COG NCTN Year 10 BIQSFP Supplement & Progress Report

项目总结 自60多年前引入化疗治疗儿童白血病以来， 儿童癌症的预后有了显著的改善。儿童总体5年存活率 癌症，其中许多在化疗前时代都是致命的，现在是84%。一个数字的进步 然而，儿童癌症的发病率是有限的，大约50%的儿童急性骨髓性白血病 白血病，50%的高危神经母细胞瘤儿童，90%以上的脑干胶质瘤儿童， 仍然屈服于他们的疾病。在美国，癌症仍然是儿童疾病死亡的主要原因 年龄超过一岁的。此外，癌症治疗的后遗症，包括永久性器官和 组织损伤、荷尔蒙和生殖功能障碍以及第二种癌症特别令人担忧，还有更多 在经历重大健康状况的500,000名儿童癌症幸存者中(估计截至2020年)，超过40%的人 儿童癌症相关生活质量并发症及其治疗。因此，尽管我们取得了进展， 开发新的治疗方法必须是儿童癌症基础、转化和治疗的优先事项 临床研究人员。儿童肿瘤学组织(COG)，世界上最大的组织，致力于 专门对儿童和青少年癌症的研究，成立于20年前。COG的 由9000多名成员组成的多学科研究团队开展了220多项研究 领先的儿童医院、大学和癌症中心。这项提案是为COG提出的，作为国家 癌症研究所(NCI)的国家临床试验网络(NCTN)，继续其合作研究工作 这支持了改善所有癌症儿童的结果的使命。中国奥组委将设计和实施 为患有癌症的儿童进行临床-翻译研究，建立在对 这是儿童恶性肿瘤的分子基础，并具有改善预后的最大潜力。vbl.使用 创新临床试验设计适合罕见病研究，我们将研究新的治疗方法 方法包括但不限于靶向小分子药物、免疫疗法和细胞疗法。 COG的研究组合还包括重要的临床试验，重点是改善患者的生活质量 患有癌症的儿童和儿童癌症幸存者。年，超过90%的儿童被诊断为癌症 美国在COG成员机构接受治疗，COG有能力为多样化的儿童群体提供服务 为癌症患者及其家人提供参与创新研究的机会。这项研究工作包括 允许收集和注释所有癌症儿童的生物标本，为 用于发现和加速在世界各地的实验室进行的最有前途的研究工作。 该提案是为了支持COG网络运营中心，临床医生-科学家开发和 进行研究，并通过注册符合条件的科目让成员站点参与。

PROJECT SUMMARY Since the introduction of chemotherapy for the treatment of childhood leukemia more than 60 years ago, the prognosis of childhood cancer has improved dramatically. The overall 5-year survival rate for childhood cancers, many of which were uniformly fatal in the pre-chemotherapy era, is now 84%. Progress for a number of childhood cancers, however, has been limited, with approximately 50% of children with acute myelogenous leukemia, 50% of children with high-risk neuroblastoma, and more than 90% of children with brainstem glioma, still succumbing to their disease. In the US, cancer remains the leading cause of death from disease in children greater than one year of age. Moreover, the late effects of cancer treatment, including permanent organ and tissue damage, hormonal and reproductive dysfunction and second cancers, are of special concern, with more than 40% of the 500,000 survivors of childhood cancer (estimated as of 2020) experiencing a significant health related quality of life complication from childhood cancer and its treatment. Thus, despite our advances, development of new therapeutic approaches must be a priority for childhood cancer basic, translational and clinical researchers. The Children’s Oncology Group (COG), the world’s largest organization devoted exclusively to childhood and adolescent cancer research, was founded 20 years ago. The COG’s multidisciplinary research team, comprised of more than 9,000 members, conducts research at more than 220 leading children’s hospitals, universities, and cancer centers. This proposal is for COG, as part of the National Cancer Institute’s (NCI) National Clinical Trials Network (NCTN), to continue its collaborative research work that supports the mission of improving the outcome for all children with cancer. The COG will design and conduct clinical-translational studies for children with cancer that builds on an increasing understanding of the molecular basis for pediatric malignancies and has the highest potential to improve the outcome. Using innovative clinical trial designs suitable for the study of rare diseases, we will study novel therapeutic approaches including but not limited to targeted small molecule drugs, immunotherapies and cellular therapies. The COG research portfolio importantly also includes clinical trials focused on improving the quality of life for children with cancer and childhood cancer survivors. As more than 90% of children diagnosed with cancer in the US are treated at COG member institutions, COG has the ability to offer a diverse population of children with cancer and their families the opportunity to participate in innovative research. This research effort includes allowing for collection and annotation of biospecimens from all children with cancer, providing the foundation for discovery and accelerating the most promising research efforts conducted in laboratories around the world. The proposal is for support of the COG Network Operations Center, clinician-scientists who develop and conduct research, and for member sites to participate through enrollment of eligible subjects.