Khakimova Gulnara R, Kozina Elena A, Kucheryanu Valerian G, Ugrumov Michael V
Laboratory of Neural and Neuroendocrine Regulations, Institute of Developmental Biology RAS, 26 Vavilov St, Moscow, 119334, Russia.
Laboratory of General Pathology of the Nervous System, Institute of General Pathology and Pathophysiology RAMS, 8 Baltiiskaya St, Moscow, 125315, Russia.
Mol Neurobiol. 2017 Jul;54(5):3618-3632. doi: 10.1007/s12035-016-9936-9. Epub 2016 May 19.
A crucial event in the pathogenesis of Parkinson's disease is the death of dopaminergic neurons of the nigrostriatal system, which are responsible for the regulation of motor function. Motor symptoms first appear in patients 20-30 years after the onset of the neurodegeneration, when there has been a loss of an essential number of neurons and depletion of compensatory reserves of the brain, which explains the low efficiency of treatment. Therefore, the development of a technology for the diagnosing of Parkinson's disease at the preclinical stage is of a high priority in neurology. In this study, we have developed at an experimental model a fundamentally novel for neurology approach for diagnosis of Parkinson's disease at the preclinical stage. This methodology, widely used for the diagnosis of chronic diseases in the internal medicine, is based on the application of a challenge test that temporarily increases the latent failure of a specific functional system, thereby inducing the short-term appearance of clinical symptoms. The provocation test was developed by a systemic administration of α-methyl-p-tyrosine (αMpT), a reversible inhibitor of tyrosine hydroxylase to MPTP-treated mice at the presymptomatic stage of parkinsonism. For this, we first selected a minimum dose of αMpT, which caused a decrease of the dopamine level in the striatum of normal mice below the threshold at which motor dysfunctions appear. Then, we found the maximum dose of αMpT at which a loss of dopamine in the striatum of normal mice did not reach the threshold level, and motor behavior was not impaired. We showed that αMpT at this dose induced a decrease of the dopamine concentration in the striatum of MPTP-treated mice at the presymptomatic stage of parkinsonism below a threshold level that results in the impairment of motor behavior. Finally, we proved that αMpT exerts a temporal and reversible influence on the nigrostriatal dopaminergic system of MPTP-treated mice with no long-term side effects on other catecholaminergic systems. Thus, the above experimental data strongly suggest that αMpT-based challenge test might be considered as the provocation test for Parkinson's disease diagnosis at the preclinical stage in the future clinical trials.
帕金森病发病机制中的一个关键事件是黑质纹状体系统多巴胺能神经元的死亡,该系统负责运动功能的调节。运动症状在神经退行性变开始20 - 30年后首次出现在患者身上,此时已经有相当数量的神经元丢失且大脑的代偿储备耗尽,这就解释了治疗效果不佳的原因。因此,开发一种在临床前期诊断帕金森病的技术是神经病学领域的高度优先事项。在本研究中,我们在实验模型上开发了一种神经病学领域全新的临床前期帕金森病诊断方法。这种在内科广泛用于诊断慢性病的方法基于激发试验的应用,该试验暂时增加特定功能系统的潜在功能障碍,从而诱发临床症状的短期出现。激发试验是通过在帕金森病症状前期对MPTP处理的小鼠全身给予α - 甲基 - 对 - 酪氨酸(αMpT)来开发的,αMpT是酪氨酸羟化酶的可逆抑制剂。为此,我们首先选择了能使正常小鼠纹状体中多巴胺水平降至运动功能障碍出现阈值以下的αMpT最小剂量。然后,我们找到了正常小鼠纹状体中多巴胺丢失未达到阈值水平且运动行为未受损的αMpT最大剂量。我们发现,该剂量的αMpT可使帕金森病症状前期MPTP处理小鼠纹状体中的多巴胺浓度降至导致运动行为受损的阈值水平以下。最后,我们证明αMpT对MPTP处理小鼠的黑质纹状体多巴胺能系统具有暂时且可逆的影响,对其他儿茶酚胺能系统无长期副作用。因此,上述实验数据强烈表明,基于αMpT的激发试验在未来临床试验中可能被视为临床前期帕金森病诊断的激发试验。