Potential disease-modifying therapies for PD should be able to interfere with processes that initiate or perpetuate neurodegenerative signals, stabilize injured or dying dopamine neurons, stimulate sprouting of new dopaminergic fibers and terminals, enhance the function of residual dopaminergic neurons and/or stimulate or maintain compensatory processes. One substance that has been suggested to potentially fulfill a number of these functions is the monosialoganglioside GM1 [5]. GM1 ganglioside is a normal constituent of nerve cell membranes, is known to modulate a number of cell surface and receptor activities and plays important roles in neuronal differentiation and development [6], protein phosphorylation [7], and synaptic function [8]. Treatment with GM1 following a number of different types of central nervous system lesions in experimental animals [9–14] has resulted in significant biochemical and behavioral recovery and pretreatment with GM1 inhibits damage resulting from a variety of neurotoxic exposures [15–17]. GM1 administration has been effective in ameliorating neurochemical and behavioral alterations in murine and non-human primate models of PD [13,18–20].

帕金森病情改善疗法可概括为启动或维持神经信号传导、稳定受损或濒死的多巴胺神经元，刺激多巴胺能纤维和末梢新生，使剩下的多巴胺能神经元的功能增强和/或刺激或维持代偿过程。有可能完成这些功能的一种物质便是单唾液酸神经节苷酯GM1[5]。GM1神经节苷酯是正常神经细胞膜的一种组成成分，已知可调节许多细胞表面和受体的活动，并且在神经分化和发育过程[8]、蛋白质磷酸化[7]和突触功能[8]中起到重要作用。在动物试验研究中，使用GM1治疗许多不同类型的中枢神经系统损伤[9-14]，结果表明，实验动物的生化指标和行为学具有显著的恢复。使用GM1进行预防性治疗，可抑制各种不同类型的神经毒性的侵害[15-17]。在鼠和非人灵长类的帕金森模型中，使用GM1治疗可改善神经化学物质和行为学上海翻译公司。[13,18-20]。

In a randomized double blind placebo controlled trial, GM1-treated PD patients showed significant improvements on Unified Parkinson's Disease Rating Scale (UPDRS) motor scores and performance of timed motor tasks, compared to baseline performance, assessed during a practically defined off-medication period [21]. UPDRS motor scores for placebo-treated patients in the same study remained essentially unchanged over the course of 16 weeks of study. At the conclusion of that study, patients were allowed the opportunity to participate in an open-extension trial with GM1 in order to assess the long-term safety and efficacy of GM1use by PD patients. The present report describes the

effects of long-termexposure to GM1 in these patients and supports the long-term safety and efficacy of this treatment.

在一项随机、双盲、安慰剂对照实验中，使用帕金森治疗的患者与基线相比，在帕金森统一评分量表（UPDRS）中的运动评分和定时运动评分这两项指标具有显著的改善[21]。在同一个研究中，使用安慰剂治疗的患者在16周研究期过后，UPDRS运动评分没有变化。该项研究的结论是：为了评估GM1治疗帕金森患者的长期安全性和有效性，患者有机会参加开放性的延伸实验。目前的报告表明，在这些患者中，长期使用GM1进行治疗是有效的，并肯定了这种疗法的长期安全性和有效性北京翻译公司。