• Philip R Holland, Simon Akerman, Anna P Andreou, Nazia Karsan, John A Wemmie, and Peter J Goadsby.
• Headache Group, Department of Neurology, University of California at San Francisco, San Francisco, CA 94115, USA.
• Ann. Neurol. 2012 Oct 1; 72 (4): 559-63.

ObjectiveMigraine with aura is a severe debilitating neurological disorder with few relatively specific therapeutic options.MethodsWe used amiloride, a blocker of epithelial sodium channels, to evaluate its pharmacological potential and explored the biology of a potential mechanism of action in well-established experimental models.ResultsAmiloride was shown to block cortical spreading depression, the experimental correlate of aura, and inhibited trigeminal activation in in vivo migraine models, via an acid-sensing ion channel 1 mechanism. Remarkably, amiloride then demonstrated good clinical efficacy in a small open-labeled pilot study of patients, reducing aura and headache symptoms in 4 of 7 patients with otherwise intractable aura.InterpretationThe observations here identify an entirely novel treatment strategy for migraine.Copyright © 2012 American Neurological Association.

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