Department of Pathology and Laboratory Medicine, University of Kansas Medical Center, Kansas City, KS 66160, USA.
Proc Natl Acad Sci U S A. 2012 Aug 21;109(34):13650-5. doi: 10.1073/pnas.1203326109. Epub 2012 Aug 6.
Expression of functional breast cancer susceptibility gene 1 (BRCA1) in human breast and ovarian cancers is associated with resistance to platinum-based chemotherapeutics and poly(ADP ribose) polymerase (PARP) inhibitors. BRCA1 is a nuclear tumor suppressor that is critical for resolving double-strand DNA breaks (DSBs) and interstrand crosslinks (ICLs) by homologous recombination (HR). In vitro, animal and human clinical data have demonstrated that BRCA1-deficient cancers are highly sensitive to ICL-inducing chemotherapeutic agents, are amenable to synthetic lethal approaches that exploit defects in DSB/ICL repair, and may be associated with improved survival. Conversely, high or restored expression of BRCA1 in breast and ovarian cancer is associated with therapeutic resistance and poor prognosis. There has been much interest in identifying agents that interfere with BRCA1-dependent DSB/ICL repair to restore or enhance sensitivity to cancer therapeutics. We demonstrate that the heat-shock protein 90 (HSP90) inhibitor 17-allylamino-17-demethoxygeldanamycin [17-AAG (Tanespimycin)], currently in Phase II/III clinical evaluation for several cancers, induces BRCA1 ubiquitination and proteasomal degradation, resulting in compromised repair of ionizing radiation- and platinum-induced DNA damage. We show that loss of HSP90 function abolishes BRCA1-dependent DSB repair and that BRCA1-deficient cells are hypersensitive to 17-AAG due to impaired Gap 2/Mitosis (G2/M) checkpoint activation and resultant mitotic catastrophe. In summary, we document an upstream HSP90-dependent regulatory point in the Fanconi anemia/BRCA DSB/ICL repair pathway, illuminate the role of BRCA1 in regulating damage-associated checkpoint and repair responses to HSP90 inhibitors, and identify BRCA1 as a clinically relevant target for enhancing sensitivity in refractory and/or resistant malignancies.
功能性乳腺癌易感基因 1(BRCA1)在人乳腺癌和卵巢癌中的表达与铂类化疗药物和聚(ADP 核糖)聚合酶(PARP)抑制剂的耐药性有关。BRCA1 是一种核肿瘤抑制因子,对于通过同源重组(HR)解决双链 DNA 断裂(DSBs)和链间交联(ICLs)至关重要。在体外、动物和人类临床数据表明,BRCA1 缺陷型癌症对 ICL 诱导的化疗药物高度敏感,适用于利用 DSB/ICL 修复缺陷的合成致死方法,并且可能与改善的生存相关。相反,乳腺癌和卵巢癌中 BRCA1 的高表达或恢复表达与治疗抵抗和预后不良相关。人们一直很感兴趣的是确定干扰 BRCA1 依赖性 DSB/ICL 修复的药物,以恢复或增强对癌症治疗的敏感性。我们证明,热休克蛋白 90(HSP90)抑制剂 17-烯丙基-17-去甲氧基格尔德霉素[17-AAG(坦那霉素)],目前正在进行几种癌症的 II/III 期临床评估,可诱导 BRCA1 泛素化和蛋白酶体降解,导致电离辐射和铂类诱导的 DNA 损伤修复受损。我们表明,HSP90 功能的丧失会破坏 BRCA1 依赖性 DSB 修复,并且 BRCA1 缺陷型细胞对 17-AAG 敏感,原因是 Gap2/有丝分裂(G2/M)检查点激活受损和随后的有丝分裂灾难。总之,我们记录了范可尼贫血症/BRCA DSB/ICL 修复途径中 HSP90 依赖性的上游调节点,阐明了 BRCA1 在调节与损伤相关的检查点和修复反应对 HSP90 抑制剂的作用,并确定 BRCA1 是增强难治性和/或耐药性恶性肿瘤敏感性的临床相关靶标。