A stroke drug called GAI-17 protected mouse brains even six hours after damage began—and may one day help fight Alzheimer’s too, without harmful side effects. Credit: Shutterstock
A powerful new molecule, GAI-17, has shown striking results in reversing brain damage caused by stroke—even when given up to six hours after the event.
Developed by researchers in Japan, the drug blocks a protein that triggers neuron death, sharply reducing paralysis and brain cell loss in mice. Even better, it caused no concerning side effects and could potentially treat other neurological diseases like Alzheimer’s. This could be a game-changer in stroke treatment—and beyond.
Stroke is said to be the second leading cause of death worldwide after heart disease. To prevent the death of neurons in the brain, a research group led by Osaka Metropolitan University Associate Professor Hidemitsu Nakajima of the Graduate School of Veterinary Science has developed a drug that inhibits a protein involved in cell death.
The multifunctional protein GAPDH (glyceraldehyde-3-phosphate dehydrogenase) is linked to pathogenesis in many intractable brain and nervous system diseases. The team developed GAI-17, a GAPDH aggregation inhibitor. When this inhibitor was administered to model mice with acute strokes, there was a significantly lower level of brain cell death and paralysis compared to untreated mice.
GAI-17 also showed no side effects of concern, such as adverse effects on the heart or cerebrovascular system. Furthermore, experiments using GAI-17 showed improvement in the mice even when administered six hours after a stroke.
Broad Promise of GAI-17 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,” 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 realization of a healthy and long-lived society.”
The findings were published in iScience.
Reference: “Inhibition of GAPDH aggregation as a potential treatment for acute ischemic stroke” by Masanori Itakura, Takeya Kubo, Akihiro Kaneshige, Masatoshi Nakatsuji, Naoki Harada, Ryoichi Yamaji, Takatoshi Hikida, Takashi Inui and Hidemitsu Nakajima, 2 May 2025, iScience.
DOI: 10.1016/j.isci.2025.112564