Therapeutics Studies of Primary CNS Malignancies

描述(由申请人提供)：Dana Farber癌症研究所(DFCI)寻求作为北美脑瘤联合会(NABTC)的正式成员参与，为脑瘤患者的I/II期试验提供患者资源和新型治疗剂。NABTC的中央运营办公室/协调中心将设在加州大学旧金山分校(UCSF)，由马里兰州迈克尔·普拉多斯领导。德克萨斯大学圣安东尼奥分校将作为药代动力学中心。NABTC的目标是在脑肿瘤患者中进行新的临床试验，并对具有潜在临床意义的中枢神经系统肿瘤生物学方面进行辅助实验室研究。在此次续签申请中，NABTC还将计划与其他资助的成人(NABTT)和儿童(PBTC)脑瘤联盟、癌症联盟的小鼠模型、最近资助的脑瘤孢子机构(加州大学旧金山分校和阿拉巴马大学)以及NIH/NCI的内科神经肿瘤科进行互动。DFCI的多学科神经肿瘤学项目致力于1)确保每年至少有20名可评估的患者被纳入NABTC试验；2)在我们拥有特殊专长的领域为该财团提供资源；例如在进行相关研究以评估肿瘤的基因型和特定信号转导通路的体内抑制证据、阵列比较基因组杂交(CGH)、胶质瘤转基因动物模型、脑肿瘤组织库和免疫疗法；3)为NABTC试验提出新的治疗方法。在这方面，我们建议I)RAD001用于复发恶性胶质瘤的I/II期研究；以及II)RAD001联合放射治疗新诊断的胶质母细胞瘤的I/II期试验。此外，我们希望与NABTC一起进行端粒酶疫苗的II期研究和CXCR4抑制剂的I/II期试验。总之，我们将带来DFCI多学科成人神经肿瘤学项目的全部资源，以及一个综合癌症中心的资源，以加强NABTC为脑瘤患者开发更有效疗法的使命。

DESCRIPTION (provided by applicant): The Dana Farber Cancer Institute (DFCI) seeks to participate as a Full Member Institution of The North American Brain Tumor Consortium (NABTC), providing patient resources and novel therapeutic agents for phase I/II trials in brain tumor patients. The Central Operations Office/Coordinating Center for the NABTC will be located at the University of California San Francisco (UCSF) under the Group Leadership of Michael Prados, MD. The University of Texas, San Antonio will serve as the Pharmacokinetics Center. The goal of NABTC is to conduct novel clinical trials in brain tumor patients and to perform ancillary laboratory investigations of aspects of CNS tumor biology with potential clinical implications. In this renewal application, the NABTC will also plan to interact with the other funded adult (NABTT) and pediatric (PBTC) brain tumor consortia, the Mouse Models of Cancer Consortium, the recently funded Brain Tumor SPORE institutions (UCSF and University of Alabama), and the intramural Neuro-Oncology Branch at the NIH/NCI. The multidisciplinary Neuro-Oncology program at DFCI is committed to 1) Ensuring that a minimum of 20 evaluable patients will be accrued into NABTC trails each year; 2) Acting as a resource to the consortium in areas where we have particular expertise; for example in performing correlative studies evaluating genotype of tumors and in vivo evidence of inhibition of specific signal transduction pathways, array comparative genomic hybridization (CGH), transgenic animal models of gliomas, brain tumor tissue bank and immunotherapies; 3) Proposing novel therapies for NABTC trials. In this regard, we have proposed i) Phase I/II study of RAD001 for recurrent malignant gliomas; and ii) Phase I/II trial of RAD001 with radiation therapy for newly-diagnosed glioblastomas. In addition, we hope to conduct a phase II study of a telomerase vaccine and a phase I/II trial of an inhibitor of CXCR4 with NABTC. In summary, we will bring the full resources of the multidisciplinary Adult Neuro-Oncology Program at DFCI, and the resources of a Comprehensive Cancer Center to enhance the mission of NABTC in developing more effective therapies for patients with brain tumors.