This review comprehensively analyzes the current landscape of tumor-agnostic therapies in oncology. Tumor-agnostic therapies are designed to target specific molecular alterations rather than the primary site of the tumor, representing a shift in cancer treatment. We discuss recent approvals by regulatory agencies such as the FDA and EMA, highlighting therapies that have demonstrated efficacy across multiple cancer types sharing common alterations. We delve into the trial methodologies that underpin these approvals, emphasizing innovative designs such as basket trials and umbrella trials. These methodologies present unique advantages, including increased efficiency in patient recruitment and the ability to assess drug efficacy in diverse populations rapidly. However, they also entail certain challenges, including the need for robust biomarkers and the complexities of regulatory requirements. Moreover, we examine the promising prospects for developing therapies for rare cancers that exhibit common molecular targets typically associated with more prevalent malignancies. By synthesizing these insights, this review underscores the transformative potential of tumor-agnostic therapies in oncology. It offers a pathway for personalized cancer treatment that transcends conventional histology-based classification.
本综述全面分析了当前肿瘤学中肿瘤不可知疗法的现状。肿瘤不可知疗法旨在针对特定的分子改变而非肿瘤原发部位，代表了癌症治疗模式的转变。我们讨论了美国食品药品监督管理局和欧洲药品管理局等监管机构的最新批准，重点介绍了在具有共同分子改变的多种癌症类型中均显示出疗效的疗法。我们深入探讨了支撑这些批准所依据的试验方法学，强调了篮式试验和伞式试验等创新设计。这些方法学具有独特优势，包括提高患者招募效率以及能够快速评估药物在不同人群中的疗效。然而，它们也面临特定挑战，包括需要可靠的生物标志物以及监管要求的复杂性。此外，我们探讨了针对罕见癌症开发疗法的前景，这些罕见癌症表现出通常与更常见恶性肿瘤相关的共同分子靶点。通过整合这些见解，本综述强调了肿瘤不可知疗法在肿瘤学领域的变革潜力，为超越传统基于组织学的分类、实现个体化癌症治疗提供了路径。
Tumor-Agnostic Therapies in Practice: Challenges, Innovations, and Future Perspectives
肿瘤(癌症)患者之家