Department of Psychiatry & Biobehavioral Sciences, University of California, Los Angeles, California, 90095-1751.
CHDI Foundation, 6080 Center Drive, Suite 100, Los Angeles, California, 90045.
Pharmacol Res Perspect. 2017 Oct;5(5). doi: 10.1002/prp2.344.
Huntington's disease (HD) is an autosomal dominant, neurodegenerative disorder characterized by motor as well as nonmotor symptoms for which there is currently no cure. The Q175 mouse model of HD recapitulates many of the symptoms identified in HD patients including disruptions of the sleep/wake cycle. In this study, we sought to determine if the daily administration of the histamine-3 receptor (H3R) antagonist/inverse agonist 6-[(3-cyclobutyl-2,3,4,5-tetrahydro-1H-3-benzazepin-7-yl)oxy]-N-methyl-3-pyridinecarboxamide hydrochloride (GSK189254) would improve nonmotor symptoms in the Q175 line. This class of drugs acts on autoreceptors found at histaminergic synapses and results in increased levels of histamine (HA). HA is a neuromodulator whose levels vary with a daily rhythm with peak release during the active cycle and relatively lower levels during sleep. H3Rs are widely expressed in brain regions involved in cognitive processes and activation of these receptors promotes wakefulness. We administered GSK189254 nightly to homozygote and heterozygote Q175 mice for 4 weeks and confirmed that the plasma levels of the drug were elevated to a therapeutic range. We demonstrate that daily treatment with GSK189254 improved several behavioral measures in the Q175 mice including strengthening activity rhythms, cognitive performance and mood as measured by the tail suspension test. The treatment also reduced inappropriate activity during the normal sleep time. The drug treatment did not alter motor performance and coordination as measured by the challenging beam test. Our findings suggest that drugs targeting the H3R system may show benefits as cognitive enhancers in the management of HD.
亨廷顿病(HD)是一种常染色体显性、神经退行性疾病,其特征是运动和非运动症状,目前尚无治愈方法。HD 的 Q175 小鼠模型再现了许多在 HD 患者中发现的症状,包括睡眠/觉醒周期中断。在这项研究中,我们试图确定每日给予组氨酸 3 受体(H3R)拮抗剂/反向激动剂 6-[(3-环丁基-2,3,4,5-四氢-1H-3-苯并氮杂䓬-7-基)氧基]-N-甲基-3-吡啶甲酰胺盐酸盐(GSK189254)是否会改善 Q175 系的非运动症状。这类药物作用于存在于组胺能突触的自受体,导致组胺(HA)水平升高。HA 是一种神经调质,其水平随每日节律变化,在活动周期中峰值释放,在睡眠期间相对较低。H3R 在参与认知过程的大脑区域广泛表达,激活这些受体可促进觉醒。我们每晚向纯合子和杂合子 Q175 小鼠给予 GSK189254 治疗 4 周,并证实药物的血浆水平升高至治疗范围。我们证明,每日给予 GSK189254 治疗可改善 Q175 小鼠的几种行为测量,包括增强活动节律、认知表现和通过悬尾试验测量的情绪。该治疗还减少了正常睡眠时间内的不当活动。药物治疗并未改变运动表现和协调,如通过挑战性横梁试验测量的那样。我们的发现表明,靶向 H3R 系统的药物可能作为 HD 管理中的认知增强剂显示出益处。