CONNECT: Comprehensive Oncology Network Evaluating Rare CNS Tumors - Cures

我们的NCI-CONNECT项目包括一个由全国33个机构组成的网络，帮助我们招募患者进行临床试验和研究。在与医学科学与计算达成具有里程碑意义的协议后，我们的网络为两项同类首次多中心临床试验的启动发挥了重要作用，为网络的临床试验提供了前所未有的校内和校外合作机会。根据NCI谅解备忘录，我们与9个患者权益团体建立了正式合作伙伴关系，分享教育内容，提高人们对改善罕见中枢神经系统肿瘤患者预后的认识。我们与合作伙伴合作举办研讨会、会议和网络研讨会，以帮助分享患者的观点，并在过去三年中合作举办了七次活动，包括2021年6月举行的神经肿瘤学幸存者护理研讨会，有200多名与会者。NCI-CONNECT的领导还参与了倡导活动，如2022年召开的国家脑肿瘤协会生活质量研究项目，第一次研讨会于2023年5月举行。该项目将患者合作伙伴和利益相关者聚集在一起，建立以患者为中心的脑肿瘤生活质量研究议程。我们举办科学研讨会，汇集世界各地的神经肿瘤学专家和患者倡导者，讨论选定的特定罕见中枢神经系统癌症类型的科学进展和挑战，并找到改进治疗和开发新的临床试验的方法。我们发表了这些研讨会的论文——四篇论文集和一篇增刊——并改变了癌症治疗指南；值得注意的是，在2020年11月，由于我们的科学研讨会报告，NCCN髓母细胞瘤指南进行了更新。我们的NCI-CONNECT网站是一个针对患者、护理人员和专业人士的教育工具。从2022年5月到2023年5月，该网站的页面浏览量超过100万，访问量达到818122次。NCI-CONNECT于2020年推出了西班牙语版网站，以覆盖更广泛、服务不足的受众，平均每月访问量为21795次。2022年游客人数最多的10个国家包括墨西哥、西班牙、哥伦比亚、阿根廷和秘鲁。2020年3月，我们发布了一个新的管理自我保健部分，并在2021年看到我们的肿瘤生活内容的页面浏览量增长了58%。我们每月向超过16,000名订阅者发送NCI-CONNECT电子通讯。我们的私人Facebook小组NCI- connect社区——NCI的第一个社区——有633名成员；我们的YouTube播放列表有35个视频，累计播放31,416次；我们的推特账号有6957个粉丝。所有这些平台共享教育和科学信息。我们还在2021年开发了一款名为My STORI的症状负担应用程序（在苹果商店和b谷歌Play商店有售），以方便患者的症状监测和跟踪。我们的NCI-CONNECT诊所致力于将具有相似罕见中枢神经系统肿瘤类型的患者聚集在一起进行特殊服务。截至2022年1月，我们已经接待了421名患者，占我们NOB患者总数的49%。这些患者接受基因检测和咨询，用先进的分子检测对他们的诊断进行审查，并参加一个名为CARES的支持小组会议，由专门的健康和健康顾问负责。在2019冠状病毒病期间，NCI-CONNECT增加了与当地医生的合作伙伴关系，提供研究药物和远程医疗，以扩大我们的患者获得护理的机会。通过NCI-CONNECT启动的临床研究及其进展包括以下治疗试验：免疫检查点抑制剂nivolumab用于罕见中枢神经系统癌症成人的一揽子试验（17C0102），进展包括在大量预处理患者队列中进行的积极中期分析，这些结果的展示在2021年SNO年会上获得了罕见中枢神经系统疾病卓越奖；口服ONC206治疗复发性和罕见原发性中枢神经系统肿瘤的首次人体研究（20C0069）；nivolumab治疗复发性IDH突变胶质瘤（包括少突胶质胶质瘤）的研究（19C0006）；以及针对胶质肉瘤和胶质母细胞瘤患者的免疫监测研究（21C0015）。临床和观察性研究包括一项组织结局和妊娠亚研究（P194734）的回顾性研究；两项自然历史研究，一项罕见中枢神经系统（CNS）癌症成人（16C0151）的自然历史和标本库研究，使用我们的自然历史研究数据的研究在2021年SNO年会上获得了生存卓越摘要奖，并根据研究数据更新了2021年WHO室管膜瘤诊断手册；成人中枢神经系统癌症患者结局和风险的在线调查（17CN141）；在临床评估时使用沉浸式虚拟现实（VR）改善心理困扰和焦虑的试验（20C0065），中期结果于2023年3月公布，支持针对原发性脑肿瘤患者心理症状的干预的可行性和可接受性；原发性脑肿瘤患者管理癌症和有意义地生活（CALM）疗法的研究；以及一项睡眠观察研究。NCI-CONNECT正在进行广泛的生物标志物研究，作为正在进行的使用免疫检查点nivolumab治疗罕见中枢神经系统癌症的免疫治疗篮子临床试验的一部分。与神经肿瘤学分支的转化免疫学项目合作，正在通过系列血液采集对个体患者的免疫反应治疗进行广泛监测。NCI-CONNECT通过识别中枢神经系统肿瘤的新分子亚型进一步推进科学发展。例如，通过与Kenneth aldap博士领导的CCR病理学实验室团队的内部合作，NCI-CONNECT重要地发现了一种以MYCN扩增为特征的室管膜瘤新亚型。与CCR的其他研究人员的合作提出了这些癌症可能对拓扑异构酶抑制剂选择性敏感的假设，一种新的抑制剂PLX-038将在临床试验中用于这些和其他携带MYCN扩增的肿瘤。NCI-CONNECT与儿科肿瘤科、放射肿瘤科和外科神经病学科建立了新的合作关系，以开发治疗高级别和放射诱导脑膜瘤的新疗法。NCI-CONNECT联盟使我们能够在美国扩展我们的研究工作，并为我们的NCI-CONNECT诊所的患者转诊提供了一个网络。我们每年为NCI-CONNECT的成员举办几次教育活动，包括虚拟期刊俱乐部会议和现场肿瘤委员会会议，我们将这些会议与我们的科学研讨会一起视频广播给NCI-CONNECT的研究人员。2020年4月，我们扩大了每周的多学科NIH神经肿瘤学肿瘤委员会，以纳入参与NCI-CONNECT联盟网站的研究人员和病例，包括16个综合癌症中心。正如我们的数据所显示的那样，这些教育工作得到了很好的参与和参与：154个提供者向来自47个外部站点的参与者介绍了154个案例。此外，NCI-CONNECT每周举办访问学者讲座，并于2023年5月举办了第一次面对面的访问学者讲座，主题是神经肿瘤患者和护理人员的生存。此外，MyPART和NCI-CONNECT网络正在NIH临床中心合作为AYAs开发一个神经肿瘤学诊所。他们于2022年10月14日举行了一次合作研讨会，此后就临床研究思路和生存护理定期举行会议，并计划对几项临床试验进行修改，以扩大到AYAs。由詹森·莱文博士领导的信息技术团队，NCI-CONNECT正在分析患者结果调查的数据，并动态地在网上公开显示。

Our NCI-CONNECT program includes a network of 33 institutions across the nation that help us enroll patients for clinical trials and studies. Our network was instrumental to the launch of two first-of-their-kind multi-center clinical trials after a landmark agreement with Medical Science & Computing that provides unprecedented opportunity for intramural and extramural collaboration on clinical trials for the network. We have formal partnership under an NCI Memorandum of Understanding with 9 patient advocacy groups to share educational content and raise awareness to improve outcomes for people with rare CNS tumors. We collaboratively host workshops, meetings, and webinars with partners to help share the patient perspective and have collaborated