Sathornsumetee Sith, Rich Jeremy N
The Preston Robert Tisch Brain Tumor Center Division of Neurosurgery/Neuro-Oncology, Duke University Medical Center, DUMC 3624, Durham, NC 27710, USA.
Expert Rev Anticancer Ther. 2006 Jul;6(7):1087-104. doi: 10.1586/14737140.6.7.1087.
Malignant gliomas are the most prevalent type of primary brain tumor in adults. Despite progress in brain tumor therapy, the prognosis of malignant glioma patients remains dismal. The median survival of patients with glioblastoma multiforme, the most common grade of malignant glioma, is 10-12 months. Conventional therapy of surgery, radiation and chemotherapy is largely palliative. Essentially, tumor recurrence is inevitable. Salvage treatments upon recurrence are palliative at best and rarely provide significant survival benefit. Therapies targeting the underlying molecular pathogenesis of brain tumors are urgently required. Common genetic abnormalities in malignant glioma specimens are associated with aberrant activation or suppression of cellular signal transduction pathways and resistance to radiation and chemotherapy. Several low molecular weight signal transduction inhibitors have been examined in preclinical and clinical malignant glioma trials. The efficacy of these agents as monotherapies has been modest, at best; however, small subsets of patients who harbor specific genetic changes in their tumors may display favorable clinical responses to defined small molecule inhibitors. Multitargeted kinase inhibitors or combinations of agents targeting different mitogenic pathways may overcome the resistance of tumors to single-agent targeted therapies. Well designed studies of small molecule kinase inhibitors will include assessment of safety, drug delivery, target inhibition and correlative biomarkers to define mechanisms of response or resistance to these agents. Predictive biomarkers will enrich for patients most likely to respond in future clinical trials. Additional clinical studies will combine novel targeted therapies with radiation, chemotherapies and immunotherapies.
恶性胶质瘤是成人中最常见的原发性脑肿瘤类型。尽管脑肿瘤治疗取得了进展,但恶性胶质瘤患者的预后仍然很差。多形性胶质母细胞瘤是最常见的恶性胶质瘤分级,其患者的中位生存期为10 - 12个月。传统的手术、放疗和化疗主要是姑息性的。从本质上讲,肿瘤复发是不可避免的。复发时的挽救治疗充其量只是姑息性的,很少能带来显著的生存益处。迫切需要针对脑肿瘤潜在分子发病机制的治疗方法。恶性胶质瘤标本中常见的基因异常与细胞信号转导通路的异常激活或抑制以及对放疗和化疗的耐药性有关。几种低分子量信号转导抑制剂已在临床前和临床恶性胶质瘤试验中进行了研究。这些药物作为单一疗法的疗效充其量只能说是一般;然而,肿瘤中存在特定基因变化的一小部分患者可能对特定的小分子抑制剂表现出良好的临床反应。多靶点激酶抑制剂或针对不同促有丝分裂途径的药物组合可能克服肿瘤对单一药物靶向治疗的耐药性。精心设计的小分子激酶抑制剂研究将包括安全性评估、药物递送、靶点抑制和相关生物标志物,以确定对这些药物的反应或耐药机制。预测性生物标志物将使未来临床试验中最可能有反应的患者富集。更多的临床研究将把新型靶向治疗与放疗、化疗和免疫治疗相结合。