Tsimberidou Apostolia M, Eggermont Alexander M M, Schilsky Richard L
From the MD Anderson Cancer Center, Houston, TX; Institute Gustave-Roussy, Paris, France; American Society of Clinical Oncology, Alexandria, VA.
Am Soc Clin Oncol Educ Book. 2014:61-9. doi: 10.14694/EdBook_AM.2014.34.61.
The promise of precision medicine for cancer is already being realized with the recent introduction of many targeted therapies, some with companion diagnostic tests that identify patients most likely to benefit from treatment. The utility of molecular profiling of cancer to identify actionable aberrations has been suggested by several small clinical trials conducted in patients with advanced cancer and by many anecdotes but is yet to be proven in well-designed, prospective, randomized trials. Several trials that will definitively test this strategy are now underway or soon to be launched. Melanoma, a disease once largely untreatable when metastatic, may be a paradigm for understanding how the molecular drivers of a disease can lead to highly effective targeted therapies, as well as for realizing the enormous therapeutic potential of unleashing the immune system against cancer to produce long-term disease control. Looking to the future, advanced omics technologies and computational techniques will enable assessment of not only genomic variants, as performed today, but also of pathway and network aberrations that will greatly facilitate selection of drug combinations likely to benefit specific patients. As our deepening understanding of tumor biology converges with rapid advances in measurement science and technology and computational analysis, we have an enormous opportunity to create a future for precision medicine in oncology that provides for highly specific, minimally toxic, and dramatically effective treatment for each patient.
随着近期多种靶向疗法的引入，癌症精准医学的前景已初步显现，其中一些疗法还配备了伴随诊断检测，以识别最有可能从治疗中获益的患者。针对晚期癌症患者开展的多项小型临床试验以及众多病例报告均表明，癌症分子谱分析在识别可用药的异常方面具有实用价值，但仍有待在精心设计的前瞻性随机试验中得到验证。目前，有几项试验正在进行或即将启动，它们将最终检验这一策略。黑色素瘤这种疾病一旦发生转移，过去大多无法治疗，它或许能成为一个范例，有助于我们理解疾病的分子驱动因素如何催生高效的靶向疗法，以及如何发挥免疫系统对抗癌症的巨大治疗潜力以实现长期疾病控制。展望未来，先进的组学技术和计算技术不仅能够像现在这样评估基因组变异，还能评估通路和网络异常，这将极大地助力选择可能使特定患者受益的药物组合。随着我们对肿瘤生物学的深入理解与测量科学、技术及计算分析的飞速发展相互融合，我们拥有巨大的机遇，为肿瘤学精准医学创造一个未来，为每位患者提供高度特异性、低毒性且疗效显著的治疗。