on seven events in the past three years, including a Survivorship Care in Neuro-Oncology Symposium virtually in June 2021 with over 200 attendees. NCI-CONNECT leadership also participates in advocacy lead events like the National Brain Tumor Society Quality of Life Research project convened in 2022 with the first workshop held in May 2023. The project brings together patient partners and stakeholders to establish a patient-centered brain tumor QoL research agenda. We host scientific workshops that bring together neuro-oncology experts and patient advocates worldwide to discuss the scientific progress and challenges of a selected specific rare CNS cancer type and find ways to improve therapies and develop new clinical trials. We have published papers from these workshops - four proceedings papers and one supplement - and changed cancer care guidelines; notably in November 2020, the NCCN guidelines for medulloblastoma were updated due to our scientific workshop report. Our NCI-CONNECT website is an educational tool for patients, caregivers, and professionals. From May 2022 to May 2023 the website exceeded 1 million page views and had 818,122 visits. NCI-CONNECT launched a Spanish-language version of its website in 2020 to reach a broader and underserved audience, and it averages 21,795 monthly visits. The top 10 countries by visits in 2022 included Mexico, Spain, Columbia, Argentina, and Peru. In March 2020, we published a new Managing Self-Care section and saw a 58 percent growth in page views to our Living with a Tumor content in 2021. Our monthly NCI-CONNECT e-newsletter is delivered to over 16,000 subscribers. Our private Facebook group NCI-CONNECT Community - the first ever for NCI - has 633 members; our YouTube Playlist has 35 videos, accumulating 31,416 views; and our Twitter account has 6,957 followers. All these platforms share educational and scientific information. We also developed a symptom-burden app called My STORI in 2021 (available in the Apple Store and Google Play Store) to facilitate patient symptom monitoring and tracking. Our NCI-CONNECT Clinic is dedicated to bringing together patients with similar rare CNS tumor types for special services. We have seen 421 patients as of January 2022, which is 49 percent of our entire NOB patient population. These patients receive genetic testing and counseling, a review of their diagnosis with advanced molecular testing, and participate in a support group meeting called CARES with a dedicated Health and Wellness Counselor. During COVID-19, NCI-CONNECT has added partnerships with local physicians to provide study drug, and telemedicine to expand access to care for our patients. Clinical studies launched and their progress through NCI-CONNECT include these treatment trials: a basket trial testing the immune checkpoint inhibitor nivolumab for adults with rare CNS cancers (17C0102), progress includes a positive interim analysis in the heavily pre-treated patient cohort and the presentation on these results won an abstract Award for Excellence in Rare CNS Disease at the 2021 SNO Annual Meeting; a first-in-human study of oral ONC206 in recurrent and rare primary CNS neoplasms (20C0069); a study of nivolumab for patients with recurrent IDH mutant gliomas including oligodendrogliomas (19C0006); and an immune monitoring study for patients with gliosarcoma and glioblastoma (21C0015). Clinical and observational studies include one retrospective study of Tissue Outcomes and Pregnancy Sub Study (P194734); two Natural History Studies, a natural history and specimen