Clinical Center for Neuroprotection in Parkinson's Disease

描述(申请人提供)：帕金森氏病是一种原因不明的不可抗拒的进行性疾病，其实质黑质神经元进行性退化，导致大脑多巴胺缺乏的程度越来越严重。虽然已经开发了一些改善神经化学缺陷的治疗方法，但还没有证据表明治疗方法可以改善神经元的退化。这种神经保护作用是非常可取的，因为如果能够实现这一点，就可以显著延缓患者的临床恶化。这里概述的提案旨在在德克萨斯大学西南医学中心建立一个神经保护研究临床中心，该中心将与全国其他多达42个此类中心合作，开发和执行一项大规模临床试验，以测试一种或多种潜在的帕金森氏症神经保护剂。 德克萨斯州达拉斯的德克萨斯大学西南医学中心的运动障碍临床中心非常适合成为这样的临床中心。它是北得克萨斯州的主要学术转介中心，其两名工作人员神经学家在帕金森氏病临床试验的设计、实施和报告方面经验丰富。一旦指定了临床中心，将首先进行一系列试点研究，以确定最有前途的一种或多种药物。随后，将开发并进行一项涉及数千名患者的大型试验，以确定所选择的药物是否真的能够减缓这种疾病的进展。通过在严格失明和受控的条件下研究如此大量的患者，应该可以确定即使是对疾病进展的微小有益影响，但这仍然是非常重要的，不仅对于延缓疾病进展的影响，而且因为观察这种影响可能会深入了解这种常见和致残疾病的潜在发病机制。

DESCRIPTION (provided by applicant): Parkinson's disease is an inexorably progressive disorder of unknown cause in which neurons of the substantial nigra progressively degenerate resulting in ever-greater degrees of brain dopamine deficiency. While a number of treatments have been developed that improve the neurochemical deficit, no treatment has been demonstrated to ameliorate the neuronal deterioration. Such a neuroprotective effect is highly desirable because if this could be achieved, a significant delay in clinical deterioration of patients could be realized. The proposal outlined here aims to establish a Clinical Center for neuroprotective research at UT Southwestern Medical Center which will cooperate with up to 42 other such centers around the country in the development and execution of a large-scale clinical trial to test one or more potentially neuroprotective agents for Parkinson's disease. The Clinical Center for Movement Disorders at UT Southwestern Medical Center in Dallas, TX is well suited to becoming such a Clinical Center. It is the primary academic referral center for North Texas and its two staff neurologists are experienced in the design, conduct, and reporting of clinical trials in Parkinson's disease. Once the Clinical Centers are designated, a series of pilot studies will be conducted first to identify the most promising agent or agents. Subsequently, a large trial involving several thousand patients will be developed and conducted to establish whether the chosen agent is actually capable of slowing down the progression of this disease. By studying such a large number of patients under rigorously blinded and controlled conditions, it should be possible to identify even a small beneficial effect on disease progression which would nevertheless be very important, not only for the implications on retarding disease progression, but also because observing such an effect might yield insights into the underlying pathogenesis of this common and disabling disease.