CoCensys has started a 250-patient study of its lead neuroactive steroid(epalon) compound, ganaxolone (CCD 1042), in migraine sufferers. Ganaxolone is a synthetic version of a naturally-occurring epalon that binds to specific sites on the GABAA receptors in the brain. The drug is already in Phase II trials for epilepsy.
Ganaxolone differs from other migraine drugs, which act via serotonergic pathways to modulate blood vessel dilation in the central nervous system. CoCensys notes that nerves innervating CNS blood vessels release vasoactive peptides, which cause local inflammation as well as vasodilation. The company hopes that using drugs such as ganaxolone to mediate this inflammation might help alleviate migraine pain.
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