Profectus Pharma Consulting Inc., San Jose, CA, United States.
Department of Pharmacology, Vertex Pharmaceuticals Inc., Cambridge, MA, United States.
Biochem Pharmacol. 2014 Jan 1;87(1):189-210. doi: 10.1016/j.bcp.2013.10.019. Epub 2013 Oct 30.
The translational sciences represent the core element in enabling and utilizing the output from the biomedical sciences and to improving drug discovery metrics by reducing the attrition rate as compounds move from preclinical research to clinical proof of concept. Key to understanding the basis of disease causality and to developing therapeutics is an ability to accurately diagnose the disease and to identify and develop safe and effective therapeutics for its treatment. The former requires validated biomarkers and the latter, qualified targets. Progress has been hampered by semantic issues, specifically those that define the end product, and by scientific issues that include data reliability, an overt reductionistic cultural focus and a lack of hierarchically integrated data gathering and systematic analysis. A necessary framework for these activities is represented by the discipline of pharmacology, efforts and training in which require recognition and revitalization.
转化科学是使生物医学科学的成果得以利用、提高药物发现的衡量标准的核心要素,其通过降低化合物从临床前研究到临床概念验证的淘汰率来实现这一目标。理解疾病因果关系的基础并开发治疗方法的关键是能够准确诊断疾病,并确定和开发用于治疗的安全有效的治疗方法。前者需要经过验证的生物标志物,后者需要合格的靶点。这些进展受到语义问题的阻碍,特别是那些定义最终产品的语义问题,还受到科学问题的阻碍,包括数据可靠性、明显的简化论文化焦点以及缺乏层次化集成的数据收集和系统分析。药理学学科代表了这些活动的必要框架,需要认识到并振兴药理学的努力和培训。