other brain disorders

Given GAPDH’s link to Alzheimer’s, the research team believes GAI-17 could have much broader uses than stroke therapy alone. Ongoing research is underway to explore its effectiveness in Alzheimer’s disease models, where GAPDH aggregation is also seen to contribute to neurodegeneration.

Professor Nakajima noted, “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.”

Why this matters for the Indo-US community

Stroke remains a leading cause of long-term disability and death in both India and the United States. According to the American Heart Association:

• Someone in the U.S. suffers a stroke every 40 seconds
• It is the 5th leading cause of death
• South Asians are particularly at risk due to genetic and lifestyle factors such as hypertension, diabetes, and obesity

If GAI-17 eventually passes clinical trials in humans, it could offer a longer treatment window, making it accessible to rural and underserved populations that typically miss the narrow emergency timeframe for existing treatments.

What’s next?

The researchers are preparing for human trials, focusing not just on stroke recovery but also long-term treatment strategies for dementia, ALS, and Huntington’s disease. While a commercial version of GAI-17 is likely still several years away, the early results are encouraging.

The study was published in the peer-reviewed journal iScience, and further testing will involve collaboration with neurology departments and global biotech firms.

Disclaimer
This article is for informational purposes only and does not constitute medical advice. GAI-17 is currently in preclinical stages and not yet approved for human use. Always consult with a licensed physician for diagnosis and treatment.