ETHYL-EPA (MIRAXION) IN SUBJECTS WITH MILD TO MODERATE HUNTINGTON'S DISEASE (HD)

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。亨廷顿氏病（HD）是一种毁灭性的脑部疾病。HD通常在30至50岁之间发病，尽管很少会早或晚发病，并在15至25岁之间进展至死亡。亨廷顿舞蹈症是一种常染色体显性遗传的神经退行性疾病，由一种名为“亨廷顿”的基因CAG重复扩增引起。HD影响了大约30,000名美国人，其特点是运动、认知和精神障碍的潜伏发展，并不可避免地走向死亡。父母的每个孩子都有50%的风险会遗传这种疾病。乙基-EPA（乙基-二十碳五烯酸）是一种半合成的、高度纯化的脂肪酸EPA，它与一个乙基相连。乙基在肠道中从EPA中分离出来EPA被吸收到血液中。虽然乙基epa在HD中的确切作用机制尚不清楚，但它被认为可以稳定线粒体完整性并改善神经元功能障碍。这可能通过几种途径直接和/或间接发生，这些途径在临床前研究中已被证明受到EPA的影响。EPA在各种研究中都涉及到触发某些酶和调节因子表达的诱导，尽管这些研究与其作为HD治疗的潜在用途的确切相关性尚不清楚。该方案将比较安慰剂和乙基epa在亨廷顿病患者中的效果。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Huntington's disease (HD) is a devastating disease of the brain. HD usually begins between the ages of 30 to 50, although rarely it can begin earlier or later, and progresses to death within 15-25 years. HD is inherited as an autosomal dominant, neurodegenerative disorder caused by a CAG repeat expansion in a gene called "huntington". HD affects approximately 30,000 Americans and is characterized by insidious development of motor, cognitive and psychiatric disturbances with an inexorable progression towards death. There is a 50% risk that each child of a parent will inherit the disease. Ethyl-EPA (ethyl-eicosapentaenoic acid) is a semi-synthetic, highly purified form of the fatty acid EPA attached to an ethyl group. The ethyl group is cleaved from the EPA in the gut and the EPA is absorbed into the blood stream. Although the exact mechanism of action of ethyl-EPA in HD is not known, it is thought to stabilize mitochondrial integrity and improve neuronal dysfunction. This could occur directly and/or indirectly via several pathways, which have been shown to be affected by EPA in pre-clinical studies. EPA has been implicated in various studies to trigger induction of expression of certain enzymes and regulatory factors though the exact relevance of these studies to its potential use as a therapeutic in HD is not yet known. This protocol will compare placebo to the effect of ethyl-EPA in subjects with Huntington's disease.