MyPART: My Pediatric and Adult Rare Tumor Network - Cures

MyPART：我的儿科和成人罕见肿瘤网络 - Cures

基本信息

批准号：
10487019
负责人：
Brigitte Widemann
金额：
$ 2185.62万
依托单位：
DIVISION OF BASIC SCIENCES - NCI
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10487019
关键词：
Acinar Cell Carcinoma Adolescent Adolescent and Young Adult Adrenocortical carcinoma Adult Advocate Affect Alveolar Soft Part Sarcoma Antibodies Back Biological Models Biology Biopsy Blood COVID-19 pandemic Cancer Therapy Evaluation Program Caregivers Child Childhood Chordoma Clinic Clinical Clinical Trials Clinical Trials Network Collaborations Communication Programs Communities Data Databases Development Developmental Therapeutics Program Diagnosis Education Educational workshop Engineering Enrollment Evaluation Experimental Models Family Funding Gastrointestinal Stromal Tumors Goals Health Personnel Histology Home Image Immune Infrastructure International Knowledge Learning Link Malignant Childhood Neoplasm Malignant Neoplasms Medical Medical History Molecular NCI Center for Cancer Research Natural History Neuroendocrine Tumors Newsletter Nucleic Acids Olfactory Neuroblastoma Operative Surgical Procedures Organoids PDL1 inhibitors Pancreas Participant Patient Outcomes Assessments Patients Pediatric Oncology Persons Population Positioning Attribute Pre-Clinical Model Process Protocols documentation Quality of life Recommendation Research Research Personnel Resources SMARCA4 gene SMARCB1 gene Sampling Solid Neoplasm Specimen Testing Time Translating Twitter United States National Institutes of Health Work advocacy organizations analysis pipeline base benefit sharing biobank clinical center cohort cytokine early phase clinical trial effective therapy improved medical specialties medullary thyroid carcinoma member mouse model novel therapeutics patient derived xenograft model patient engagement phase II trial pre-clinical rare cancer research clinical testing sarcoma single cell sequencing symposium treatment trial tumor web site working group

