Developing Medications for Nicotine Cessation

描述（由申请人提供）：烟草使用是可预防疾病、残疾和死亡的主要原因。尽管成瘾占大脑相关疾病的40%以上，但缺乏创新的治疗方法，而且制药和生物技术行业对开发新的治疗方法几乎没有兴趣。必须采取创新的方法来开发更有效的治疗尼古丁戒烟的药物。我们提议的公私合作（PPP）将提供一种综合的方法，利用尼古丁依赖的最新研究发现，并将其转化为戒烟药物。该团队在将基础研究发现在短时间内转化为治疗成瘾的药物方面具有专业知识和良好的记录。通过与学术中心、研究机构、政府和私营公司建立合作伙伴关系，我们开发、实施和管理了一个产品开发管道。目前开发新化学实体的成本约为10亿美元，由于NIDA计划在5年内向公私合作实体承诺5000万美元，管理合伙人的关键职能之一将是通过合作伙伴关系利用资源筹集额外资金。在UH2规划阶段，该奖项将成立一个公私伙伴关系实体，作为一个“虚拟制药公司”，致力于开发尼古丁戒烟药物。到UH2阶段结束时，我们将建立董事会、顾问委员会，与顾问委员会专家和外部顾问协商建立产品开发管道，制定组织的运营，通过与学术中心和研究所、制药和生物技术公司、非政府组织、非营利组织建立合作伙伴关系。基金会以及与中国等国家的国际关系，在这些国家，吸烟的流行程度如此之高，被视为对该国健康和经济的重大威胁。在UH2规划阶段结束时，管理合作伙伴（MP）将准备在UH3阶段开始时实施产品开发管道。PPP的使命将是“以病人为中心，以药物为中心”。产品开发管道将有10个项目同时运作，以期开发两种尼古丁戒烟新药。开发过程的方法与学术实验室的方法有很大的不同，更倾向于线性的项目管理风格。策略将是专注于手头的项目，因为每个项目都需要一组独特的组件进行翻译。我们将从模型生物（如啮齿动物模型）、分子神经科学、系统神经科学、遗传学和临床研究的发现中获得丰富的基础和临床研究信息。我们已经建立了广泛的行业合作，与具有药物开发和临床试验专业知识的合同化学公司，生物技术和制药公司合作采购和合成化合物。目标将是通过加速基础研究发现走向临床的途径来开发治疗成瘾的药物。如果不与企业就知识产权、许可和投资进行合作，就不可能开发药物，因为这项工作需要大量资金和资源。盖洛中心、桑福德伯纳姆医学研究所和加州大学圣地亚哥分校已经证明了作为合作伙伴的记录，在开发新疗法的从实验室到床边的成功项目方面有经验。我们共同建立了一个药物开发项目，从成瘾的基础研究、临床前开发、药物发现和开发到人体临床试验。此外，通过知识产权许可和与生物技术和制药公司的联合开发关系开发技术。如果我们有机会，我们将开发一个多学科合作的公私产品开发伙伴关系，旨在通过应用虚拟制药方法开发尼古丁戒烟的新型药物，从分子转向药物。

DESCRIPTION (provided by applicant): Tobacco use is the leading cause of preventable disease, disability, and death. Despite the fact that addiction represents more than 40% of brain-related illnesses, there is a dearth of innovative treatments, and there is little interest in developing new treatments in the pharmaceutical and biotechnology industry. It is essential that an innovative approach is taken to develop more effective medications for the treatment of nicotine cessation. Our proposed public private partnership (PPP) will provide a comprehensive approach that exploits the latest research discoveries in nicotine dependence and translates this into medications for nicotine cessation. The assembled team has expertise and a demonstrated track record in translating basic research discoveries into medications for addiction in short time-frames. We have developed, implemented and managed a product development pipeline by establishing collaborative partnerships with academic centers, research institutes, government, and private companies. The current cost for developing a new chemical entity is ~$1 billion and as NIDA plans to commit $50 million dollars over 5 years to a public private partnership entity, one of the critical functions of the managing partner will be to raise additional funds by leveraging resources through partnerships. During the UH2 planning phase of the award will form a public private partnership entity that will function as a "virtual pharmaceutical company" devoted to Developing Nicotine Cessation Medicines. By the end of the UH2 phase, we will have established a Board of Directors, advisory boards, established the product development pipeline in consultation with experts in the advisory boards and outside consultants, developed the operations of the organization, establish partnerships via letters of understanding with academic centers and Institutes, pharmaceutical and biotechnology companies, non-government organizations, not-for profit organizations, Foundations and international relations with countries such as China, where the prevalence of smoking is so great it is considered a significant health and economic threat for the country. At the end of the UH2 planning phase the managing partner (MP) will be ready to implement the product development pipeline at the beginning of the UH3 phase. The mission of the PPP will be "patient focused and medicines centered". The product development pipeline will have 10 projects operating simultaneously with the view of developing two new medicines for nicotine cessation. The development process differs significantly in its approach from that of the academic lab and favors a more linear, project management style. The strategy will be to focus on the project at hand, as each project requires a unique array of components for translation. We will draw on the wealth of information from basic and clinical research generated from model organisms such as rodent models, discoveries in molecular neuroscience, systems neuroscience, genetics and clinical research. We have established extensive industry collaborations to source and synthesize compounds using contract chemical companies, biotechnology and pharmaceutical companies that have drug development and clinical trial expertise. The goal will be to develop medicines for addiction by accelerating the pathway of basic research discoveries to the clinic. It is not possible to develop medicines without collaborating with business for intellectual property, licensing and investment as this endeavor needs significant funds and resources. The Gallo Center, the Sanford Burnham Medical Research Institute and University of California San Diego have demonstrated track record as collaborative partners, have experience with a successful bench-to-bedside program for the development of novel therapeutics. Together we have established a medications development program that extends from basic research in addiction, preclinical development, drug discovery and development to human clinical trials. Furthermore, technologies are developed through intellectual property licensing and joint development relationships with biotech and pharmaceutical companies. If we are given the opportunity, we will develop a multidisciplinary collaborative public private product development partnership that aims to move from molecules to medicines by applying a virtual pharma approach to develop novel medications for nicotine cessation. PUBLIC HEALTH RELEVANCE: Despite the fact that by 2030 around 10 million people a year will die of smoking-related illness, there is a dearth of innovative treatments, and relatively little interest in developing new treatments in the pharmaceutical and biotechnology industry. We will develop a multidisciplinary collaborative public private product development partnership that aims to move from molecules to medicines by applying a virtual pharma approach to develop novel medications for nicotine cessation.