Division of Medical Oncology, Santa Maria della Misericordia Hospital, Azienda Ospedaliera di Perugia, Via Dottori, 1, 06156 Perugia, Italy.
Expert Opin Emerg Drugs. 2012 Mar;17(1):31-6. doi: 10.1517/14728214.2012.656588. Epub 2012 Jan 31.
Small cell lung cancer (SCLC) is a biologically complex tumor whose medical treatment has remained largely unchanged over the last 30 years. The frustration for the invariably negative results reported in the early 2000s in clinical studies investigating new agents for this disease have contributed to the little interest shown by pharmaceutical industries in funding SCLC research. However, recent advances in the molecular understanding of the oncogenic mechanisms underlying SCLC have renewed the attraction for the clinical development of novel active drugs for the treatment of this challenging disease.
The authors briefly touch on the most promising agents under clinical development for the treatment of SCLC, either chemotherapeutic or targeted drugs. Relevant and recent (2 years) studies obtained through Pubmed literature research or released at International scientific meetings are presented.
The data discussed herein provide evidence in support of the fact that only rationally designed clinical trials including relevant translational research may lead to successful results. Therefore, a thoughtful change in trial construction is crucial for the approval of new active drugs for this orphan disease.
小细胞肺癌（SCLC）是一种生物学上复杂的肿瘤，其医学治疗在过去 30 年中基本没有改变。在 21 世纪初，针对这种疾病的新药进行的临床研究报告的始终为负面结果，这令人感到沮丧，这导致制药行业对 SCLC 研究的兴趣不大。然而，对 SCLC 致癌机制的分子理解的最新进展重新引起了人们对开发新型有效药物治疗这种具有挑战性疾病的临床开发的兴趣。
作者简要介绍了目前处于临床开发阶段的、用于治疗 SCLC 的最有前途的药物，包括化疗药物和靶向药物。通过 Pubmed 文献研究或在国际科学会议上发布的相关最新（2 年）研究进行了介绍。
本文讨论的数据提供了证据支持，即只有包括相关转化研究的合理设计的临床试验才可能产生成功的结果。因此，改变试验设计对于批准这种孤儿病的新型有效药物至关重要。
