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IIH headaches could be treated with exenatide

Type 2 diabetes therapy is being tested on patients in phase 2 clinical trials.

A clinical trial has discovered that patients with relentlessly painful headaches – often known as idiopathic intercranial hypertension (IIH) – could potentially be treated with an injectable peptide. The therapy is typically used for individuals with type 2 diabetes.

The research, which was published in the journal Brain, revealed the findings from a phase 2 trial of the drug exenatide – a GLP-1 receptor agonist – as a candidate treatment for IIH.

The ‘IIH Pressure Trial’, led by the University of Birmingham team of neurologists and University Hospitals Birmingham, realised that for the seven patients who received regular injections of the drug, there was a notable drop in pressure in the brain during both short – 2.5 hours and 24 hours – and long-term measurements (12 weeks).

Meanwhile, the trial witnessed a significant reduction in the number of headaches across the 12 weeks that patients participated in the trial. Indeed, on average there were 7.7 fewer days per month of headaches compared to the baseline. This also contrasted favorably with the placebo arm which recorded only 1.5 fewer days.

Alex Sinclair, professor of neurology at the Institute of Metabolism and Systems Research at the University of Birmingham, was optimistic about the results of the study: “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 licensed drugs to treat IIH and hence this result is a major step forward for IIH patients.”

She added: “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 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.

“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.”

IIH is a debilitating illness that raises the pressure in the brain and can lead to chronic headaches and even permanent sight loss. The condition – which often leaves patients with a reduced quality of life – predominately affects women aged 25 to 36.