Pediatric Oncology Program

项目摘要-儿科肿瘤学(PO)计划 艾布拉姆森癌症中心(ACC)的儿科肿瘤学(PO)项目发现、开发和 翻译新的治疗方法以提高治愈率并减少急性和长期毒性 患有癌症的儿童。该计划成立于1992年，总体目标是改变护理标准 美国和世界各地患有癌症的儿童。科学目标是：1)表征分子 儿童癌症的机制和风险评估和精确治疗的易感性；2)进一步 为恶性血液病开发创新的细胞疗法，以提高治愈率并最大限度地减少使用 异基因干细胞移植；以及3)识别高危和复发实体肿瘤的靶点并开发 针对这些具有挑战性的肿瘤的新疗法。该项目由斯蒂芬·格鲁普领导，医学博士， 儿科，细胞治疗和移植科科长，细胞和基因医学主任 治疗实验室博士，儿科学副教授谭凯博士。Grupp博士是世界领先的 细胞疗法、免疫疗法和转化研究。谭恩美博士是癌症研究的领军人物 基因组学和系统生物学。他们为35个项目提供翻译和跨学科领导 成员，他们组成了一个由来自佩雷尔曼学院三个部门的调查人员组成的动态社区 医学。该计划集成了基础、翻译和临床研究组件，具有多样化的 一组在癌症基因组学、细胞生物学和信号转导、肿瘤方面具有专长的研究人员 免疫学和免疫疗法，药物开发，临床药理学，流行病学，临床研究， 癌症控制、生存和行为肿瘤学。成员在儿科肿瘤科内合作，以及 癌症控制、癌症治疗、肿瘤生物学、血液肿瘤和免疫生物学 程序。临床研究是稳健的，介入试验产生了1,156项，非介入试验产生了2,946项 在本供资期间进行的干预试验。会员从事与专业相关的研究 儿科癌症问题在我们的集水区内，并高度参与教育活动 宾夕法尼亚大学和更远的地方。最值得注意的是，在当前的资助期，PO成员与 其他计划的成员开发CD19靶向的CAR-T细胞(组织白血球)，采取第一个CAR-T 治疗急性淋巴细胞白血病的儿童和年轻人需经FDA批准。其他成就包括预付款 儿童白血病的靶向治疗，神经母细胞瘤和胶质瘤新癌症途径的发现，以及 FDA批准了两种治疗儿童实体瘤的新药。计划成员还在以下方面发挥主要领导作用 全国儿科合作小组。计划成员拥有1510万美元的年度赠款资金(直接)， 其中900万美元由同行评审，630万美元由NCI资助。该计划拥有21个R01等价物。有几个 自2015年以来，该计划发表了570份与癌症相关的出版物。其中，30%是内部计划，18%是内部计划 来自方案间协作的，83%是多机构的。

PROJECT SUMMARY – Pediatric Oncology (PO) Program The Pediatric Oncology (PO) Program of the Abramson Cancer Center (ACC) discovers, develops and translates novel therapeutic approaches to improve cure rates and reduce acute and long-term toxicities in children with cancer. Established in 1992, the Program has the overall goal to change the standard of care for children with cancer in the US and across the world. Scientific aims are: 1) Characterize molecular mechanisms of childhood cancers and predisposition for risk evaluation and precision therapies; 2) Further develop innovative cell therapies for hematologic malignancies to improve cure rates and minimize the use of allogeneic stem cell transplantation; and 3) Identify targets in high risk and relapsed solid tumors and develop new therapies for these challenging tumors. The Program is led by Stephan Grupp, MD, PhD, Professor of Pediatrics, Chief of the Cell Therapy and Transplant Section and Medical Director of the Cell and Gene Therapy Laboratory, and Kai Tan, PhD, Associate Professor of Pediatrics. Dr. Grupp is a world leader in cellular therapy, immunotherapy and translational research. Dr. Tan is a leading researcher in cancer genomics and systems biology. They provide translational and transdisciplinary leadership for 35 Program members, who form a dynamic community of investigators from three Departments in the Perelman School of Medicine. The Program has integrated basic, translational, and clinical research components, with a diverse group of investigators who have expertise in cancer genomics, cell biology and signal transduction, tumor immunology and immunotherapy, drug development, clinical pharmacology, epidemiology, clinical research, cancer control, survivorship, and behavioral oncology. Members collaborate within Pediatric Oncology, and with the Cancer Control, Cancer Therapeutics, Tumor Biology, Hematologic Malignancies, and Immunobiology Programs. Clinical investigations are robust, with 1,156 accruals to interventional trials and 2,946 to non- interventional trials in the current funding period. Members engage in research that is relevant to major pediatric cancer issues within our catchment area, and are highly engaged in educational activities across Penn and beyond. Most notably in the current funding period, PO members worked collaboratively with members of other Programs to develop CD19-targeted CAR-T cells (tisagenlecleucel), taking the first CAR-T therapy to FDA approval for children and young adults with ALL. Other accomplishments include advances for targeted therapy of pediatric leukemia, discovery of novel cancer pathways in neuroblastoma and gliomas, and two new FDA approved drugs for pediatric solid tumors. Program members also play major leadership roles in the national pediatric cooperative groups. Program members have $15.1M in annual grant funding (direct), of which $9M is peer-reviewed and $6.3M is NCI-funded. The Program holds 21 R01 equivalents. There were 570 cancer-related publications from the Program since 2015. Of these, 30% are intra-Programmatic, 18% resulted from inter-Programmatic collaboration, and 83% are multi-institutional.