Schneider J S, Roeltgen D P, Mancall E L, Chapas-Crilly J, Rothblat D S, Tatarian G T
Department of Pathology, Anatomy and Cell Biology,Thomas Jefferson University, Philadelphia, PA 19107, USA.
Neurology. 1998 Jun;50(6):1630-6. doi: 10.1212/wnl.50.6.1630.
BACKGROUND/OBJECTIVE: Studies in animal models of Parkinson's disease (PD) suggest that GM1 ganglioside treatment can restore neurologic and dopaminergic function. In view of positive preclinical findings and the results of a previous open-label study demonstrating efficacy of GM1 in PD patients, this study compared effects of GM1 ganglioside and placebo on motor functions in PD patients.
Forty-five patients with mild to moderate PD were studied. The primary efficacy measure was change in the Unified Parkinson's Disease Rating Scale (UPDRS) motor score. After three independent baseline assessments, patients received IV infusion of the test drug (1,000 mg GM1 or placebo) and then self-administered either GM1 or placebo twice daily (200 mg/day, subcutaneously) for 16 weeks. Patients were examined during monthly follow-up visits.
There was a significant difference between groups in UPDRS motor scores at 16 weeks (p=0.0001). The activities of daily living portion of the UPDRS (off-period assessment) also showed a significant effect in favor of the GM1-treated patients (p=0.04). GM1-treated patients also had significantly greater mean improvements than placebo-treated patients in performance of timed motor tests including tests of arm, hand, and foot movements, and walking. GM1 was well tolerated and no serious adverse events were reported.
This study demonstrates that GM1 ganglioside treatment enhances neurologic function significantly in PD patients. Further study is warranted to evaluate long-term effects of GM1 in PD patients and to elucidate further the mechanisms underlying patient improvements.
背景/目的：帕金森病（PD）动物模型研究表明，GM1神经节苷脂治疗可恢复神经和多巴胺能功能。鉴于临床前研究结果阳性，以及之前一项开放标签研究显示GM1对PD患者有效，本研究比较了GM1神经节苷脂和安慰剂对PD患者运动功能的影响。
对45例轻度至中度PD患者进行研究。主要疗效指标为统一帕金森病评定量表（UPDRS）运动评分的变化。经过三次独立的基线评估后，患者接受静脉输注试验药物（1000mg GM1或安慰剂），然后每日两次（200mg/天，皮下注射）自行服用GM1或安慰剂，持续16周。在每月的随访中对患者进行检查。
16周时，两组UPDRS运动评分有显著差异（p=0.0001）。UPDRS日常生活部分（关期评估）也显示对GM1治疗的患者有显著影响（p=0.04）。在包括手臂、手部和足部运动以及步行测试在内的定时运动测试中，GM1治疗的患者平均改善也显著大于安慰剂治疗的患者。GM1耐受性良好，未报告严重不良事件。
本研究表明，GM1神经节苷脂治疗可显著增强PD患者的神经功能。有必要进一步研究评估GM1对PD患者的长期影响，并进一步阐明患者改善的潜在机制。
