The Idiopathic intracranial Hypertension (IIH) headaches are an illness that is characterized by an increase in pressure inside the skull, but without a clear reason. They are often misinterpreted as migraines and migraines, these headaches can be debilitating and can be related to symptoms like blindness, blurred vision and sometimes, even temporary loss of vision.




The phase 2 trial is the first to show a rapid and significant decrease in brain pressure as well as the regularity of headaches.

A recent study has found patients who suffer of “blinding” headaches, known as Idiopathic Intracranial Hypertension (IIH) may be treated using an injectable peptide that is commonly used to treat type 2 diabetes.

The study, which was published within the journal Brain is the result of a second phase study that looked at the possibility of using exenatide, a GLP-1 antagonist, as a possible treatment for IIH.




The IIH Pressure Trial led by neurologists of University Hospitals Birmingham and the University of Birmingham as well as University Hospitals Birmingham discovered that the seven patients who were regularly injected with the drug, which is currently accepted for treatment in Type 2 Diabetes, led to a reduction in cerebral pressure over shorter (2.5hrs and 24 hours) as well as long-term (12 weeks) measures.

The study also witnessed substantial reductions of the amount of headaches throughout the 12 weeks participants were enrolled in and experienced on average 7.7 less than the average number of days in a month with headaches when compared to baseline, in contrast to just 1.5 less than the baseline group.

Alex Sinclair is a Professor of Neurology at the Institute of Metabolism and Systems Research at the University of Birmingham and An Honorary Consultant Neurologist at the University Hospitals Birmingham NHS Foundation Trust and Chief Investigator of the research. Prof. Alex Sinclair said:




“This is a significant trial to treat the debilitating, rare condition IIH which can result in women and men becoming blind and suffering from every day headaches. There are currently no approved treatments for IIH so this study is an important step in the right direction for IIH patients.

“We are extremely pleased to note that the phase 2 trial led to our group experiencing lower pressure in the brain immediately and also after 12 weeks, and almost 8 fewer days of headaches over the 12 weeks, and that all women could keep the treatment going with no adverse consequences. We are now hoping to see an additional trial of exenatide that will literally reduce the pressure of the millions of patients around the globe suffering in IIH.”

In the arm shot to treat IIH treatment

Idiopathic Intracacranial Hypertension (IIH) is a chronic condition that increases the pressure of the brain. It may cause chronic headaches and eventually permanent loss of sight. The condition, which typically causes patients to have a lower level of quality of living, most often is experienced by women between the ages of 25 and 36. Weight growth is a major risk factor for the development of IIH and it is also a risk factor for relapses.

At one time, it was thought to be rare. the rate of IIH has been rising rapidly due to the worldwide rise in obesity and there is an increase of 350 percent in incidence over the past 10 years. At present, there aren’t any alternatives to licensed drugs and the existing drugs which are sold off-label can cause dangerous adverse negative effects.




The most significant finding was the speed of effects of the drug which indicated that the pressure in the brain was dramatically diminished within about two hours after having taken the drug. The rapid time to action is crucial in the case of a disease that could cause rapid blindness when not treated.

Dr. James Mitchell, Lecturer in Neurology at the University of Birmingham and first writer of the paper, stated: “The results of this clinical trial provide an important step to find treatments that can be used in clinical trials for IIH. Although we’ll need to conduct more trials before a treatment is available to patients in the near future we are pleased with the impressive results of this trial which had a positive effect on those who were in the treatment group and the treatment could be useful for other conditions that result in a rise in the pressure in your brain.”

In this study the drug was administered in two doses daily into the subcutaneous tissues. In order to reduce the requirement to have frequent injections in future, a once-weekly injection dubbed Presendin will be tested through University of Birmingham Start-up company, Invex Therapeutics.

Shelly Williamson, Chair of the patient charity IIH UK said: “This is an exciting development. The development of new drug options is critically crucial in the treatment of IIH as this study offers hope to the many IIH patients. We are looking at the next steps, and watching the drug being tested in two major clinical trials of Phase 3.”



Source “The impact of exenatide GLP-1RA in idiopathic intracranial Hypertension in a controlled clinical trial” written 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

IIH Advance IIH Advance is a Phase 3 clinical trial for adolescents that is conducted in the UK and supported by University of Birmingham and IIH Evolve is a trial for adults across the world, supported by Invex Therapeutics. In the end, the goal is to gather enough evidence for this drug to become approved to be used in IIH sufferers in the near future.