Parkinson's Disease Research Laboratory, Department of Neurology, Baylor College of Medicine, NB205, One Baylor Plaza, Houston, TX 77030, USA.
J Neural Transm (Vienna). 2013 Jan;120(1):197-210. doi: 10.1007/s00702-012-0840-9. Epub 2012 Jun 26.
Parkinson's disease (PD), the second most common neurodegenerative disorder, is characterized by the loss of dopamine (DA) neurons in the substantia nigra (SN). Currently, there are numerous therapeutic drugs for the treatment of PD; however, they are limited in efficacy and primarily target motor symptoms. Furthermore, these drugs have various adverse effects after long-term use. Usually, PD patients begin to take anti-parkinsonian drugs when they have developed obvious motor symptoms. At that time, a significant portion of the DA neurons in SN has been lost and the biology of the disease may have already been present for over a decade. This stage of PD diagnosis underscores the need for biomarkers that accurately indicate the onset of PD in order to apply disease-modifying therapies at an earlier stage of disease. However, development of disease modifying drugs has faced many setbacks, mostly due to the ways in which clinical trials are planned and executed. In this review paper, we summarize the recent findings of genetic biomarkers such as SNCA, LRRK2, parkin, PINK1, DJ1, etc., as well as evaluate the imaging techniques such as single proton emission computed tomography and positron emission tomography for their potential in diagnosing PD at earlier stages. Clinical trial designs, along with a comprehensive analysis of neuroprotective drugs for future treatment of PD, are also reviewed.
帕金森病(PD)是第二常见的神经退行性疾病,其特征是黑质(SN)中多巴胺(DA)神经元的丧失。目前,有许多治疗 PD 的治疗药物;然而,它们的疗效有限,主要针对运动症状。此外,这些药物长期使用后会有各种不良反应。通常,PD 患者在出现明显运动症状时开始服用抗帕金森病药物。此时,SN 中的大部分 DA 神经元已经丢失,疾病的生物学可能已经存在了十多年。PD 的这一诊断阶段强调需要生物标志物来准确指示 PD 的发病,以便在疾病的早期阶段应用疾病修饰疗法。然而,疾病修饰药物的开发遇到了许多挫折,主要是由于临床试验的计划和执行方式。在这篇综述论文中,我们总结了 SNCA、LRRK2、parkin、PINK1、DJ1 等遗传生物标志物的最新发现,并评估了单光子发射计算机断层扫描和正电子发射断层扫描等成像技术在早期诊断 PD 方面的潜力。还回顾了临床试验设计以及对 PD 未来治疗的神经保护药物的综合分析。