Department of Pathology, Anatomy and Cell Biology, Thomas Jefferson University, Philadelphia, PA 19107, USA.
J Neurol Sci. 2010 May 15;292(1-2):45-51. doi: 10.1016/j.jns.2010.02.009. Epub 2010 Mar 5.
Previous work demonstrated that short-term (i.e., 16 weeks) use of GM1 ganglioside resulted in significant symptom reduction in Parkinson's disease (PD) patients. As GM1 use may have long-term benefit for PD patients, the present study was conducted to evaluate the long-term safety and efficacy of GM1 in PD patients. Twenty-six patients who concluded a previous randomized double blind placebo controlled trial of GM1 volunteered for this open-extension study. At the end of 5 years of GM1 use, patients generally had lower Unified Parkinson's Disease Rating Scale (UPDRS) motor scores (assessed during a practically defined "off" period) than at baseline prior to randomization into the original study. A similar result was found for UPDRS Activities of Daily Living scores. Performance of timed motor tests also remained mostly stable over the 5 year observation period. No consistent clinically significant changes in blood chemistry, hematologic indices or urine chemistry were noted over the course of this study. These results suggest that long-term GM1 use by PD patients is safe and may provide some clinical benefit for PD patients. Additional study is required to more completely assess the degree to which GM1 treatment may be a symptomatic and/or disease-modifying agent for treatment of PD.
先前的研究表明，GM1 神经节苷脂的短期（即 16 周）使用可显著减轻帕金森病（PD）患者的症状。由于 GM1 的使用可能对 PD 患者具有长期益处，因此进行了本研究以评估 GM1 在 PD 患者中的长期安全性和疗效。26 名完成了 GM1 先前随机双盲安慰剂对照试验的患者自愿参加了这项开放延伸研究。在 GM1 使用 5 年后，患者在“关闭”期（在实际定义的“关闭”期进行评估）的统一帕金森病评定量表（UPDRS）运动评分通常低于原始研究随机分组前的基线水平。日常生活活动评分（UPDRS-ADL）也有类似的结果。在 5 年的观察期间，定时运动测试的表现也基本保持稳定。在整个研究过程中，未发现血液化学，血液学指标或尿液化学的一致的临床上有意义的变化。这些结果表明，PD 患者长期使用 GM1 是安全的，并且可能为 PD 患者提供一些临床益处。需要进一步的研究来更全面地评估 GM1 治疗可能在多大程度上成为治疗 PD 的症状缓解剂和/或疾病修饰剂。
