Schork Nicholas J, Goetz Laura H
The Translational Genomics Research Institute, a part of the City of Hope National Medical Center, Phoenix, AZ (NJS, LHG), USA.
The City of Hope National Medical Center, Duarte, CA (NJS), USA.
Nat Commun. 2025 May 30;16(1):5024. doi: 10.1038/s41467-025-60395-z.
Precision medicines, or those medicines that are tailored to individual genetic, molecular, physiologic, behavioral, and/or exposure profiles, are being developed at a rapid pace. However, just how precise these interventions are in terms of their mechanisms of action (MOAs), clinical effects, and utility in different individuals, are hard anticipate with current preclinical research and clinical trials strategies. To understand how various genes, processes, organs, clinical phenotypes, etc. may be impacted by an intervention, as well as how many people might benefit from it, appropriate data on living human beings needs to be collected as part of built-for-purpose clinical trials.
精准药物,即那些根据个体的基因、分子、生理、行为和/或暴露特征量身定制的药物,正在迅速研发中。然而,就其作用机制(MOA)、临床效果以及在不同个体中的效用而言,这些干预措施究竟有多精准,很难通过当前的临床前研究和临床试验策略进行预测。为了了解各种基因、过程、器官、临床表型等可能如何受到一种干预的影响,以及有多少人可能从中受益,需要收集有关活体人类的适当数据,作为专门设计的临床试验的一部分。