Scientists discover common Diabetes drug reduces sight-threatening rise in brain pressure

Birmingham scientists conduct research to demonstrate that the GPL-1 agonist exendin-4 reduces intracranial pressure both rapidly and dramatically.

Research led by the University of Birmingham, UK, published today in Science Translational Medicine, shows that a GLP-1 agonist drug, currently used to treat patients with Type II diabetes could be repurposed to treat raised brain pressure [1].

Raised brain pressure is common in emergency situations such as traumatic brain injury, hydrocephalus and stroke, and is also the cardinal feature of Idiopathic Intracranial Hypertension (IIH), which causes disabling daily headaches and severely raised pressure around the nerves in the eye.  It also causes permanent vision loss in 25% of people.

The Birmingham researchers examined whether GLP-1 agonist drugs could reduce intracranial pressure in an animal model of raised brain pressure, and found that the GPL-1 agonist exendin-4 reduces intracranial pressure both rapidly and dramatically.

Corresponding author Dr Alexandra Sinclair, of the University of Birmingham’s Institute of Metabolism and Systems Research, said: “Our findings show that exendin-4 reduces brain pressure within 10 minutes of dosing and by 44%, which is a greater extent than anything else we’ve tested before, and the treatment effects last at least 24 hours.

There are currently no bespoke treatments for IIH, which mostly affects women in their 20s and 30s.  The primary treatment in IIH is acetazolamide, but many patients deteriorate and remain unwell despite treatment and the drug also has such severe side effects that our previous trials have shown that 48 per cent of patients stop taking it.”

Obesity is the major causative risk factor for IIH and GLP-1 agonists cause weight loss in both diabetic and non-diabetic patients. Thus this new therapeutic approach provides both a direct treatment for raised brain pressure and also a disease modifying approach through driving weight loss in IIH. 

The Birmingham team will be moving forward rapidly to clinical trials. 

Alexandra Sinclair commented: “GLP-1 agonists are safe and widely used drugs for the treatment of diabetes which means that these findings are rapidly translatable into a novel treatment strategy for IIH.  They are also potentially game-changing for other conditions featuring raised brain pressure including stroke and brain trauma.” 

The findings will be presented on September 8th and 9th in Vancouver at the International Headache Society Meeting, followed by the British Endocrine Society meeting in the UK from November 6th to 8th.

Exenatide, a synthetic version of exendin-4, has received Orphan Drug Designation for the treatment of Idiopathic Intracranial Hypertension from the European Medicines Agency and the US Food & Drug Administration.


1.      1.  Botfield et al (2017). Glucagon-like peptide-1 receptor agonists as a therapeutic to reduce intracranial pressure.  Science Translational Medicine

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