Modulation of the endocannabinoid system in the brain can ameliorate disease symptoms in a mouse model of fragile X syndrome reports a study published this week in Nature Medicine. The findings suggest a promising therapeutic approach to treat the abnormal behaviors exhibited by people with this disorder.

Fragile X syndrome is a neurodevelopmental disease that includes autism-like features and is caused by a drop in the expression of the synaptic protein FMRP. Affected individuals exhibit cognitive dysfunction, enhanced susceptibility to seizures, and less sensitivity to pain, along with other abnormal behaviors. Using a mouse model lacking the Fmr1 gene that recapitulates some of the human symptoms, Andres Ozaita and colleagues found that treatment with the cannabinoid receptor antagonist, rimonabant, could normalize cognition, sensitivity to pain, and seizure susceptibility.