Barrett Jeffrey S
Laboratory for Applied PK/PD, Clinical Pharmacology & Therapeutics Division, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA.
AAPS J. 2008;10(1):9-14. doi: 10.1208/s12248-007-9002-7. Epub 2008 Feb 5.
Translational research is generally described as the application of basic science discoveries to the treatment or prevention of disease or injury. Its value is usually determined based on the likelihood that exploratory or developmental research can yield effective therapies. While the pharmaceutical industry has evolved into a highly specialized sector engaged in translational research, the academic medical research community has similarly embraced this paradigm largely through the motivation of the National Institute of Health (NIH) via its Roadmap initiative. The Clinical and Translational Science Award (CTSA) has created opportunities for institutions which can provide the multidisciplinary environment required to engage such research. A key component of the CTSA and an element of both the NIH Roadmap and the FDA Critical Path is the bridging of bench and bedside science via quantitative pharmacologic relationships. The infrastructure of the University of Pennsylvania/Children's Hospital of Philadelphia CTSA is highlighted relative to both research and educational objectives reliant upon quantitative pharmacology. A case study, NIH-sponsored research program exploring NK1r antagonism for the treatment NeuroAIDS is used to illustrate the application of quantitative pharmacology in a translational research paradigm.
转化研究通常被描述为将基础科学发现应用于疾病或损伤的治疗或预防。其价值通常根据探索性或开发性研究能否产生有效疗法的可能性来确定。虽然制药行业已发展成为从事转化研究的高度专业化部门,但学术医学研究界同样在很大程度上通过美国国立卫生研究院(NIH)的路线图计划的推动而接受了这一模式。临床与转化科学奖(CTSA)为那些能够提供开展此类研究所需的多学科环境的机构创造了机会。CTSA的一个关键组成部分以及NIH路线图和FDA关键路径的一个要素是通过定量药理学关系在实验室科学和临床科学之间架起桥梁。相对于依赖定量药理学的研究和教育目标,突出了宾夕法尼亚大学/费城儿童医院CTSA的基础设施。一个由NIH资助的探索NK1受体拮抗剂治疗神经艾滋病的研究项目的案例研究,用于说明定量药理学在转化研究模式中的应用。