National Cancer Institute's Precision Medicine Initiatives for the new National Clinical Trials Network.
National Cancer Institute's Precision Medicine Initiatives for the new National Clinical Trials Network.
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DOI：
10.14694/edbook_am.2014.34.71
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发表时间：
2014-01-01
期刊：
影响因子：
--
通讯作者：
Doroshow, James
中科院分区：
文献类型：
--
作者：
Abrams, Jeffrey;Conley, Barbara;Doroshow, James
只有当研究界能够调整其临床试验方法来研究分子特征肿瘤而不是传统的组织学分类时，精准医学的前景才能完全实现。这些试验将取决于足够的组织收集，质量控制的可用性，高通量分子测定，以及筛选大量肿瘤以发现具有所需分子改变的肿瘤的能力。美国国家癌症研究所（NCI）新的国家临床试验网络（NCTN）处于进行此类试验的有利地位。NCTN能够无缝地进行伦理审查，登记患者，管理数据，并在其许多站点（包括城市和农村社区，学术中心和私人诊所）提供研究药物。最初的一组试验将集中在不同的问题上：（1）特殊反应者倡议-为什么少数实体瘤或淋巴瘤患者对某些药物反应非常好，即使大多数没有？(2)NCI MATCH试验：分子标记物能否预测对标准治疗耐药的晚期癌症患者对靶向治疗的反应？(3)ALCHEMIST试验：靶向表皮生长因子受体（EGFR）和间变性淋巴瘤激酶（ALK）抑制剂是否能改善肺腺癌辅助治疗的生存率？和（4）晚期鳞状细胞肺癌的肺癌主方案试验--在一个单一的多组试验设计中开发针对小分子特征肿瘤亚群的药物是否有优势？这些研究将有望催生一个新的治疗试验时代，这些试验将仔细选择可能对研究性治疗反应最好的肿瘤。
The promise of precision medicine will only be fully realized if the research community can adapt its clinical trials methodology to study molecularly characterized tumors instead of the traditional histologic classification. Such trials will depend on adequate tissue collection, availability of quality controlled, high throughput molecular assays, and the ability to screen large numbers of tumors to find those with the desired molecular alterations. The National Cancer Institute's (NCI) new National Clinical Trials Network (NCTN) is well positioned to conduct such trials. The NCTN has the ability to seamlessly perform ethics review, register patients, manage data, and deliver investigational drugs across its many sites including both in cities and rural communities, academic centers, and private practices. The initial set of trials will focus on different questions: (1) Exceptional Responders Initiative-why do a minority of patients with solid tumors or lymphoma respond very well to some drugs even if the majority do not?; (2) NCI MATCH trial-can molecular markers predict response to targeted therapies in patients with advanced cancer resistant to standard treatment?; (3) ALCHEMIST trial-will targeted epidermal growth factor receptor (EGFR) and anaplastic lymphoma kinase (ALK) inhibitors improve survival for adenocarcinoma of the lung in the adjuvant setting?; and (4) Lung Cancer Master Protocol trial for advanced squamous cell lung cancer-is there an advantage to developing drugs for small subsets of molecularly characterized tumors in a single, multiarm trial design? These studies will hopefully spawn a new era of treatment trials that will carefully select the tumors that may respond best to investigational therapy.