Pediatric Preclinical Testing Consortium: Research Programs non-CNS

儿科临床前测试联盟：非中枢神经系统研究项目

• 批准号: 10076139
• 负责人: PETER J HOUGHTON
• 金额: $ 31.52万
• 依托单位: UNIVERSITY OF TEXAS HLTH SCIENCE CENTER
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-01 至 2021-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10076139
• 关键词: Cells; Childhood; Childhood Solid Neoplasm; Data; Drug Combinations; Ewings sarcoma; Future; In Vitro; Kidney; Kidney Neoplasms; Manuscripts; Modeling; Molecular; New Agents; Pre-Clinical Model; Preclinical Testing; Radiation; Renal carcinoma; Reporting; Research; Rhabdomyosarcoma; Vincristine; Xenograft Model; childhood sarcoma; cohort; exome; exome sequencing; high risk; in vivo; novel; novel drug combination; programs; sarcoma; screening; transcriptome sequencing

The group at GCCRI will continue studies focused on identifying active agents and combinations using xenograft models of pediatric sarcoma and kidney tumors. We will complete ongoing studies, and start new studies as described below. In addition we plan to characterize 18 new sarcoma PDX models (exome seq and RNAseq), and complete characterization of 33 PDX models where we have exome sequence data from the TARGET initiative, but where we lack RNAseq data. This will greatly expand the number of available sarcoma models that can be used in the future PPP being developed by the FNIH. Our aims for the 6 month extension are: Specific Aims Proposed studies during the extension period. 1. Complete studies with Amgen 509 (ES-7, ES-3, EW-5). 2. Complete in vivo studies with ONC201. 3. Undertake screening for PPTC drugs/combinations. 4. Evaluate PLX-038A combined with vincristine in sarcoma and kidney models. 5. Evaluate Talazoparib + Radiation in CHLA258 cell in vitro and in vivo, then expand if justified 6. To characterize 33 models by RNAseq that were omitted from the ALS cohort. 7 To characterize 18 rhabdomyosarcoma and 5 Ewing sarcoma PDX models, not previously characterized. 8. Complete submission of manuscripts reporting PPTC results.

GCCRI的小组将继续研究侧重于确定活性剂和 使用小儿肉瘤和肾脏肿瘤的异种移植模型的组合。我们将完成 正在进行的研究，并如下所述开始新的研究。此外，我们计划表征 18种新的肉瘤PDX型号（外显子SEQ和RNASEQ），并完全表征33 PDX模型，我们从目标计划中获得了外显子序列数据，但我们的位置 缺乏RNASEQ数据。这将大大扩大可用的肉瘤模型的数量 在将来使用的PPP由FNIH开发。我们的6个月延长目标是： 具体目标 在延长期间提出的研究。 1。对Amgen 509（ES-7，ES-3，EW-5）的完整研究。 2。与ONC201的体内研究。 3。对PPTC药物/组合进行筛查。 4。评估PLX-038A与肉瘤和肾脏模型中的长春克氨酸结合。 5。在体外和体内评估CHLA258细胞中的Talazoparib +辐射 6。以ALS队列省略的RNASEQ表征33个模型。 7表征18种横纹肌肉瘤和5个Ewing肉瘤PDX模型，而不是以前 特征。 8。全面提交手稿报告PPTC结果。