Institut National du Cancer, 52 avenue André Morizet, F-92513 Boulogne-Billancourt, France.
Centre Léon Bérard, 28 Rue Laennec, F-69008, Lyon, France.
Nat Rev Clin Oncol. 2016 Jun;13(6):385-93. doi: 10.1038/nrclinonc.2016.31. Epub 2016 Mar 22.
Patients with cancers of differing histologies that express certain biomarkers are likely to benefit from treatment with targeted therapies. However, targets can be present in malignancies other than those indicated by a drug's label, and as a result, affected patients will have no access to those potentially useful drugs. To tackle this issue, the French National Cancer Institute developed the AcSé Programme in 2013. This programme is designed to make treatment decisions or recommendations on the basis of the presence of relevant biomarkers for malignancies with no targeted therapies available and also aims to improve safety, and evaluate the efficacy of targeted drugs used outside of their approved indications. Patients across France have access to molecular testing in 28 molecular genetics centres and to targeted therapies within phase II trials provided no other trials exist in which they could reasonably be included. Trials include patients below the age of 18 if safe dosing data are available. As of January 2016, 183 French clinical sites and over 7,000 patients are participating in AcSé led trials. Proof of concept is being demonstrated through trials designed to investigate the effectiveness of crizotinib and vemurafenib in a wide variety of cancers.
表达某些生物标志物的不同组织学癌症患者可能受益于靶向治疗。然而，靶点可能存在于药物标签所示之外的恶性肿瘤中，因此，受影响的患者将无法获得这些潜在有用的药物。为了解决这个问题，法国国家癌症研究所于 2013 年制定了 AcSé 计划。该计划旨在根据无靶向治疗的恶性肿瘤的相关生物标志物做出治疗决策或建议，还旨在提高安全性，并评估批准适应症外使用的靶向药物的疗效。法国各地的患者可以在 28 个分子遗传学中心进行分子检测，并在没有其他合理纳入试验的情况下参加 II 期试验中的靶向治疗。如果有安全剂量数据，试验包括 18 岁以下的患者。截至 2016 年 1 月，183 个法国临床站点和 7000 多名患者正在参与 AcSé 主导的试验。通过旨在研究克唑替尼和vemurafenib在各种癌症中的有效性的试验来证明这一概念。
