改善并加速针对神经系统疾病的药物研发。
Improving and accelerating drug development for nervous system disorders.
作者信息
Pankevich Diana E, Altevogt Bruce M, Dunlop John, Gage Fred H, Hyman Steve E
机构信息
Forum on Neuroscience and Nervous System Disorders, Institute of Medicine, Washington, DC 20001, USA.
Forum on Neuroscience and Nervous System Disorders, Institute of Medicine, Washington, DC 20001, USA.
出版信息
Neuron. 2014 Nov 5;84(3):546-53. doi: 10.1016/j.neuron.2014.10.007.
Advances in the neurosciences have placed the field in the position where it is poised to significantly reduce the burden of nervous system disorders. However, drug discovery, development, and translation for nervous system disorders still pose many unique challenges. The key scientific challenges can be summarized as follows: mechanisms of disease, target identification and validation, predictive models, biomarkers for patient stratification and as endpoints for clinical trials, clear regulatory pathways, reliability and reproducibility of published data, and data sharing and collaboration. To accelerate nervous system drug development, the Institute of Medicine's Forum on Neuroscience and Nervous System Disorders has hosted a series of public workshops that brought together representatives of industry, government (including both research funding and regulatory agencies), academia, and patient groups to discuss these challenges and offer potential strategies to improve the translational neuroscience.
神经科学的进展使该领域有望大幅减轻神经系统疾病的负担。然而，神经系统疾病的药物发现、开发及转化仍面临许多独特挑战。关键的科学挑战可概括如下：疾病机制、靶点识别与验证、预测模型、用于患者分层及作为临床试验终点的生物标志物、明确的监管途径、已发表数据的可靠性和可重复性，以及数据共享与合作。为加速神经系统药物开发，美国国家医学院神经科学与神经系统疾病论坛举办了一系列公开研讨会，汇集了行业、政府（包括研究资助机构和监管机构）、学术界及患者群体的代表，共同探讨这些挑战，并提供改善转化神经科学的潜在策略。
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