A New Era of Targeted Drug Discovery and the Path of Development: From Molecular

描述（由申请人提供）：我们建议将一系列课程修改为高度相关的NIDDK教育活动。尽管在患有糖尿病并发症（例如糖尿病肾病、视网膜病变和心血管疾病）的患者中进行了有希望的临床研究，但制药公司一直不愿意在这一重要领域进行药物开发。NIDDK适时地介入了几项倡议，以支持糖尿病并发症的研究和药物开发。因此，我们的最终目标将是在研究生水平及以后教目标学习者的多样化群体，以利用他们的研究中新颖的板凳到床边的经验知识{和相关的努力}，重点是糖尿病，糖尿病并发症}和肥胖症，具体的，共病的，和普遍的公共卫生问题。{We将强调根据疾病期间的靶点变化设计治疗干预措施的重要性;例如，某些靶点可能在启动时更相关，其他靶点可能在进展中更相关，而其他靶点可能在消退和再生失败中更相关。强调将研究发现转化为患者护理的方案主题将是药物发现和开发。目前为期一年的课程系列回顾了第一阶段至第四阶段的试验，上市后的轨迹，安全监测，药物专利，经济刺激改革以及道德和成本分析在秋季学期。在春季学期，成功和不成功的药物的分子途径和科学基础进行了讨论。我们的初步经验已经确定，两个课程部分（和正在进行的教学研讨会系列）一起有助于消除个人不能直接致力于发明药物的限制性信念， 而教育活动则引导广大学员的不同兴趣，将基础科学发现转化为有用的病人护理工具，从而获得灵感和回报。定性和定量报告工具和绩效任务将评估该计划，以确保实现教育目标以及该计划的未来扩展。实现这些目标的进展将意味着提高不同参与者的NIDDK相关转化研究技能，最终改善患者护理和公共卫生。

DESCRIPTION (provided by applicant): We propose to modify a series of courses into a highly relevant NIDDK educational activity. {Despite promising clinical studies in patients with diabetic complications, such as diabetic nephropathy, retinopathy and cardiovascular disease as examples, pharmaceutical companies have been reluctant to pursue drug development in this important area. NIDDK has duly stepped in with several initiatives to support research and drug development for diabetic complications. As such, our ultimate goal will be to teach a diverse group of targeted learners at the graduate level and beyond} to harness novel bench-to-bedside empirical knowledge in their research {and related endeavors} with a focus on diabetes, {diabetic complications}, and obesity, specific, comorbid, and pervasive public health problems. {We will highlight the importance of designing therapeutic interventions based on target changes during the disease; for example, some targets may be more relevant in initiation, others more in progression, and some more in failure of regression and regeneration.} The programmatic theme that emphasizes translation of research discoveries to patient care will be drug discovery and development. The current yearlong course series reviews Phase I-IV trials, post- marketing trajectories, safety monitoring, drug patents, and economic stimulus reform as well as ethics and cost analysis in the fall semester. In the spring semester, the molecular pathways and scientific basis of successful and unsuccessful drugs are discussed. Our preliminary experience has established that together, the two course segments (and ongoing didactic seminar series) help to disabuse the limiting belief that individuals cannot directly work towards inventing drugs, and the educational activity directs the diverse interests of this wide range of trainees towards the inspirations and rewards of translating basic science discoveries into useful patient care tools. Qualitative and quantitative reporting tools and performance tasks will evaluate the program to assure achievement of educational goals as well as future expansion of the program. Progress in achieving these objectives would mean increased NIDDK-relevant translational research skills for the diverse participants to ultimately improve patient care and public health.