Department of Pediatric Oncology, Hematology, and Hemostaseology, Children’s Hospital, University of Leipzig, Leipzig, Germany.
J Clin Invest. 2011 Feb;121(2):545-53. doi: 10.1172/JCI44415.
Reliable diagnostic strategies for individuals with cancer demand practical methods for highly sensitive and specific detection of tumor cells. Amplification of genomic regions that include putative oncogenes is common in tumor cells of various types. Genomic array platforms offer the opportunity to identify and precisely map amplified genomic regions (ampGRs). The stable existence of these tumor cell–specific genomic aberrations during and after therapy, in theory, make ampGRs optimal targets for cancer diagnostics. In this study, we mapped ampGRs around the proto-oncogene MYCN of human neuroblastomas using a high-resolution tiling array (HR-TA). Based on the HR-TA data, we were able to precisely describe the telomeric and centromeric borders of the ampGRs and deduce virtual fusion sites of the joined ampGRs (amplicon fusion sites [AFSs]). These AFSs served as blueprints for the subsequent design of AFS bridging PCR assays (AFS-PCRs). Strikingly, these assays were absolutely tumor cell specific and capable of detecting 1 tumor cell in 1 × 10(6) to 8 × 10(6) control cells. We successfully proved the in vivo practicability of AFS-PCR by detecting and quantifying the specific AFS DNA of human MYCN-amplified neuroblastomas in the patients’ corresponding peripheral blood and bone marrow samples. Thus, we believe AFS-PCR could become a powerful and nevertheless feasible personalized diagnostic tool applicable to a large number of cancer patients, including children with MYCN-amplified neuroblastomas.
对于癌症患者,可靠的诊断策略需要实用的方法来高度敏感和特异性地检测肿瘤细胞。扩增包括推测的癌基因的基因组区域在各种类型的肿瘤细胞中很常见。基因组阵列平台提供了识别和精确绘制扩增基因组区域(ampGRs)的机会。这些肿瘤细胞特异性基因组异常在治疗期间和之后的稳定存在,理论上使 ampGRs 成为癌症诊断的最佳靶标。在这项研究中,我们使用高分辨率平铺阵列(HR-TA)绘制了人类神经母细胞瘤原癌基因 MYCN 周围的 ampGRs。基于 HR-TA 数据,我们能够精确描述 ampGRs 的端粒和着丝粒边界,并推断出连接的 ampGRs 的虚拟融合位点(ampGR 融合位点 [AFS])。这些 AFS 作为随后设计 AFS 桥接 PCR 检测(AFS-PCR)的蓝图。引人注目的是,这些检测绝对是肿瘤细胞特异性的,能够在 1 × 10(6) 到 8 × 10(6) 个对照细胞中检测到 1 个肿瘤细胞。我们通过在患者相应的外周血和骨髓样本中检测和定量人 MYCN 扩增神经母细胞瘤的特异性 AFS DNA,成功证明了 AFS-PCR 的体内实用性。因此,我们相信 AFS-PCR 可以成为一种强大且可行的个性化诊断工具,适用于包括 MYCN 扩增神经母细胞瘤患儿在内的大量癌症患者。