[SP4-3B-8] A New Therapeutic Approach for Rett Syndrome: A Single-Institution Trial of Ghrelin Treatment
[Introduction] Rett syndrome (RTT) is a neurodevelopmental disorder and a potential clinical key entity in the pathogenesis of intellectual disability, autistic disorders, and epilepsy. Extrapyramidal involvement, autonomic nerve dysfunction, and sleep impairment are important clinical symptoms of RTT. Candidate therapies for extrapyramidal signs including dopamine agonists and other agents have been tested, but an effective treatment has not been established. We speculated that ghrelin may play an important role in RTT's pathogenesis, based on our previous findings. Ghrelin, a 28-amino-acid peptide, exerts multiple physiological functions including a protective effect in an animal model of Parkinson disease.
[Methodology] We evaluated the past 10 years of clinical trials of grehlin including those for eating disorders, CHF, and chronic obstructive pulmonary disease. Based on this evaluation, we designed a pilot study. We selected ghrelin administered intravenously 3 µg/kg/dose 1/day for 3 days, and then maintained for 3 weeks in two patients with RTT. We selected severity score of RTT and the Dystonia Rating Scale, Visual Analog Scale, sleep diary, and biological markers. The ethical committee at Kurume University School of Medicine approved the present study.
[Results] Our pilot study appears to be a feasible design that will not result in serious adverse reactions in RTT patients, and the study design may be useful for evaluations of the clinical efficacy of grehlin treatment, including its effects on extrapyramidal symptoms, autonomic and sleep dysfunction.
[Conclusion] Ghrelin treatment may be a new therapeutic approach for the clinical symptoms and signs of RTT patients.
[Methodology] We evaluated the past 10 years of clinical trials of grehlin including those for eating disorders, CHF, and chronic obstructive pulmonary disease. Based on this evaluation, we designed a pilot study. We selected ghrelin administered intravenously 3 µg/kg/dose 1/day for 3 days, and then maintained for 3 weeks in two patients with RTT. We selected severity score of RTT and the Dystonia Rating Scale, Visual Analog Scale, sleep diary, and biological markers. The ethical committee at Kurume University School of Medicine approved the present study.
[Results] Our pilot study appears to be a feasible design that will not result in serious adverse reactions in RTT patients, and the study design may be useful for evaluations of the clinical efficacy of grehlin treatment, including its effects on extrapyramidal symptoms, autonomic and sleep dysfunction.
[Conclusion] Ghrelin treatment may be a new therapeutic approach for the clinical symptoms and signs of RTT patients.