New drug sparks fresh hope for stroke patients by doubling intervention window

But now, researchers at Osaka Metropolitan University have developed a drug, called GAI-17, which works by preventing a protein from forming toxic clumps that kill brain cells during strokes.

The drug showed remarkable results in laboratory mice, significantly reducing brain damage and paralysis even when administered six hours after stroke onset.

The primary symptoms associated with strokes can occur suddenly, including weakness across facial muscles and arm muscles, as well as difficulty with speech. The memorable acronym that comes with the symptoms is Fast (Face, Arms, Speech, Time to call 999), the NHS advises.

Other lesser-known indicators include weakness and numbness across one side of your body, blurred vision or loss of sight in one or both eyes, finding it hard to speak or think of words, overall confusion and memory loss, dizziness and nausea.

During a stroke, oxygen deprivation causes the normally beneficial protein GAPDH (glyceraldehyde-3-phosphate dehydrogenase) to malfunction and stick together through chemical bonds.

This latest breakthrough could potentially help countless patients who currently arrive at hospital too late for existing treatments, which must be given within 4.5 hours for clot-busting drugs or six hours for mechanical clot removal procedures.

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These protein clumps then invade mitochondria, the cell's powerhouses, causing them to fail and ultimately killing brain cells. GAI-17 specifically targets this aggregation process without interfering with GAPDH's normal energy-production functions.

The three-amino-acid peptide binds near the protein's aggregation-prone site, preventing the formation of these deadly clusters.

Lead researcher Associate Professor Hidemitsu Nakajima discovered that GAPDH aggregation occurs before brain tissue actually dies, suggesting it might be a cause rather than merely a consequence of stroke damage.

The research team tested GAI-17 on mice with acute ischaemic strokes, the most common type affecting 87 per cent of stroke patients. Mice treated with the drug showed 45 per cent smaller areas of brain damage compared to untreated animals.

Crucially, GAI-17 demonstrated no concerning side effects on heart rate, blood pressure, or cerebral blood flow — critical safety factors for stroke treatments. The drug also didn't interfere with GAPDH's essential metabolic functions.

However, the compound's effectiveness has limits. When treatment began nine hours post-stroke, researchers found no benefit, suggesting biological constraints on how long brain cells can be rescued after oxygen deprivation.

The research has implications beyond stroke treatment. GAPDH aggregation occurs in Alzheimer's disease, Parkinson's disease, and other neurodegenerative conditions where oxidative stress damages brain cells.

"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," stated Professor Nakajima.

"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."