Center for Translational Research in Neurodegenerative Disease, Department of Neuroscience, McKnight Brain Institute, College of Medicine, University of Florida, Gainesville, Florida 32610, USA.
J Clin Invest. 2013 May;123(5):1847-55. doi: 10.1172/JCI66029. Epub 2013 May 1.
Many neurodegenerative disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, and frontotemporal dementia, are proteinopathies that are associated with the aggregation and accumulation of misfolded proteins. While remarkable progress has been made in understanding the triggers of these conditions, several challenges have hampered the translation of preclinical therapies targeting pathways downstream of the initiating proteinopathies. Clinical trials in symptomatic patients using therapies directed toward initiating trigger events have met with little success, prompting concerns that such therapeutics may be of limited efficacy when used in advanced stages of the disease rather than as prophylactics. Herein, we discuss gaps in our understanding of the pathological processes downstream of the trigger and potential strategies to identify common features of the downstream degenerative cascade in multiple CNS proteinopathies, which could potentially lead to the development of common therapeutic targets for multiple disorders.
许多神经退行性疾病,包括阿尔茨海默病、帕金森病、亨廷顿病和额颞叶痴呆,都是与错误折叠蛋白的聚集和积累相关的蛋白病。尽管在理解这些疾病的触发因素方面已经取得了显著进展,但仍有几个挑战阻碍了针对起始蛋白病下游途径的临床前治疗的转化。针对起始触发事件的治疗方法在有症状的患者中进行的临床试验收效甚微,这引发了人们的担忧,即当这些治疗方法在疾病的晚期而不是预防性使用时,其疗效可能有限。在此,我们讨论了我们对触发事件下游病理过程的理解存在的差距,以及确定多种中枢神经系统蛋白病下游退行性级联反应的共同特征的潜在策略,这可能为多种疾病的共同治疗靶点的开发提供依据。