CUHK Completes First Clinical Trial of GLP-1 Diabetes Drug for Severe Stroke Recovery
Key Insights
Researchers at The Chinese University of Hong Kong conducted the world's first randomized clinical trial evaluating GLP-1 receptor agonists (search) in severe stroke (search) patients undergoing reperfusion therapy.
The Phase 2 trial involving 140 patients demonstrated that semaglutide is safe and well-tolerated in acute stroke (search) settings with no severe adverse events attributed to the drug.
Exploratory analyses revealed a potential 20% improvement in neurological recovery among patients who did not receive intravenous thrombolysis before thrombectomy.
Researchers from The Chinese University of Hong Kong (CUHK) Faculty of Medicine have completed the world's first randomized clinical trial evaluating glucagon-like peptide-1 (search) receptor agonists (GLP-1RAs) as a potential neuroprotective therapy for severe stroke (search) patients. The groundbreaking Phase 2 study demonstrates the safety of repurposing diabetes (search) medications for stroke treatment and suggests potential benefits in specific patient populations.
Novel Therapeutic Approach for Stroke Recovery
The research team, led by Dr. Bonaventure Ip Yiu-ming, Assistant Professor in the Department of Medicine and Therapeutics at CU Medicine, conducted a randomized clinical trial involving 140 patients with severe large vessel occlusion (search) (LVO) stroke (search) who had undergone thrombectomy. Patients were randomized to receive either semaglutide, a GLP-1RA, in addition to standard therapy, or standard therapy alone.
GLP-1 is a peptide hormone produced naturally in the gut and brain that regulates bodily functions. GLP-1RAs, which are designed to mimic its action, are already widely used in the treatment of diabetes (search) and obesity (search). The team's previous studies demonstrated the anti-neuroinflammatory and blood-brain barrier stabilizing effects of GLP-1RAs in rodent models, underscoring their potential neuroprotective role in stroke (search).
Clinical Trial Results Show Safety and Potential Efficacy
The trial results showed that 56.5% of patients with severe LVO stroke (search) who received semaglutide achieved good functional recovery at 90 days, compared to 54.9% in the standard therapy group, indicating comparable efficacy between the two treatments. Importantly, no severe adverse events were attributed to the drug, and key safety outcomes, including death, malignant brain edema and intracranial hemorrhage, were similar between the two groups.
"These findings provide early clinical evidence supporting the safety of GLP-1RAs in acute stroke (search) settings, suggest potential benefits in selected patient groups and pave the way for a larger, phase 3 randomised trial to confirm the drug's neuroprotective potential," Dr. Ip commented.
Promising Results in Specific Patient Subgroups
Exploratory analyses revealed particularly encouraging results in patients who did not receive intravenous thrombolysis before thrombectomy. In this subgroup, there was a 20% higher proportion of patients treated with semaglutide achieving favorable neurological recovery compared to standard therapy alone.
"Among patient subgroups who did not receive intravenous thrombolysis before the therapy, patients who receive GLP-1RA actually had better neurological outcomes," Professor Bonaventure Ip explained. "The rate of good neurological outcomes was 20 percent more in the GLP-1RA group compared to the standard therapy group."
Addressing Critical Unmet Medical Need
Professor Thomas Leung Wai-hong, Head of the Division of Neurology and Lee Quo Wei Professor of Neurology in the Department of Medicine and Therapeutics at CU Medicine, highlighted the urgent need for such therapies. "While endovascular thrombectomy is highly effective at reopening blocked vessels, many patients still experience significant disability due to reperfusion injury and delayed treatment. This highlights the urgent need for adjunct neuroprotective therapies that can protect vulnerable brain tissue during and after treatment to maximise efficacy."
Scientific Foundation in Aging Biology
The clinical trial builds on extensive preclinical research led by Dr. Ko Ho, Director of the Gerald Choa Neuroscience Institute (search) and Associate Professor in the Department of Medicine and Therapeutics at CU Medicine. Using advanced multi-omics technologies, the team demonstrated that GLP-1RAs can counteract key biological processes associated with aging, with widespread molecular effects across multiple organs, including the brain and heart.
"Our work represents an important step in translating fundamental ageing research into clinical application. By linking multi-omic laboratory discoveries with patient-based investigation, our findings highlight the potential for GLP-1RAs to be repurposed as a new therapeutic indication in stroke (search) treatment," Dr. Ko remarked.
Future Clinical Development
The research findings, published in the internationally renowned journals Cell Metabolism and Nature Communications, establish a foundation for larger-scale clinical studies. Dr. Ip noted that the GLP-1RA adjunctive treatment could serve as "a complementary medication among patients who might not be able to receiving intravenous thrombolysis for further neuroprotection" within a 24-hour treatment window.
The study represents a significant "bench-to-bedside" advancement, translating discoveries in aging biology into a potential new treatment strategy for stroke (search), one of the leading causes of death and long-term disability worldwide.