Accelerate cellular immunotherapy development for treatment of life-threatening childhood disorders
Accelerate cellular immunotherapy development for treatment of life-threatening childhood disorders
批准号:
10251074
负责人:
JULIE R PARK
金额:
$139.08万
依托单位国家:
美国
项目类别:
财政年份:
2018
资助国家:
美国
项目状态:
已结题
起止时间:
2018-08-01 至 2023-07-31
关键词:
AcademiaAccelerated PhaseAdolescentAdultAffectAllogenicAutoimmune DiseasesAutoimmunityAwardBiologicalBiological AssayCAR T cell therapyCaringCell TherapyCellsCellular immunotherapyChildChildhoodClinical ResearchClinical TrialsClinical and Translational Science AwardsCollaborationsDiseaseEligibility DeterminationEngineeringEnsureFutureGenerationsGoalsGraft RejectionGrantImmuneImmune ToleranceImmune systemImmunityImmunotherapeutic agentIndustryInfectionInfrastructureInstitutionInvestigational DrugsInvestigational New Drug ApplicationInvestmentsLeadLifeLongevityLymphoproliferative DisordersMalignant NeoplasmsMedicineMinority GroupsModalityMonitorMulti-Institutional Clinical TrialNK cell therapyOrgan TransplantationPatientsPediatric HospitalsPharmaceutical PreparationsPilot ProjectsPositioning AttributeProceduresProcessProductionRare DiseasesRefractoryRegulatory AffairsRegulatory T-LymphocyteResearch PersonnelRoleSafetySiteSolidT cell therapyTherapeuticTimeTrainingTranslational ResearchTranslationsUnderserved PopulationViralVirus DiseasesWorkbasecellular developmentcellular engineeringclinical developmentclinical efficacyclinical research sitedesigngraft vs host diseasehematopoietic cell transplantationhigh riskhuman diseaseimmunoengineeringimmunotherapy clinical trialsimmunotherapy trialsinnovationinnovative technologiesinvestigator trainingleukemianovelphase 2 studyphase I trialprogramsresearch and developmentresponsesafety outcomessuccesstherapy development
中文摘要
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
我们将开发有限的CTSA站点联盟,我们的长期目标是扩展这些流程,以实现
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。
英文摘要
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.
期刊论文(0)
专著(0)
科研奖励(0)
会议论文
Accelerate cellular immunotherapy development for treatment of life-threatening childhood disorders
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批准号:9750848
-
项目类别:
-
资助金额:$141.62万
-
财政年份:2018
-
负责人:JULIE R PARK
-
依托单位:
Accelerate cellular immunotherapy development for treatment of life-threatening childhood disorders
-
批准号:10460283
-
项目类别:
-
资助金额:$139.56万
-
财政年份:2018
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负责人:JULIE R PARK
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依托单位:
Clinical Trials and Translation
-
批准号:10017939
-
项目类别:
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资助金额:$36.74万
-
财政年份:2017
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负责人:JULIE R PARK
-
依托单位:
Clinical Trials and Translation
-
批准号:10265476
-
项目类别:
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资助金额:$33.56万
-
财政年份:2017
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负责人:JULIE R PARK
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依托单位:
COG ADVL0413: A PHASE I STUDY OF THE RAF KINASE AND RECEPTOR TYROSINE KINASE
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批准号:7603585
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项目类别:
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资助金额:$0.03万
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财政年份:2007
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负责人:JULIE R PARK
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依托单位:
COG ADVL0416: A PHASE 1 STUDY OF SAHA IN PEDIATRIC PATIENTS WITH RECURRENT
-
批准号:7603565
-
项目类别:
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资助金额:$0.02万
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财政年份:2007
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负责人:JULIE R PARK
-
依托单位:
NANT 2003-01: A PHASE I STUDY OF ORAL IRINOTECAN, TEMOZOLOMIDE, AND CEFIXIME
-
批准号:7603541
-
项目类别:
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资助金额:$0.01万
-
财政年份:2007
-
负责人:JULIE R PARK
-
依托单位:
NANT 2001-03: PHASE 1 STUDY OF CEP-701 IN PATIENTS WITH REFRACTORY NEUROBLASTOMA
-
批准号:7603529
-
项目类别:
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资助金额:$0.02万
-
财政年份:2007
-
负责人:JULIE R PARK
-
依托单位:
ADVL0319 PHASE I STUDY OF CC-5013 (LENALIDOMIDE) IN PEDIATRIC PATIENTS
-
批准号:7603559
-
项目类别:
-
资助金额:$0.08万
-
财政年份:2007
-
负责人:JULIE R PARK
-
依托单位:
NANT 2004-01: A PHASE I STUDY OF ZOLEDRONIC ACID (ZOMETA) WITH CYCLOPHOSPHAMIDE
-
批准号:7603561
-
项目类别:
-
资助金额:$0.02万
-
财政年份:2007
-
负责人:JULIE R PARK
-
依托单位:
COG ADVL0419: VALPROIC ACID IN CHILDREN WITH RECURRENT/PROGRESSIVE SOLID TUMORS
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批准号:7603578
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项目类别:
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资助金额:$0.08万
-
财政年份:2007
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负责人:JULIE R PARK
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依托单位:
COG ADVL0214: A PHASE I STUDY OF SINGLE AGENT OSI-774 (TARCEVA)
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批准号:7379432
-
项目类别:
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资助金额:$0.25万
-
财政年份:2006
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负责人:JULIE R PARK
-
依托单位:
NANT 2001-03: PHASE 1 STUDY OF CEP-701 IN PATIENTS WITH REFRACTORY NEUROBLASTOMA
-
批准号:7379410
-
项目类别:
-
资助金额:$0.17万
-
财政年份:2006
-
负责人:JULIE R PARK
-
依托单位:
ADVL0319 PHASE I STUDY OF CC-5013 (LENALIDOMIDE) IN PEDIATRIC PATIENTS
-
批准号:7379444
-
项目类别:
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资助金额:$0.08万
-
财政年份:2006
-
负责人:JULIE R PARK
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依托单位:
COG ADVL0416: A STUDY OF SAHA IN PEDIATRIC PATIENTS WTH RECURRENT SOLID TUMORS
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批准号:7379449
-
项目类别:
-
资助金额:$0.25万
-
财政年份:2006
-
负责人:JULIE R PARK
-
依托单位:
NANT 2004-01: A PHASE I STUDY OF ZOLEDRONIC ACID (ZOMETA) WITH CYCLOPHOSPHAMIDE
-
批准号:7379446
-
项目类别:
-
资助金额:$0.25万
-
财政年份:2006
-
负责人:JULIE R PARK
-
依托单位:
NANT 2003-01: A PHASE I STUDY OF ORAL IRINOTECAN, TEMOZOLOMIDE, AND CEFIXIME
-
批准号:7379424
-
项目类别:
-
资助金额:$0.25万
-
财政年份:2006
-
负责人:JULIE R PARK
-
依托单位:
COG ADVL0316: A PHASE I STUDY OF 17-AAG IN RELAPSED/REFRACTORY PEDIATRIC
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批准号:7379433
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项目类别:
-
资助金额:$0.46万
-
财政年份:2006
-
负责人:JULIE R PARK
-
依托单位:
COG ADVL0314: A PHASE 1 STUDY OF BEVACIZUMAB IN REFRACTORY SOLID TUMORS
-
批准号:7379415
-
项目类别:
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资助金额:$0.83万
-
财政年份:2006
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负责人:JULIE R PARK
-
依托单位:
NANT 2002-01 PHASE 1 STUDY OF HIGH DOSE PYRASOLACRIDINE (PZA)(NSC 366140) SUPPOR
-
批准号:7379411
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项目类别:
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资助金额:$0.33万
-
财政年份:2006
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负责人:JULIE R PARK
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依托单位:
海外基金