Sobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology, Queen Square, London WC1N 3BG, UK.
Parkinsons Dis. 2011;2011:804041. doi: 10.4061/2011/804041. Epub 2011 Dec 27.
Neural cell transplantation and gene therapy have attracted considerable interest as promising therapeutic alternatives for patients with Parkinson's disease (PD). Preclinical and open-label studies have suggested that grafted fetal neural tissue or viral vector gene transfer can achieve considerable biochemical and clinical improvements, whereas subsequent double-blind, placebo-controlled protocols have produced rather more modest and variable results. Detailed evaluation of these discordant findings has highlighted several crucial issues such as patient selection criteria, details surrounding transplantation or gene therapy methodologies, as well as the study designs themselves that ought to be carefully considered in the planning phases of future clinical trials. Beyond the provision of symptomatic efficacy and safety data, it also remains to be identified whether the possibilities offered by stem cell and gene therapy technological advances might translate to meaningful neuroprotection and/or disease-modifying effects or alleviate the nonmotor aspects of PD and thus offer additional benefits beyond those achieved through conventional pharmacotherapy or deep brain stimulation (DBS).
神经细胞移植和基因治疗作为治疗帕金森病（PD）的有前途的治疗方法引起了相当大的关注。临床前和开放性研究表明，移植的胎儿神经组织或病毒载体基因转移可以实现相当大的生化和临床改善，而随后的双盲、安慰剂对照方案产生的结果则更为温和和多变。对这些不一致发现的详细评估强调了几个关键问题，例如患者选择标准、移植或基因治疗方法的细节，以及研究设计本身，这些都应该在未来临床试验的规划阶段仔细考虑。除了提供症状疗效和安全性数据之外，还需要确定干细胞和基因治疗技术进步所提供的可能性是否可以转化为有意义的神经保护和/或疾病修饰作用，或者减轻 PD 的非运动方面的影响，从而提供超越传统药物治疗或深部脑刺激（DBS）所获得的额外益处。
