Marron Thomas U, Luke Jason J, Hoffner Brianna, Perlmutter Jane, Szczepanek Connie, Anagnostou Valsamo, Silk Ann W, Romero Pedro J, Garrett-Mayer Elizabeth, Emens Leisha A
Tisch Cancer Center, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
UPMC Hillman Cancer Center, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA.
J Immunother Cancer. 2025 Mar 22;13(3):e010760. doi: 10.1136/jitc-2024-010760.
Clinical trials of cancer immunotherapy (IO) were historically based on a drug development paradigm built for chemotherapies. The remarkable clinical activity of programmed cell death protein 1/programmed death ligand 1 blockade, chimeric antigen receptor-T cells, and T cell engagers yielded new insights into how the mechanistic underpinnings of IO are reflected in the clinic. These insights and the sheer number of novel immunotherapies currently in the pipeline have made it clear that our strategies and tools for IO drug development must adapt. Recent innovations like engineered T cells and tumor-infiltrating lymphocytes demonstrate that immune-based treatments may rely on real-time manufacturing programs rather than off-the-shelf drugs. We now recognize adoptively transferred cells as living drugs. Progression criteria have been redefined due to the unique response patterns of IO. Harnessing the power of both biomarkers and the neoadjuvant setting earlier in drug development is of broad interest. The US Food and Drug Association is increasingly impacting the design of trials with respect to dose optimization and clinical endpoints. The use of novel endpoints such as pathologic complete/major response, treatment-free survival, and minimal residual disease is becoming more common. There is growing acceptance of using patient-reported outcomes as trial endpoints to better measure the true clinical benefit and impact of novel IO agents on quality of life. New opportunities created by modern data science and artificial intelligence to inform and accelerate drug development continue to emerge. The importance of streamlining the clinical research ecosystem and enhancing clinical trial access to facilitate the enrollment of diverse patient populations is broadly recognized. Patient advocacy is critical both to drive the science of IO, and to promote patient satisfaction. To capitalize on these opportunities, the Society for Immunotherapy of Cancer (SITC) has established a goal of at least 100 new, unique IO approvals over the next 10 years. Accordingly, SITC has developed initiatives designed to integrate the viewpoints of diverse stakeholders and galvanize the field in further adapting clinical trials to the unique features of IO, moving us closer to our ultimate goal of using IO to cure and prevent cancer.
癌症免疫疗法(IO)的临床试验在历史上是基于为化疗建立的药物开发模式。程序性细胞死亡蛋白1/程序性死亡配体1阻断、嵌合抗原受体T细胞和T细胞衔接器显著的临床活性,为IO的机制基础如何在临床中体现带来了新的见解。这些见解以及目前正在研发的大量新型免疫疗法表明,我们用于IO药物开发的策略和工具必须做出调整。工程化T细胞和肿瘤浸润淋巴细胞等近期创新表明,基于免疫的治疗可能依赖实时制造程序而非现成药物。我们现在将过继转移的细胞视为活性药物。由于IO独特的反应模式,疾病进展标准已被重新定义。在药物开发早期利用生物标志物和新辅助治疗的力量受到广泛关注。美国食品药品监督管理局在剂量优化和临床终点方面对试验设计的影响越来越大。使用病理完全/主要缓解、无治疗生存期和微小残留病等新型终点变得越来越普遍。越来越多的人接受将患者报告的结果用作试验终点,以更好地衡量新型IO药物对生活质量的真正临床益处和影响。现代数据科学和人工智能为药物开发提供信息并加速其进程所创造的新机会不断涌现。简化临床研究生态系统并增加临床试验机会以促进不同患者群体入组的重要性已得到广泛认可。患者宣传对于推动IO科学发展以及提高患者满意度至关重要。为了利用这些机会,癌症免疫治疗学会(SITC)设定了在未来10年至少批准100种新的、独特的IO药物的目标。因此,SITC制定了一些举措,旨在整合不同利益相关者的观点,并激励该领域进一步使临床试验适应IO的独特特征,使我们更接近使用IO治愈和预防癌症的最终目标。