Desmoplastic Small Round Cell Tumor (DSRCT) is a rare sarcoma tumor of adolescence and young adulthood, which harbors a recurrent chromosomal translocation between the Ewing's sarcoma gene (EWSR1) and the Wilms' tumor suppressor gene (WT1). Patients usually develop multiple abdominal tumors with liver and lymph node metastasis developing later. Survival is poor using a multimodal therapy that includes chemotherapy, radiation and surgical resection, new therapies are needed for better management of DSRCT. Triggering cell apoptosis is the scientific rationale of many cancer therapies. Here, we characterized for the first time the expression of pro-apoptotic receptors, tumor necrosis-related apoptosis-inducing ligand receptors (TRAILR1-4) within an established human DSRCT cell line and clinical samples. The molecular induction of TRAIL-mediated apoptosis using agonistic small molecule, ONC201 in vitro cell-based proliferation assay and in vivo novel orthotopic xenograft animal models of DSRCT, was able to inhibit cell proliferation that was associated with caspase activation, and tumor growth, indicating that a cell-based delivery of an apoptosis-inducing factor could be relevant therapeutic agent to control DSRCT.

中文翻译：

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ONC201在增生性小圆形细胞肿瘤中的功效。

增塑小圆形细胞瘤（DSRCT）是一种青春期和成年后罕见的肉瘤瘤，在尤文氏肉瘤基因（EWSR1）和威尔姆斯抑癌基因（WT1）之间具有反复的染色体易位。患者通常会发展为多发性腹部肿瘤，并随后发展为肝和淋巴结转移。使用包括化疗，放疗和手术切除在内的多模式疗法，生存期很差，需要新的疗法来更好地处理DSRCT。触发细胞凋亡是许多癌症疗法的科学原理。在这里，我们首次表征了已建立的人DSRCT细胞系和临床样品中促凋亡受体，肿瘤坏死相关凋亡诱导配体受体（TRAILR1-4）的表达。