NCTN BIQSFP ANBL1531 (NRT)

项目摘要 自从60多年前引入化疗治疗儿童白血病以来， 儿童癌症的预后已显著改善。儿童的5年生存率 癌症，其中许多在化疗前的时代都是致命的，现在是84%。一个数字的进展 然而，儿童癌症的发病率是有限的，大约50%的儿童患有急性骨髓性白血病， 白血病，50%的高危神经母细胞瘤儿童，以及90%以上的脑干胶质瘤儿童， 仍然在疾病中挣扎在美国，癌症仍然是儿童疾病死亡的主要原因 超过一岁。此外，癌症治疗的晚期效应，包括永久性器官和 组织损伤、激素和生殖功能障碍以及第二种癌症是特别令人关注的问题， 在50万儿童癌症幸存者中，超过40%（估计截至2020年）经历了显著的健康状况。 儿童癌症相关生活质量并发症及其治疗因此，尽管我们取得了进步， 开发新的治疗方法必须是儿童癌症基础，转化和 临床研究者儿童肿瘤学组织（COG）是世界上最大的致力于 专门从事儿童和青少年癌症研究，成立于20年前。COG的 由9,000多名成员组成的多学科研究团队，在220多个国家开展研究。 领先的儿童医院大学和癌症中心本提案适用于COG，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 支持改善所有癌症儿童预后的使命。组委会将设计和 为癌症儿童进行临床转化研究，建立在对癌症的日益了解的基础上。 儿童恶性肿瘤的分子基础，并具有最大的潜力，以改善结果。使用 创新的临床试验设计适合罕见疾病的研究，我们将研究新的治疗方法， 这些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的研究组合还包括临床试验，重点是改善生活质量， 癌症儿童和癌症幸存者。超过90%的儿童被诊断患有癌症， 美国在COG成员机构治疗，COG有能力提供多样化的儿童人口 癌症患者及其家属有机会参与创新研究。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， 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，临床科学家谁开发和 开展研究，并通过招募合格受试者供成员研究中心参与。

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.