Clinical and basic studies on the dynamics of ghrelin in Rett syndrome (RTT) together with a pilot-study on the clinical implication of ghrelin for the treatment of RTT-patients.

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In this study, we investigated the dynamics of ghrelin production in patients with Rett syndrome (RTT) together with that in mecp2-null mouse model of RTT (RTT-mouse). We also examined the effectiveness of “ghrelin-injection” on several clinical symptoms of RTT by using this RTT-mouse. Plasma levels of ghrelin were significantly lower in RTT patients with eating disorder or patients with insufficiency in gastro-intestinal movement. Deregulation of ghrelin production was also noted in the RTT-mouse. Daily injection of ghrelin into the RTT-mouse ameliorated the clinical symptom of them including “tremor”, “gait disturbance”, “hindlimb clasping” and “malnutrition” (body weight loss) from 6-9 weeks age. These findings of us support the future implication of “ghrelin-treatment” against several clinical symptoms typically seen in RTT patients.