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

美国国立卫生研究院的使命是“…”寻求有关生命系统的性质和行为的基本知识，并应用这些知识来增强健康、延长寿命、减少疾病和残疾。实现这一目标需要科学家不仅能够产生这种基本知识，而且还能够推动将这种知识成功地转化为治疗干预措施，以减少疾病和残疾。拟议的为期两年的培训计划的目标是每年为一名博士后和一名博士后/临床研究员提供将基础科学发现转化为临床实践所需的翻译过程方面的正规教育。该计划将利用犹他大学现有的强大的翻译培训机会，并将以新的计划补充这些机会。具体地说，学员将受益于：1)每月举办一系列研讨会，在将药品、设备和应用程序从发现转移到临床使用的过程中提供正式教育；2)在诊所获得实质性经验，在那里可以看到与学员研究相关的疾病患者，学员将与他们一起参与诊所和患者支持小组，以更好地了解患者的需求；以及3)实习，以获得将产品转移到临床使用所需的监管要求和实践的第一手知识。受训人员将在一个翻译指导小组的指导下进行他们的初步研究，该小组由一名基础科学家、一名临床医生/临床科学家和一名具有将基础研究成果转化为临床经验的研究人员组成。在与指导团队协商后，学员将使用最佳实践来设计和进行临床前研究，以增强其工作的翻译潜力。培训结束后，学员将能够：1)描述在设计基础、临床前研究时要考虑的问题，并决定是否在治疗剂、设备或APP的开发方面推进临床前研究；2)将这些考虑因素的知识应用到他们的研究方案的制定中；3)评估其他人关于翻译考虑的研究方案；以及4)通过包含适当翻译原则的研究和出版物创造新的知识。总而言之，这些经验将培养出新兴的“专业翻译人员”，以促进基础研究成果的翻译，以改善患者的治疗结果。

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.