UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania.
Department of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania.
Clin Cancer Res. 2024 May 1;30(9):1724-1732. doi: 10.1158/1078-0432.CCR-22-2241.
The field of oncology has been transformed by immune checkpoint inhibitors (ICI) and other immune-based agents; however, many patients do not receive a durable benefit. While biomarker assessments from pivotal ICI trials have uncovered certain mechanisms of resistance, results thus far have only scraped the surface. Mechanisms of resistance are as complex as the tumor microenvironment (TME) itself, and the development of effective therapeutic strategies will only be possible by building accurate models of the tumor-immune interface. With advancement of multi-omic technologies, high-resolution characterization of the TME is now possible. In addition to sequencing of bulk tumor, single-cell transcriptomic, proteomic, and epigenomic data as well as T-cell receptor profiling can now be simultaneously measured and compared between responders and nonresponders to ICI. Spatial sequencing and imaging platforms have further expanded the dimensionality of existing technologies. Rapid advancements in computation and data sharing strategies enable development of biologically interpretable machine learning models to integrate data from high-resolution, multi-omic platforms. These models catalyze the identification of resistance mechanisms and predictors of benefit in ICI-treated patients, providing scientific foundation for novel clinical trials. Moving forward, we propose a framework by which in silico screening, functional validation, and clinical trial biomarker assessment can be used for the advancement of combined immunotherapy strategies.
肿瘤学领域已经发生了变革,免疫检查点抑制剂(ICI)和其他免疫疗法药物已经问世;然而,许多患者并未从中获得持久的获益。虽然关键性 ICI 试验中的生物标志物评估揭示了某些耐药机制,但迄今为止,这些研究结果只是冰山一角。耐药机制与肿瘤微环境(TME)本身一样复杂,只有通过构建精确的肿瘤-免疫界面模型,才能开发出有效的治疗策略。随着多组学技术的进步,现在可以对 TME 进行高分辨率的特征描述。除了对肿瘤进行批量测序外,现在还可以同时测量和比较对 ICI 有反应者和无反应者的单细胞转录组、蛋白质组和表观基因组数据以及 T 细胞受体谱。空间测序和成像平台进一步扩展了现有技术的维度。计算和数据共享策略的快速发展使具有生物学可解释性的机器学习模型得以开发,从而整合来自高分辨率、多组学平台的数据。这些模型促进了对 ICI 治疗患者耐药机制和获益预测因子的识别,为新的临床试验提供了科学基础。展望未来,我们提出了一个框架,通过该框架可以进行计算机筛选、功能验证和临床试验生物标志物评估,从而推进联合免疫治疗策略。