The potential for molecular therapeutic targets in Ewing's sarcoma.
The potential for molecular therapeutic targets in Ewing's sarcoma.
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DOI：
10.1007/s11864-005-0025-y
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发表时间：
2005-11-01
影响因子：
4.3
通讯作者：
Lessnick, Stephen L
中科院分区：
文献类型：
--
作者：
McAllister, Nancy R;Lessnick, Stephen L
尤文氏肉瘤是一种儿童和年轻人的顽固性肿瘤。在尤文氏肉瘤的化疗引入之前，几乎所有的患者都死于他们的疾病，即使是高度积极的局部控制方法。认识到大多数患者在就诊时具有微转移性疾病，以及针对该肿瘤的活性化疗剂的鉴定，已经导致患者存活率的显著改善。尤文氏肉瘤的现代治疗结合了用于全身控制疾病的高剂量化疗，以及用于局部控制的先进手术和/或放射治疗方法。目前的治疗仍然不完善。尽管有最佳的管理，局部疾病的治愈率仅为约70%，而转移性疾病的治愈率低于30%。经历长期无病生存的患者存在治疗的显著副作用的风险，包括不孕症、肢体功能障碍和继发恶性肿瘤的风险增加。需要更有效和毒性更小的治疗方法。本报告概述了尤文氏肉瘤中失调的分子通路，并强调了它们可能作为该疾病治疗靶点的可能性。虽然在这些方法中的大多数可以在临床上进行评估之前需要大量的额外调查，但我们认为这些潜在的新靶点为尤文肉瘤患者提供了很大的希望。
Ewing's sarcoma is an uncompromising tumor of children and young adults. Before the introduction of chemotherapy for Ewing's sarcoma, nearly all patients succumbed to their disease, even with highly aggressive approaches to local control. The realization that most patients have micrometastatic disease at presentation, and the identification of active chemotherapeutic agents for this tumor, have resulted in significant improvements in patient survival. Modern therapy for Ewing's sarcoma combines high-dose chemotherapy for systemic control of disease, with advanced surgical and/or radiation therapeutic approaches for local control. Current therapy remains imperfect. Despite optimal management, the cure rate for localized disease is only approximately 70%, whereas the cure rate for metastatic disease at presentation is less than 30%. Patients who experience long-term disease-free survival are at risk for significant side effects of therapy, including infertility, limb dysfunction, and an increased risk for second malignancies. More effective and less toxic therapies are needed. This report presents an overview of dysregulated molecular pathways in Ewing's sarcoma and highlights the possibility that they may serve as therapeutic targets for the disease. Although a great deal of additional investigation is required before most of these approaches can be assessed in the clinic, we think that these potential new targets offer a great deal of hope for patients with Ewing's sarcoma.