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Accelerate cellular immunotherapy development for treatment of life-threatening childhood disorders

加速细胞免疫疗法的开发，用于治疗危及生命的儿童疾病

基本信息

批准号：
10460283
负责人：
JULIE R PARK
金额：
$ 139.56万
依托单位：
SEATTLE CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2018
资助国家：
美国
起止时间：
2018-08-01 至 2024-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10460283
关键词：
Academia Accelerated Phase Adolescent Adult Affect Allogenic Autoimmune Diseases Autoimmunity Award Biological Biological Assay CAR T cell therapy Caring Cell Therapy Cells Cellular immunotherapy Child Childhood Clinical Research Clinical Trials Clinical and Translational Science Awards Collaborations Disease Eligibility Determination Engineering Ensure Future Generations Goals Graft Rejection Grant Immune Immune Tolerance Immune system Immunity Immunotherapeutic agent Industry Infection Infrastructure Institution Investigational Drugs Investigational New Drug Application Investments Lead Life Longevity Lymphoproliferative Disorders Malignant Neoplasms Medicine Minority Groups Modality Monitor Multi-Institutional Clinical Trial NK cell therapy Organ Transplantation Patients Pediatric Hospitals Pharmaceutical Preparations Pilot Projects Positioning Attribute Procedures Process Production Rare Diseases Refractory Regulatory Affairs Regulatory T-Lymphocyte Research Personnel Role Safety Site Solid T cell therapy Therapeutic Time Training Translational Research Translations Underserved Population Viral Virus Diseases Work base cellular development cellular engineering clinical development clinical efficacy clinical research site design graft vs host disease hematopoietic cell transplantation high risk human disease immunoengineering immunotherapy clinical trials immunotherapy trials innovation innovative technologies investigator training leukemia novel phase 2 study phase I trial programs research and development response safety outcomes success therapy development

项目摘要

Project Summary & Abstract Dysregulated or dysfunctional immunity is well documented in human disease states ranging from auto- immunity to infection and cancer. A deeper understanding of the role of the immune system in human disease brings with it the real potential of immune-directed cellular therapies. However, the complexity and expense associated with the generation of cell therapies that are both patient- and disease-specific prohibit broad application at the current time. Progress in the setting of rare pediatric conditions is further hampered by the fact that the financial returns on investment are in many cases not considered favorable for industry-sponsored research and development. This U01 Innovative Award application is designed to accelerate the translation of cellular immunotherapies to treat disorders that affect children and adolescents through the establishment of the Consortium for Pediatric Cellular Immunotherapy comprised of quaternary care pediatric hospitals affiliated with their Clinical and Translational Science Award (CTSA) programs. We aim to accelerate the implementation of engineered cellular therapeutic products for cancer (including chimeric-antigen receptor-T cell therapy and NK cell therapy) or selected immune cellular therapies for treatment of lymphoproliferative disorders, and viral diseases (viral-specific T cell therapy). In addition, we will also accelerate the novel implementation of engineered regulatory T-cells to invoke immune tolerance as a therapeutic modality for a wide range of disorders that include graft vs. host disease following allogeneic hematopoietic cell transplantation, rejection after solid organ transplantation and pediatric auto-immune diseases. We propose a multi-pronged approach to spearhead the development of cellular immunotherapy clinical trials in pediatric medicine. We aim to expand cGMP manufacturing programs with the capacity to supply products through multi-center clinical trials, to establish a centralized clinical trials/regulatory affairs coordinating office to efficiently implement cellular immunotherapy clinical research for rare pediatric diseases, to increase efficiency and reliability of analytic assays to monitor safety and clinical efficacy of cellular immunotherapy trials and to develop collaborations necessary to sustain this infrastructure beyond the life span of the U01 grant mechanism. There will be a directed focus on training the translational workforce at each participating CTSA hub and establishing standard processes and procedures that can be easily disseminated to other hubs in the future. Moreover, within we will establish key collaborations between academia and pharma to ensure long term sustainability and to broaden our advances towards applicable adult disease states. While our initial work will develop a limited consortium of CTSA sites, our long-term goal is to expand these processes to enable export of cellular immunotherapy trials to all CTSA sites and ultimately beyond.

项目概要和摘要免疫失调或功能失调在人类疾病状态中有充分的记载，对感染和癌症免疫。更深入地了解免疫系统在人类疾病中的作用带来了免疫导向细胞疗法的真实的潜力。然而，复杂性和费用与患者和疾病特异性的细胞疗法的产生相关，目前的应用。罕见儿科疾病的进展进一步受到事实上，在许多情况下，投资的财务回报并不被认为是有利于行业赞助的研发的U 01创新奖申请旨在加速以下内容的翻译细胞免疫疗法，以治疗影响儿童和青少年的疾病，儿科细胞免疫治疗联盟由四级护理儿科医院组成临床和转化科学奖（CTSA）。我们的目标是加快用于癌症的工程化细胞治疗产品（包括嵌合抗原受体-T 细胞疗法和NK细胞疗法）或选择的免疫细胞疗法疾病和病毒性疾病（病毒特异性T细胞疗法）。此外，我们还将加快小说实施工程化的调节性T细胞以引起免疫耐受作为治疗模式，包括同种异体造血细胞移植后的移植物抗宿主病在内的多种疾病移植、实体器官移植后排斥反应和儿科自身免疫性疾病。我们提出了一个多管齐下的方法，率先发展细胞免疫治疗的临床试验，儿科医学我们的目标是扩大cGMP生产计划，以提供产品通过多中心临床试验，建立集中的临床试验/法规事务协调办公室，高效开展儿科罕见病细胞免疫治疗临床研究，提高效率分析测定的可靠性，以监测细胞免疫治疗试验的安全性和临床疗效，发展必要的合作，以在U 01赠款的有效期之后维持这一基础设施机制将有一个直接的重点培训翻译劳动力在每个参与CTSA 建立标准流程和程序，便于向联合国其他中心传播。未来此外，我们将在学术界和制药业之间建立关键的合作，以确保长期的长期可持续性，并扩大我们对适用的成人疾病状态的进展。虽然我们最初的工作我们将开发一个有限的CTSA网站联盟，我们的长期目标是扩大这些流程，将细胞免疫治疗试验输出到所有CTSA研究中心，并最终输出到其他研究中心。