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

靶向药物发现的新时代及发展路径：从分子开始

• 批准号: 8487400
• 负责人: Gabrielle Gold-von Simson
• 金额: $ 9.11万
• 依托单位: NEW YORK UNIVERSITY SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-07-01 至 2017-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8487400
• 关键词: Achievement; Address; Advisory Committees; Americas; Area; Back; Basic Science; Behavioral Research; Belief; Biomedical Research; Breathing; Businesses; Capital; Cardiovascular Diseases; Case Study; Clinical Research; Clinical Trials; Collaborations; Complications of Diabetes Mellitus; Cost Analysis; Data; Development; Diabetes Mellitus; Diabetic Nephropathy; Discipline; Disease; Doctor of Philosophy; Drug Monitoring; Drug Targeting; Economics; Educational Activities; Educational Curriculum; Educational Status; Educational process of instructing; Ensure; Ethics; Evaluation; Faculty; Failure; Future; Goals; Grant; Health; Health Policy; Individual; Industry; Investments; Knowledge; Legal patent; Logistics; Manuscripts; Marketing; Metric; Mission; Molecular; National Institute of Diabetes and Digestive and Kidney Diseases; Natural regeneration; Nature; Obesity; Outcome; Outcome Measure; Participant; Pathway interactions; Patient Care; Patient Self-Report; Patients; Performance; Pharmaceutical Preparations; Pharmacologic Substance; Phase; Press Releases; Process; Program Development; Public Health; Public Opinion; Qualitative Evaluations; Quantitative Evaluations; Recommendation; Regulation; Reporting; Research; Research Infrastructure; Research Personnel; Resources; Retinal Diseases; Rewards; Running; Safety; Schools; Scientist; Series; Shapes; Signal Pathway; Signal Transduction; Specific qualifier value; Stimulus; Students; Surveys; Task Performances; Technology; Therapeutic; Therapeutic Intervention; Training; Translating; Translational Research; United States National Institutes of Health; Work; Writing; base; bench to bedside; design; drug development; drug discovery; experience; falls; follow-up; health practice; improved; innovation; interest; meetings; multidisciplinary; news; novel; post-market; programs; public health relevance; research and development; skills; success; symposium; tool

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.

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。 {尽管针对糖尿病并发症（例如糖尿病肾病、视网膜病变和心血管疾病）患者的临床研究前景广阔，但制药公司一直不愿在这一重要领域进行药物开发。 NIDDK 已适时介入并采取了多项举措来支持糖尿病并发症的研究和药物开发。因此，我们的最终目标是教授研究生及以上水平的不同目标学习者}在他们的研究{和相关工作}中利用新颖的从实验室到临床的经验知识，重点关注糖尿病、{糖尿病并发症}和肥胖等特定的、共病的和普遍的公共卫生问题。 {我们将强调根据疾病期间目标变化设计治疗干预措施的重要性；例如，一些目标可能在启动时更相关，另一些目标在进展中更相关，而另一些则在回归和再生失败中更相关。} 强调将研究发现转化为患者护理的规划主题将是药物发现和开发。当前为期一年的课程系列回顾了秋季学期的 I-IV 期试验、上市后轨迹、安全监测、药物专利和经济刺激改革以及伦理和成本分析。春季学期，讨论成功和不成功药物的分子途径和科学基础。我们的初步经验表明，这两个课程部分（以及正在进行的教学研讨会系列）一起有助于打破个人不能直接致力于发明药物的限制性信念， 教育活动将广大学员的不同兴趣引向将基础科学发现转化为有用的患者护理工具的灵感和奖励。定性和定量报告工具以及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不同参与者提高 NIDDK 相关转化研究技能，最终改善患者护理和公共卫生。