项目摘要

Through collaboration between MyPART, the Center for Cancer Research (CCR) Rare Tumor Initiative (RTI), and the CCR Pediatric Oncology Branch (POB), we have developed a rare tumor natural history and biospecimen trial to study rare solid tumors in pediatric, adolescent, and adult patients. The Natural History Study of Rare Solid Tumors (NCT03739827) has been open for enrollment since January 2019 and has enrolled more than 330 patients with a variety of very rare solid tumors. Patients can enroll by providing information from their home (field cohort) or by coming to the NIH Clinical Center to be seen by expert clinicians and other healthcare providers (clinic cohort) for a more extensive work up. Anyone with a rare solid tumor can participate in the field cohort and patients will be invited to come the NIH Clinical Center to join the clinic cohort based on the details of their medical history. The ability to enroll participants remotely allowed us to continue to remain active during the COVID pandemic where we were not in the position to bring most patients to the NIH Clinical Center for evaluation. Sub-protocols established under the Natural History Study allow the infrastructure of the master study to be used by both MyPART and non-MyPART investigators to carry out more in-depth studies of specific rare tumor types. A sub-protocol for chordoma has opened and has enrolled over 45 participants. Additional sub-protocols for neuroendocrine tumors (51 patients enrolled) and adrenocortical carcinoma (58 patients enrolled) have been developed and are open to enrollment. Subprotocols for olfactory neuroblastoma and pancreatic acinar cell carcinoma are currently in development. Participants in the main study and sub-protocols are followed longitudinally over time. The protocol database is established and holds a wide range of information including patient reported outcomes, medical history, tumor specimens, imaging information, and clinical evaluations performed at the NIH Clinical Center. A research analysis pipeline for blood and tumor samples has been developed for nucleic acids, immune characterization, and cytokines. All information provided is reviewed and a clinical recommendation is given, when feasible. In addition, we have established a molecular tumor board we are returning molecular tumor data to patients. Research tumor sequencing is ongoing. For patients who undergo surgery at the NIH Clinical Center for clinical reasons, we are comprehensively analyzing tumors including single cell sequencing and we are establishing preclinical models (patient derived xenografts and organoid lines). Our collaboration established with the NCI Developmental Therapeutics Clinic is active and we have 5 new treatment trials opened, including a new trial of the PD-L1 inhibitor atezolizumab that has opened for children and adolescents with specific rare sarcomas, following its Breakthrough Therapy Designation by the FDA for alveolar soft part sarcoma. In addition to histology-specific trials, the Developmental Therapeutics Clinic has early clinical trials open to patients with many different types of rare tumors. Eight additional clinical trials for very rare tumors are in development including a phase II trial of a TIGIT antibody and a PD-L1 inhibitor for children and adults with SMARCB1- or SMARCA4-deficient tumors. This trial will be a collaboration between MyPART and the NCI-CTEP Pediatric Early Clinical Trials Network, with MyPART investigators serving as PIs and several NCI Center for Cancer Research (CCR) Investigators contributing to enrollment on 6 rare solid tumor strata. In 2019 and 2020 MyPART held specialty rare tumor clinics for patients with "wild-type" (SDH-deficient) GIST (gastrointestinal stromal tumor), medullary thyroid carcinoma, and pediatric chordoma. These clinics assemble clinicians, researchers, patients, families, and advocates from across the US and the world. Patients and families benefit from receiving medical opinions from world experts in their tumor while also having the opportunity to meet others with the same rare tumor diagnosis, often for the first time. Clinicians and researchers benefit from sharing their knowledge about a particular rare tumor, which has also led to increased collaboration between groups. Due to the COVID pandemic we have not been able to hold rare tumor clinics in person at the NIH Clinical Center, but we had several remote clinics. New, and hopefully in person clinics, for additional rare tumors are planned. MyPART has also made progress in pre-clinical rare tumor research. Work is ongoing to engineer a mouse model of SDH-deficient GIST that can be used to learn more about this rare tumor and test potential therapies. With its focus on patient engagement, MyPART has established a robust communications program. We have a website (www.cancer.gov/mypart) that provides information on a number of rare tumors, the Natural History Study of Rare Solid Tumors, support for patients and their caregivers, and links to additional resources. We also send out a monthly newsletter and have a Twitter account we share with the POB. MyPART has established partnerships with 17 advocacy organizations in order build strong relationships with the rare tumor community and to provide education and information to rare tumor patients and their families about the importance of participating in rare tumor research to accelerate the development of new and effective therapies. During the past year we have conducted a landscape analysis of rare tumor efforts nationally and internationally and we plan to host a rare tumor symposium for groups with specific rare tumor efforts workshop in 2022. Efforts of My PART align well with the goals of the NCI Childhood cancer Data Initiative (CCDI) and MyPART members are actively engaged with several CCDI working groups.

