Department of Biomedical Engineering, Columbia University, New York, New York, USA.
HM CINAC, Hospital Universitario HM Puerta del Sur, Móstoles, Madrid, Spain.
Mov Disord. 2019 Sep;34(9):1252-1261. doi: 10.1002/mds.27804. Epub 2019 Jul 30.
Parkinson's disease has many symptomatic treatments, but there is no neuroprotective therapy currently available. The evolution of this disease is inexorably progressive, and halting or stopping the neurodegenerative process is a major unmet need. Parkinson's disease motor features at onset are typically limited to 1 body segment, that is, focal signs, and the nigrostriatal degeneration is highly asymmetrical and mainly present in the caudal putamen. Thus, clinically and neurobiologically the process is fairly limited early in its evolution. Tentatively, this would allow the possibility of intervening to halt neurodegeneration at the most vulnerable site. The recent use of new technologies such as focused ultrasound provides interesting prospects. In particular, the possibility of transiently opening the blood-brain barrier to facilitate penetrance of putative neuroprotective agents is a highly attractive approach that could be readily applied to Parkinson's disease. However, because there are currently effective treatments available (ie, dopaminergic pharmacological therapy), more experimental evidence is needed to construct a feasible and practical therapeutic approach to be tested early in the evolution of Parkinson's disease patients. In this review, we provide the current evidence for the application of blood-brain barrier opening in experimental models of Parkinson's disease and discuss its potential clinical applicability. © 2019 International Parkinson and Movement Disorder Society.
帕金森病有许多对症治疗方法,但目前尚无神经保护疗法。该疾病的发展是不可避免的进行性的,阻止或停止神经退行性过程是一个主要的未满足的需求。帕金森病运动特征在发病时通常局限于 1 个身体部位,即局灶性体征,黑质纹状体变性高度不对称,主要存在于尾状壳核。因此,在疾病演变的早期,临床上和神经生物学上的病变相当有限。这可能允许在最脆弱的部位进行干预以阻止神经退行性变。最近使用新技术,如聚焦超声,提供了有趣的前景。特别是,暂时打开血脑屏障以促进潜在神经保护剂的渗透的可能性是一种极具吸引力的方法,可容易地应用于帕金森病。然而,由于目前有有效的治疗方法(即多巴胺能药物治疗),因此需要更多的实验证据来构建一种可行的、实用的治疗方法,以便在帕金森病患者的疾病早期进行测试。在这篇综述中,我们提供了血脑屏障开放在帕金森病实验模型中的应用的现有证据,并讨论了其潜在的临床适用性。© 2019 国际帕金森病和运动障碍协会。