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“……寻求关于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。”要实现这一目标，科学家不仅要有能力产生这些基础知识，还要将这些知识成功地转化为治疗干预措施，以减少疾病和残疾。这项为期两年的培训计划的目标是每年为一名博士预科生和一名博士后/临床研究员提供将基础科学发现转化为临床实践所需的正规教育。该项目将充分利用犹他大学已经提供的强大的翻译培训机会，并将以新的项目补充这些机会。具体来说，学员将受益于：1)一系列每月的研讨会，在将药物、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的教育；2)在诊所的实质性经验，在那里看到与实习生研究相关的疾病患者，实习生将与他们一起参与诊所和患者支持小组，以更深入地了解患者的需求；3)实习，以获得第一手的监管要求和实践，将产品通过临床使用所需的知识。受训者将在一个由一名基础科学家、一名临床医生/临床科学家和一名具有将基础研究发现转化为临床经验的研究人员组成的转化监督小组的指导下进行初步研究。在与该指导团队协商后，受训者将使用最佳实践来设计和开展临床前研究，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，并决定是否将临床前研究向前推进，以开发治疗剂、设备或应用程序；2)将这些考虑因素的知识应用于他们的研究计划的发展；3)根据翻译考虑，评估他人的研究建议；4)通过纳入适当翻译原则的研究和出版物创造新知识。这些经验将共同发展新兴的“专业翻译”，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.