Neuroscience Blueprint: Neurotherapeutics Grand Challenge

美国国立卫生研究院神经科学研究蓝图为应对神经系统疾病缺乏有效治疗而设立了神经治疗学的重大挑战。该计划旨在通过临床第一阶段成功开发药物，并促进行业合作伙伴关系，使其充分发展。这一重大挑战的长期目标是为目前治疗不佳或无法治愈的神经系统疾病研制出至少一种新颖有效的药物。 通过基础研究确定的大多数有希望的化合物还不够像药物一样用于人体测试。在一种新的化学实体可以在临床环境中进行测试之前，它必须经过一系列化学优化过程，以提高效力、活性和药物相似性，并进行临床前安全性测试，以满足美国食品和药物管理局(FDA)为临床测试设定的标准。这些活动在很大程度上是制药业和合同研究组织的领域，基础研究人员通常不能获得必要的专业知识和资源。为了解决这个问题，NIH神经科学研究蓝图正在建立一个由具有丰富行业经验的合同服务提供商和顾问组成的虚拟制药网络，以支持NIH神经科学研究社区的药物开发。这一资助机会公告(FOA)正在向拥有小分子化合物的研究人员征集U01合作协议奖的申请，这些小分子化合物有可能在这个网络中发展成为临床候选化合物。 这个FOA和蓝图神经治疗虚拟药物网络是美国国立卫生研究院神经科学研究蓝图的倡议，它为16个参与的美国国立卫生研究院研究所、中心和支持神经系统(http://neuroscienceblueprint.nih.gov/blueprint_basics/about_blueprint.htm).研究的办公室的合作活动提供了一个框架通过汇集资源和专业知识，NIH神经科学研究蓝图利用规模经济，面对对任何一个研究所或中心都太大的挑战，并开发服务于整个神经科学界的研究工具和基础设施。

The NIH Blueprint for Neuroscience Research established the Grand Challenge for Neurotherapeutics in response to the paucity of effective treatments for disorders of the nervous system. This program intends to develop drugs successfully through clinical Phase I and facilitate industry partnerships for their full development. The long-term goal of this grand challenge is to produce at least one novel and effective medication for a disorder of the nervous system that is currently poorly treated or untreatable. Most promising compounds identified through basic research are not sufficiently drug-like for human testing. Before a new chemical entity can be tested in a clinical setting, it must undergo a process of chemical optimization to improve potency, activity and drug-likeness and pre-clinical safety testing to meet the standards set by the Food and Drug Administration (FDA) for clinical testing. These activities are largely the domain of the pharmaceutical industry and contract research organizations, and the necessary expertise and resources are not commonly available to basic researchers. To address this problem, the NIH Blueprint for Neuroscience Research is establishing a ¿virtual pharma¿ network of contract service providers and consultants with extensive industry experience to enable drug development in the NIH neuroscience research community. This Funding Opportunity Announcement (FOA) is soliciting applications for U01 cooperative agreement awards from investigators with small molecule compounds that have potential for development into clinical candidates within this network. This FOA and the Blueprint Neurotherapeutics ¿virtual pharma¿ network is an initiative of the NIH Blueprint for Neuroscience Research, which provides a framework for collaborative activities involving sixteen participating NIH Institutes, Centers, and Offices that support research on the nervous system (http://neuroscienceblueprint.nih.gov/blueprint_basics/about_blueprint.htm). By pooling resources and expertise, the NIH Blueprint for Neuroscience Research takes advantage of economies of scale, confronts challenges too large for any single Institute or Center, and develops research tools and infrastructure that serve the entire neuroscience community.