Patients with ‘blinding’ headaches known as idiopathic intracranial hypertension (IIH) could be treated with an injectable peptide used for type 2 diabetes, a new trial has found.
The study was led by a team of neurologists from the University of Birmingham and University Hospitals Birmingham, and published in the journal Brain. It reports on a Phase II trial of a drug called exenatide, a GLP-1 receptor agonist, as a potential treatment for IIH.
For the seven patients who received regular injections of the drug in the IIH Pressure Trial, there was a drop in pressure in the brain during both short (2.5hrs and 24hrs) and long term (12 weeks) measurements.
The trial also saw significant reductions in the numbers of headaches across the 12 weeks in which participants took part, with an average of 7.7 fewer days per month of headaches compared to the baseline, compared to only 1.5 fewer days in the placebo arm.
Alex Sinclair, Professor of Neurology in the Institute of Metabolism and Systems Research at the University of Birmingham and Principal Investigator of the study, said: “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.
“We are delighted to see that the Phase II trial resulted in our treatment group having lower brain pressure both immediately and after 12 weeks and nearly eight fewer headache days across the 12-week period, and that all the women were able to continue the treatment throughout with few adverse effects.”
Shot in the arm for IIH treatment
IIH predominately affects women aged 25 to 36 and weight gain is a major risk factor of developing IIH and relapses of the disease.
A key finding was the rapid action of the drug, with results indicating that brain pressure was significantly reduced within two and half hours of taking the medication.
Dr James Mitchell, Lecturer in Neurology at the University of Birmingham and first author of the paper, said: “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.”
In this study, the drug was given as a twice-daily injection into the subcutaneous tissue. In the future, a once-weekly subcutaneous injection called Presendin will be trialled though University of Birmingham start-up company, Invex Therapeutics.
Shelly Williamson, Chair of patient charity IIH UK, said: “This is such exciting progress. New drug options are vitally important for IIH and this trial brings hope to the millions of patients living with the condition. We very much look forward to the next steps and seeing the drug tested in two large Phase III clinical trials.”