Potential New Drug for Blinding Idiopathic Intracranial Hypertension

A new study has found that patients with ‘blinding’ headaches known as idiopathic intracranial hypertension (IIH) could be treated with an injectable peptide used for type 2 diabetes. The study, published in the journal Brain, reports on a phase two trial of a drug called exenatide, a GLP-1 receptor agonist, as a potential treatment for IIH.

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Injection Used for Type 2 Diabetes may Help with Idiopathic Intracranial Hypertension

The IIH Pressure Trial was led by a team of neurologists from the University of Birmingham and University Hospitals Birmingham. The researchers found that for the seven patients who received regular injections of the drug, which is currently approved for use in type 2 diabetes, led to a drop in the pressure of the brain during both - short (2.5 hours and 24 hours) and long-term (12 weeks) measurements.

Exenatide works by stimulating the pancreas to secrete insulin when blood sugar levels are high. Insulin helps move sugar from the blood into other body tissues where it is used for energy.

The trial also saw a significant reduction in the numbers of headaches across the 12 weeks that the participants took part in. The drug showed 7.7 fewer days per month of headaches compared to the baseline, which was only 1.5 fewer days for the placebo.

“This is a major trial for the rare and debilitating condition IIH that can lead to people, usually women, going blind and suffering disabling daily headaches. There are no current licenced drugs to treat IIH and hence this result is a major step forward for IIH patients,” said Alex Sinclair, Professor of Neurology in 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 study.

“We are delighted to see that the phase two trial resulted in our treatment group having lower brain pressure both immediately and after 12 weeks and nearly 8 fewer headache days across the 12-week period, and that all the women were able to continue the treatment throughout with few adverse effects. We now hope to see a much larger trial of exenatide to literally ease the pressure for the many people around the world suffering with IIH,” added Sinclair.

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IIH Affects Quality of Life

Idiopathic intracranial hypertension (IIH) is a debilitating disorder that causes increased pressure in the brain, resulting in chronic headaches, and possibly permanent vision loss. The illness, which often leaves patients with a reduced quality of life, usually affects women between the ages of 25 to 36 years old. Weight gain is a major risk factor of developing IIH and relapses of the disease.

The incidence of IIH is rising rapidly in line with the global rise in obesity and there has been a 350% increase in incidence in the last 10 years. Currently, there are no licensed drugs option and existing medications used off label are often complicated due to undesirable side effects.

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Shot in the Arm for Treatment of IIH

The best part about the drug is its rapid action. The results indicated that brain pressure was significantly reduced within two and a half hours of taking the medication. This rapid onset of action is imperative in a condition which can cause rapid blindness if left untreated.

“The results of this clinical trial are a shot in the arm for finding clinical treatments for IIH. While we need to do further trials before such a treatment could be available for patients in the future, we are encouraged by the significant results from this trial that made a real difference for those in the treatment arm and this treatment may prove relevant for other conditions resulting in raised brain pressure,” said Dr James Mitchell, Lecturer in Neurology at the University of Birmingham and first author of the paper.

In this study the drug was given as a twice-daily injection into the subcutaneous tissue. To reduce the need for frequent injections in the future a once weekly subcutaneous injection called Presendin will be trialed through University of Birmingham Start-up company, Invex Therapeutics.

The drug is vitally important for patients living with IIH and this trial brings hope to the millions of patients suffering due to the condition. Researchers are looking forward to the next steps and seeing the drug tested in two large Phase 3 clinical trials.

Ultimately the objective is to gain enough evidence to allow the drug to be licensed for use in IIH patients in the future.

References:

1. The effect of GLP-1RA exenatide on idiopathic intracranial hypertension: a randomized clinical trial - (https://academic.oup.com/brain/advance-article/doi/10.1093/brain/awad003/7065075)

Source-Medindia