通过 MyPART、癌症研究中心 (CCR) 罕见肿瘤计划 (RTI) 和 CCR 儿科肿瘤科 (POB) 之间的合作，我们开发了一项罕见肿瘤自然史和生物样本试验，以研究儿科、青少年中的罕见实体瘤和成年患者。罕见实体瘤自然史研究（NCT03739827）自2019年1月起开放招募，已入组超过330名患有各种非常罕见实体瘤的患者。患者可以通过在家中提供信息（现场队列）或前往 NIH 临床中心接受专家临床医生和其他医疗保健提供者（临床队列）的检查来进行更广泛的检查。任何患有罕见实体瘤的人都可以参加现场队列，并且将根据患者的病史详细信息邀请患者前往 NIH 临床中心加入临床队列。远程招募参与者的能力使我们能够在新冠病毒大流行期间继续保持活跃，当时我们无法将大多数患者带到 NIH 临床中心进行评估。自然历史研究下建立的子方案允许 MyPART 和非 MyPART 研究人员使用主研究的基础设施，对特定的罕见肿瘤类型进行更深入的研究。脊索瘤的子方案已经开放，并已招募了超过 45 名参与者。针对神经内分泌肿瘤（入组 51 名患者）和肾上腺皮质癌（入组 58 名患者）的其他子方案已经制定并开放入组。嗅神经母细胞瘤和胰腺腺泡细胞癌的子方案目前正在开发中。随着时间的推移，对主要研究和子方案的参与者进行纵向跟踪。该方案数据库已建立并保存了广泛的信息，包括患者报告的结果、病史、肿瘤标本、影像信息以及在 NIH 临床中心进行的临床评估。已经开发了针对核酸、免疫特征和细胞因子的血液和肿瘤样本的研究分析管道。所有提供的信息都会经过审查，并在可行的情况下给出临床建议。此外，我们还建立了分子肿瘤委员会，将分子肿瘤数据返回给患者。肿瘤测序研究正在进行中。对于因临床原因在 NIH 临床中心接受手术的患者，我们正在全面分析肿瘤，包括单细胞测序，并正在建立临床前模型（患者来源的异种移植物和类器官系）。我们与 NCI 发展治疗诊所建立的合作非常活跃，我们已启动 5 项新的治疗试验，其中包括一项针对患有特定罕见肉瘤的儿童和青少年的 PD-L1 抑制剂 atezolizumab 的新试验，该试验已被美国国家癌症研究所 (NCI) 授予突破性疗法称号。 FDA 用于治疗肺泡软组织肉瘤。除了组织学特异性试验外，发育治疗诊所还向许多不同类型的罕见肿瘤患者开放早期临床试验。另外八项针对非常罕见肿瘤的临床试验正在进行中，其中包括针对患有 SMARCB1 或 SMARCA4 缺陷肿瘤的儿童和成人的 TIGIT 抗体和 PD-L1 抑制剂的 II 期试验。该试验将由 MyPART 和 NCI-CTEP 儿科早期临床试验网络合作进行，MyPART 研究人员将担任 PI，数名 NCI 癌症研究中心 (CCR) 研究人员将参与 6 种罕见实体瘤分层的入组工作。 2019 年和 2020 年，MyPART 为“野生型”（SDH 缺乏）GIST（胃肠道间质瘤）、甲状腺髓样癌和小儿脊索瘤患者举办了罕见肿瘤专科门诊。这些诊所聚集了来自美国和世界各地的临床医生、研究人员、患者、家属和倡导者。患者和家属可以从世界肿瘤专家的医疗意见中受益，同时也有机会与具有相同罕见肿瘤诊断的其他人见面（通常是第一次）。临床医生和研究人员受益于分享他们关于特定罕见肿瘤的知识，这也促进了团体之间的合作。由于新冠病毒大流行，我们无法在 NIH 临床中心亲自举办罕见肿瘤诊所，但我们有几个远程诊所。计划针对其他罕见肿瘤开设新的诊所，希望是在个人诊所。 MyPART在临床前罕见肿瘤研究方面也取得了进展。目前正在努力设计 SDH 缺陷型 GIST 小鼠模型，该模型可用于更多地了解这种罕见肿瘤并测试潜在的疗法。 MyPART 注重患者参与，建立了强大的沟通计划。我们有一个网站 (www.cancer.gov/mypart)，提供有关多种罕见肿瘤的信息、罕见实体瘤的自然历史研究、对患者及其护理人员的支持以及其他资源的链接。我们还每月发送一份时事通讯，并与 POB 共享一个 Twitter 帐户。 MyPART 已与 17 个倡导组织建立了合作伙伴关系，以便与罕见肿瘤社区建立牢固的关系，并向罕见肿瘤患者及其家人提供有关参与罕见肿瘤研究以加速开发新的有效疗法的重要性的教育和信息。在过去的一年里，我们对国内和国际上的罕见肿瘤工作进行了景观分析，并计划在 2022 年为具有特定罕见肿瘤工作研讨会的团体举办一次罕见肿瘤研讨会。My PART 的努力与 NCI Childhood 的目标非常吻合癌症数据倡议 (CCDI) 和 MyPART 成员积极参与多个 CCDI 工作组的工作。