Cancer Pharmacology and Computational Medicine, Department of Medical Sciences, Uppsala University, Uppsala, Sweden.
Signals and Systems, Department of Electrical Engineering, Uppsala University, Uppsala, Sweden.
PLoS One. 2020 May 14;15(5):e0232989. doi: 10.1371/journal.pone.0232989. eCollection 2020.
Multi drug treatments are increasingly used in the clinic to combat complex and co-occurring diseases. However, most drug combination discovery efforts today are mainly focused on anticancer therapy and rarely examine the potential of using more than two drugs simultaneously. Moreover, there is currently no reported methodology for performing second- and higher-order drug combination analysis of secretomic patterns, meaning protein concentration profiles released by the cells. Here, we introduce COMBSecretomics (https://github.com/EffieChantzi/COMBSecretomics.git), the first pragmatic methodological framework designed to search exhaustively for second- and higher-order mixtures of candidate treatments that can modify, or even reverse malfunctioning secretomic patterns of human cells. This framework comes with two novel model-free combination analysis methods; a tailor-made generalization of the highest single agent principle and a data mining approach based on top-down hierarchical clustering. Quality control procedures to eliminate outliers and non-parametric statistics to quantify uncertainty in the results obtained are also included. COMBSecretomics is based on a standardized reproducible format and could be employed with any experimental platform that provides the required protein release data. Its practical use and functionality are demonstrated by means of a proof-of-principle pharmacological study related to cartilage degradation. COMBSecretomics is the first methodological framework reported to enable secretome-related second- and higher-order drug combination analysis. It could be used in drug discovery and development projects, clinical practice, as well as basic biological understanding of the largely unexplored changes in cell-cell communication that occurs due to disease and/or associated pharmacological treatment conditions.
多药物治疗越来越多地用于临床以对抗复杂和同时发生的疾病。然而,当今大多数药物组合发现工作主要集中在抗癌治疗上,很少检查同时使用两种以上药物的潜力。此外,目前还没有报道用于执行第二级和更高级别的细胞分泌组模式药物组合分析的方法学,这意味着细胞释放的蛋白质浓度谱。在这里,我们介绍 COMBSecretomics(https://github.com/EffieChantzi/COMBSecretomics.git),这是第一个专门设计的实用方法学框架,用于全面搜索候选治疗方法的第二级和更高级别的混合物,这些混合物可以修饰甚至逆转人类细胞功能失调的细胞分泌组模式。该框架具有两种新的无模型组合分析方法;最高单药原则的定制推广和基于自上而下层次聚类的数据挖掘方法。还包括消除异常值的质量控制程序和用于量化所得结果不确定性的非参数统计。COMBSecretomics 基于标准化可重复的格式,可以与提供所需蛋白质释放数据的任何实验平台一起使用。通过与软骨降解相关的药理学研究证明了其实际应用和功能。COMBSecretomics 是第一个能够实现与细胞分泌组相关的第二级和更高级别的药物组合分析的方法学框架。它可用于药物发现和开发项目、临床实践以及对细胞间通信的广泛探索性变化的基本生物学理解,这些变化是由于疾病和/或相关的药理治疗条件引起的。