Training in the Development of Novel Interventions for the Treatment of Neurological and Neurobehavioral Disorders

NIH的使命是“.实现这一目标需要科学家不仅能够产生基本知识，而且能够将这些知识成功转化为治疗干预措施，以减少疾病和残疾。拟议的两年期培训计划的目标是每年为一名博士前和一名博士后/临床研究员提供将基础科学发现转化为临床实践所需的转化过程的正规教育。该计划将利用犹他州大学现有的强大翻译培训机会，并将通过新的编程来补充这些机会。具体而言，学员将受益于：1）一系列每月研讨会，在将制药，设备和应用程序从发现到临床使用的过程中提供正规教育; 2）临床实践经验，其中，与受训者的研究相关的疾病患者被看到，受训者将与他们一起参与诊所和患者支持小组，以更深入地了解病人;和3）实习，以获得第一手的监管要求和实践所需的移动产品通过临床使用的知识。学员将在由基础科学家，临床医生/临床科学家和具有将基础研究发现转化为临床经验的研究人员组成的翻译监督团队的指导下进行主要研究。在与该指导团队协商后，学员将使用最佳实践设计和进行临床前研究，以提高其工作的转化潜力。在培训结束时，参与者将能够：1）描述在设计基础临床前研究时需要考虑的问题，并决定是否在开发治疗药物、器械或应用程序方面推进临床前研究; 2）将这些考虑因素的知识应用于研究提案的开发; 3）评估其他人关于翻译考虑的研究建议; 4）通过纳入适当翻译原则的研究和出版物创造新知识。总之，这些经验将发展新兴的“专业翻译”，以促进基础研究成果的翻译，以改善患者的治疗效果。

The mission of the NIH is to “…seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability.” Achieving this end requires scientists who are not only capable of generating that fundamental knowledge, but also driving the successful translation of that knowledge into therapeutic interventions to reduce illness and disability. The objective of the proposed two-year training program is to provide one pre-doctoral and one post- doctoral/clinical fellow per year with formal education in the translational processes needed for moving basic science discoveries to clinical practice. The program will capitalize on strong translational training opportunities already available at the University of Utah and will complement those opportunities with new programming. Specifically, trainees will benefit from: 1) a series of monthly workshops delivering formal education in the process of moving pharmaceutics, devices, and apps from discovery to clinical use; 2) substantive experiences in clinics, in which patients with disorders of relevance to the trainee’s research are seen and with whom the trainee will engage in the clinic and in patient support groups to gain greater insight into the needs of patients; and 3) an internship to gain first-hand knowledge of the regulatory requirements and practices required to move products through to clinical use. Trainees will conduct their primary research under the direction of a translational supervisory team consisting of a basic scientist, a clinician/clinical scientist, and a researcher with experience in translating basic research discoveries into the clinic. In consultation with this mentoring team, the trainee will design and conduct preclinical research using best practices for enhancing the translational potential of their work. At the end of this training, participants will be able to: 1) Describe the issues to be considered in designing basic, preclinical studies and making decisions about whether to move a preclinical study forward with respect to development of a therapeutic agent, device, or app; 2) Apply knowledge of these considerations to the development of their research proposal; 3) Evaluate the research proposals of others with respect to translational considerations; and 4) Create new knowledge through research and publications that incorporate appropriate translational principles. Together, these experiences will develop burgeoning “professional translators” poised to promote the translation of basic research findings to improved therapeutic outcomes in patients.