A new Phase II trial led by the University of Birmingham and University Hospitals Birmingham neurologists has found that an injectable peptide used for type 2 diabetes, exenatide, could treat patients with ‘blinding’ headaches known as Idiopathic Intercranial Hypertension (IIH).

The IIH Pressure Trial of the GLP-1 receptor agonist exenatide also saw a significant reduction in pressure in the brain.

In the trial, exenatide was given as an injection into the subcutaneous tissue twice-a-day.

Seven patients who received regular injections of the drug in the Phase II trial reported a drop in pressure in the brain during short (2.5 hours and 24 hours) and long term (12 weeks) measurements.

Additionally, the trial saw reductions in the number of headaches across the 12-week period of the trial, with an average of 7.7 fewer headache days a month compared to the baseline, against only 1.5 fewer days in the placebo arm.

University of Birmingham Institute of Metabolism and Systems Research Neurology professor Alex Sinclair 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 licensed drugs to treat IIH and hence this result is a major step forward for IIH patients.

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“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.

“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.”

Sinclair is also the principal investigator of the trial.

A key observation in the trial was the drug’s rapid action and results indicated a significant reduction in brain pressure within two and a half hours of taking the medication.