Clinical Center for Neuroprotection in Parkinson's Disease

描述（由申请人提供）：帕金森病是一种不可避免的进行性疾病，原因不明，其中实质性黑质神经元进行性退化，导致大脑多巴胺缺乏症的程度越来越大。虽然已经开发了许多改善神经化学缺陷的治疗方法，但没有一种治疗方法被证明可以改善神经元退化。这种神经保护作用是非常可取的，因为如果可以实现，可以实现患者临床恶化的显著延迟。这里概述的建议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，该中心将与全国多达42个这样的中心合作，开发和执行一项大规模的临床试验，以测试一种或多种潜在的帕金森病神经保护药物。

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.