GLP-1 Receptor Agonists Show Promise in Treating Idiopathic Intracranial Hypertension

In a significant advancement in the treatment of idiopathic intracranial hypertension (IIH), a recent study published on July 14, 2025, in the *JAMA Neurology* journal indicates that glucagon-like peptide-1 receptor agonists (GLP-1 RAs) may lead to improved clinical outcomes compared to conventional therapies. Conducted by Dr. Georgios S. Sioutas and colleagues from the Virginia Commonwealth University Health System, the research utilized data from the TriNetX U.S. Collaborative Network, covering patient records from 67 health care organizations between 2005 and 2024.

The study's findings are particularly noteworthy, as they reveal that the use of GLP-1 receptor agonists is associated with a reduction in medication use and a significant decrease in the incidence of headaches, visual disturbances, and papilledema—key symptoms of IIH. Specifically, the results showed risk ratios of 0.53 for reduced medication use, 0.45 for headaches, 0.60 for visual disturbances, and 0.19 for papilledema. Additionally, the GLP-1 RA cohort exhibited a lower risk of requiring medical procedures and mortality, with risk ratios of 0.44 and 0.36, respectively. Importantly, the study noted no significant differences in mean body mass index (BMI) at follow-up among participants, highlighting the effectiveness of GLP-1 RAs irrespective of weight status.

Dr. Sioutas emphasized, "This retrospective multicenter study suggests that GLP-1 RAs may benefit IIH management. However, prospective studies are warranted to validate these findings." This statement is echoed by Dr. Nancy J. Newman, a renowned neurologist, who stated in a related commentary in *JAMA Neurology*, "The time has come for rigorous trials to explore the full potential of GLP-1 receptor agonists in IIH treatment."

The study involved a total of 44,373 patients with IIH, from which 555 GLP-1 RA users were matched with 555 nonusers through propensity score matching. The control group primarily consisted of patients treated with conventional methods such as acetazolamide, topiramate, and dietary counseling, which have historically been the standard treatment for IIH.

The implications of these findings are profound, as IIH is often associated with obesity and can lead to serious complications such as permanent vision loss. The potential for GLP-1 receptor agonists to not only alleviate symptoms but also decrease the necessity for more invasive procedures marks a promising shift in treatment paradigms.

Experts are now calling for further investigation into the long-term effects of GLP-1 RAs on IIH and their underlying mechanisms. The findings also align with a broader trend in the medical community, which is increasingly recognizing the role of metabolic therapies in neurological conditions. As Dr. Sioutas concluded, "The results pave the way for innovative approaches in the management of IIH, potentially changing the therapeutic landscape for affected patients."

In summary, while the study presents compelling evidence supporting the use of GLP-1 receptor agonists in the management of idiopathic intracranial hypertension, ongoing research will be critical to confirm these observations and to establish new treatment guidelines that incorporate these findings into clinical practice.