February 26, 2024

New Drug Could Treat “Blinding” IIH Headaches

The trial also saw substantial decreases in the number of headaches throughout the 12 weeks that participants took part, with approximately 7.7 fewer days monthly of headaches compared to the standard, compared to only 1.5 less days in the placebo arm.
Alex Sinclair is a Professor of Neurology at the Institute of Metabolism and Systems Research at the University of Birmingham, an Honorary Consultant Neurologist at University Hospitals Birmingham NHS Foundation Trust, and Principal Investigator of the research study. Teacher Alex Sinclair said:
” This is a significant trial for the devastating and uncommon condition IIH that can lead to people, generally females, going blind and suffering disabling day-to-day headaches. There are no current licensed drugs to treat IIH and hence this outcome is a major action forward for IIH clients.
” We are delighted to see that the stage 2 trial led to our treatment group having lower brain pressure both immediately and after 12 weeks and almost 8 fewer headache days across the 12-week duration, and that all the females had the ability to continue the treatment throughout with few negative effects. We now want to see a much larger trial of exenatide to actually ease the pressure for the lots of individuals around the world experiencing IIH.”
Shot in the arm for IIH treatment
Idiopathic Intracranial Hypertension (IIH) is a debilitating condition that raises pressure in the brain and can cause chronic headaches and even long-term sight loss. The illness, which often leaves patients with a decreased quality of life, predominately affects women aged 25 to 36 and weight gain is a major risk factor for establishing IIH and regressions of the illness.
As soon as considered uncommon, the incidence of IIH is now rising considerably in line with the international rise in weight problems and there has been a 350 percent rise in incidence in last 10 years. Currently, there are no licensed drug alternatives, and existing medications utilized off-label are made complex by troublesome adverse effects.
A crucial finding was the quick action of the drug, with outcomes indicating that brain pressure was significantly lowered within 2 and a half hours of taking the medication. This rapid start of action is vital in a condition which can cause fast loss of sight if left untreated.
Dr James Mitchell, Lecturer in Neurology at the University of Birmingham and very first author of the paper said: “The outcomes of this scientific trial are a shot in the arm for finding clinical treatments for IIH. While we need to do more trials prior to such a treatment might be readily available for clients in the future, we are motivated by the significant outcomes from this trial that made a real difference for those in the treatment arm and this treatment may prove pertinent for other conditions leading to raised brain pressure.”
In this study, the drug was given as a twice-daily injection into the subcutaneous tissue. To minimize the need for regular injections in the future a once-weekly subcutaneous injection called Presendin will be trialed though University of Birmingham Start-up business, Invex Therapeutics.
Shelly Williamson, the Chair of client charity IIH UK said: “This is such amazing progress. New drug alternatives is extremely essential for IIH and this trial brings hope to the countless clients coping with the condition. We extremely much look forward to the next steps and seeing the drug tested in 2 large Phase 3 scientific trials.”
Reference: “The effect of GLP-1RA exenatide on idiopathic intracranial hypertension: a randomized medical trial” by James L Mitchell, Hannah S Lyons, Jessica K Walker, Andreas Yiangou, Olivia Grech, Zerin Alimajstorovic, Nigel H Greig, Yazhou Li, Georgios Tsermoulas, Kristian Brock, Susan P Mollan and Alexandra J Sinclair, 13 March 2023, Brain.DOI: 10.1093/ brain/awad003.
The IIH Advance is a Phase 3 clinical trial in Adolescents run in the UK, sponsored by the University of Birmingham and IIH Evolve is running in grownups globally sponsored by Invex Therapeutics. Eventually the aim is to get adequate evidence to allow the drug to be certified for use in IIH clients in the future.

Idiopathic Intracranial Hypertension (IIH) headaches are a condition characterized by increased pressure within the skull without a recognizable cause. Often mistaken for migraines, these headaches can cause incapacitating discomfort and are commonly connected with signs such as blurred vision, tinnitus, and even momentary vision loss.
The stage 2 trial is the first research study to demonstrate a swift and considerable reduction in brain pressure and the frequency of monthly headaches.
A new trial has found that people experiencing “blinding” headaches, called Idiopathic Intracranial Hypertension (IIH), could possibly be treated with an injectable peptide generally used for type 2 diabetes.
The research study, released in the journal Brain, presents the results of a phase two trial checking out using exenatide, a GLP-1 receptor agonist, as a potential therapy for IIH.
The IIH Pressure Trial led by a team of neurologists from the University of Birmingham and University Hospitals Birmingham found that for the seven clients who received regular injections of the drug, currently authorized for use in Type 2 Diabetes, resulted in a drop in pressure in the brain during both brief (2.5 hrs and 24hrs) and long term (12 weeks) measurements.