Therapeutics Studies of Primary CNS Malignancies

Dana Farber癌症研究所（DFCI）寻求作为北美脑肿瘤联盟（NABTC）的正式成员机构参与，为脑肿瘤患者的I/II期试验提供患者资源和新型治疗药物。NABTC的中央运营办公室/协调中心将位于加州旧金山弗朗西斯科（UCSF），由医学博士Michael Prados领导。德克萨斯大学圣安东尼奥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CNS肿瘤生物学方面进行辅助实验室研究。在此更新申请中，NABTC还将计划与其他受资助的成人（NABTT）和儿童（PBTC）脑肿瘤联盟，癌症小鼠模型联盟，最近资助的脑肿瘤孢子机构（UCSF和亚拉巴马大学）以及NIH/NCI的内部神经肿瘤学分支进行互动。DFCI的多学科神经肿瘤学项目致力于：1）确保每年至少有20名可评估的患者进入NABTC试验; 2）在我们拥有特殊专业知识的领域为联盟提供资源;例如在进行评价肿瘤基因型的相关研究和特异性信号转导途径抑制的体内证据中，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治疗; 3）为NABTC试验提出新的治疗方法。在这方面，我们提出了i）RAD 001治疗复发性恶性胶质瘤的I/II期研究;和ii）RAD 001与放射治疗联合治疗新诊断的胶质母细胞瘤的I/II期试验。此外，我们希望进行端粒酶疫苗的II期研究和CXCR 4抑制剂与NABTC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，以及综合癌症中心的资源，以加强NABTC为脑肿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.