Schneider J S, Roeltgen D P, Rothblat D S, Chapas-Crilly J, Seraydarian L, Rao J
Department of Neurology, Hahnemann University, Philadelphia, PA 19102, USA.
Neurology. 1995 Jun;45(6):1149-54. doi: 10.1212/wnl.45.6.1149.
We performed an open-label study testing the effects of GM1 ganglioside on 10 Parkinson's disease (PD) patients. Patients received 1,000 mg of GM1 by IV infusion after the last of three baseline functional assessments. Patients then self-administered GM1 at a dose of 200 mg/d, by subcutaneous injection, for 18 weeks. Under these conditions, GM1 ganglioside proved to be safe and well tolerated. There were no serious adverse events and none of the patients developed elevated anti-GM1 antibody titers. Improvements on at least some functional measures were present in most patients, beginning after 4 to 8 weeks of GM1 treatment. When functional improvements occurred, they lasted for the duration of the study. These results suggest that GM1 ganglioside is well tolerated and may be a useful adjunct to the treatment of PD, and that a double-blind, placebo-controlled study is now warranted.
我们进行了一项开放标签研究，以测试神经节苷脂GM1对10名帕金森病（PD）患者的影响。在进行了三次基线功能评估中的最后一次评估后，患者通过静脉输注接受了1000毫克的GM1。然后，患者以200毫克/天的剂量通过皮下注射自行给药GM1，持续18周。在这些条件下，神经节苷脂GM1被证明是安全且耐受性良好的。没有严重不良事件发生，且没有患者出现抗GM1抗体滴度升高。大多数患者在GM1治疗4至8周后，至少在某些功能指标上出现了改善。当功能改善出现时，它们在研究期间持续存在。这些结果表明，神经节苷脂GM1耐受性良好，可能是PD治疗的一种有用辅助药物，并且现在有必要进行一项双盲、安慰剂对照研究。