banking study for adults with rare central nervous system (CNS) cancers (16C0151), research using data from our Natural History Study received an Abstract Award for Excellence in Survivorship at the 2021 SNO Annual Meeting, and based on study data, the 2021 WHO diagnosis book for ependymoma was updated; an online survey of outcomes and risk for patients with adult CNS cancers (17CN141); a trial using immersive Virtual Reality (VR) at the time of clinical evaluation to improve psychological distress and anxiety (20C0065), which the interim results were published in March 2023 and support feasibility and acceptability of the intervention to target psychological symptoms for primary brain tumor patients; a study on managing Cancer And Living Meaningfully (CALM) therapy in individuals diagnosed with a primary brain tumor; and a Sleep Observation Study. NCI-CONNECT is conducting extensive biomarker research as a part of the ongoing immunotherapy basket clinical trial using the immune checkpoint nivolumab for rare CNS cancers. Collaborating with the Translational Immunology Program in the Neuro-Oncology Branch, extensive monitoring of individual patient's immune response treatment is underway using serial blood collections. NCI-CONNECT is further advancing science through the identification of new molecular subtypes of CNS tumors. For example, through intramural collaborations with the CCR Laboratory of Pathology team led by Dr. Kenneth Aldape, NCI-CONNECT importantly uncovered a new subtype of ependymoma characterized by MYCN amplification. Collaborations with other investigators in CCR led to the hypothesis that these cancers may be selectively sensitive to topoisomerase inhibitors, and a novel inhibitor, PLX-038, will be tested for these and other tumors harboring MYCN amplification in a clinical trial. NCI-CONNECT established a new collaboration with the Pediatric Oncology Branch, Radiation Oncology Branch, and the Surgical Neurology Branch to develop novel therapies for high-grade and radiation-induced meningioma. The NCI-CONNECT consortium has allowed us to expand our research efforts across the United States and provides a network for patient referrals to our NCI-CONNECT Clinics. We have led educational activities for NCI-CONNECT members several times per year, including virtual journal club sessions and live tumor board sessions that we videocast to NCI-CONNECT investigators in conjunction with our scientific workshops. In April 2020, we expanded our weekly multidisciplinary NIH Neuro-Oncology Tumor Board to include investigators and cases from participating NCI-CONNECT consortium sites, including 16 Comprehensive Cancer Centers. These educational efforts have been well-attended and engaging as exhibited by our data: 154 providers have presented 154 cases to participants from 47 external sites. In addition, NCI-CONNECT holds weekly lectures from visiting scholars and in May 2023 held its first in-person visiting scholar lecture on Survivorship in Neuro-Oncology with a Patient and Caregiver. Further, MyPART and NCI-CONNECT networks are collaborating on developing a neuro-oncology clinic for AYAs at the NIH Clinical Center. They held a collaborative workshop October 14, 2022, and since are holding regular meetings on clinical research ideas and survivorship care with plans for several clinical trials to be modified to expand to AYAs. With the information technology team led by Dr. Jason Levine, NCI-CONNECT is analyzing data from its patient outcomes surveys to dynamically display it publicly online.