CONNECT: Comprehensive Oncology Network Evaluating Rare CNS Tumors - Cures

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Our progress to date includes the establishment of an NCI-CONNECT network of researchers and institutions committed to the program's mission. This group meets biannually. This includes a two-day meeting in partnership with our already existing Brain Tumor Trials Collaborative (BTTC) Annual Meeting at NIH; this meeting brings together key stakeholders and investigators to discuss program advances and updates and new clinical trials designs and concepts, enabled our BTTC network to formally join NCI-CONNECT. The second focused meeting is held in conjunction with the annual Society for Neuro-Oncology meeting each November. In April 2020, our third annual meeting was postponed due to the novel coronavirus disease (COVID-19), but to date we have a robust network and infrastructure of 32 institutions and sites across the nation partnering to study select rare CNS tumors. Our progress includes establishing a mechanism for formal relationships with patient advocacy groups. We have nine advocacy partnerships with world-renowned national and international organizations. Under Memorandum of Understandings (MOUs) with NCI, these organizations partner with us to share educational content and raise awareness with the goal of improving outcomes for people with rare CNS tumors. They also partner with us to host workshops and meetings, and importantly, they bring the patient perspective and concerns to our workshops. This perspective is instrumental in our understanding and ability to develop clinical trials that benefit patients. Our partners include the American Brain Tumor Association, Brains for the Cure, Brain Tumor Network, Collaborative Ependymoma Research Network Foundation, The EndBrainCancer Initiative, Glioma Center Foundation of Poland, the International Brain Tumour Alliance, the National Brain Tumor Society, and Oligo Nation. Additionally, NCI-CONNECT is working with the Society for Neuro-Oncology, the largest international professional organization that is helping to distribute NCI-CONNECT educational materials and promote awareness of these efforts to the neuro-oncology community. Our network of investigators and advocacy partners have helped us successfully host or co-host five scientific or clinical outcomes workshops and an international patient advocacy summit at NIH. The scientific workshops 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 collaborate to improve therapies and develop new clinical trials. Histone Mutated Glioma Workshop, September 27, 2018 Oligodendroglioma Workshop, November 19, 2018 Clinical Outcomes Assessment Meeting, November 20, 2018 The Fourth Biennial Ependymoma Consensus, February 20 to 22, 2019 Fourth Biennial World Summit of Brain Tumor Patient Advocates, October 9 to 12, 2019 Medulloblastoma Workshop, November 25, 2019 These workshops are an instrumental method to develop an infrastructure to improve adult patient outcomes. We have published papers from these workshops and changed cancer care guidelines as highlighted in the accomplishments section below. Our efforts in education and outreach have been extremely successful. Since the development and launching of the NCI-CONNECT website in September 2018, this has proven to be a very important and popular online destination. Currently the website receives about 22,000 monthly visitors and of those 19,000 are unique. It also receives over 30,000 page views monthly. Our monthly NCI-CONNECT e-newsletter or blog publishes at least three new articles each month and is delivered to over 4,000 subscribers. We developed the first NCI private Facebook group and as of April 2020 have 238 members. We also are the only NCI program with a dedicated YouTube Playlist or Channel. These are substantial accomplishments to engage patients, enroll patients in clinical trials, and generally, promote our program to bring patients to NIH. Our progress includes the establishment of the NCI-CONNECT Clinic in January 2018, which is dedicated to bringing together patients with similar rare CNS tumor types for special services. As shown in the below graphic, 45 percent of our clinic patients have a rare CNS tumor. We have seen 289 patients as part of NCI-CONNECT Clinics. 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 (Coping, Advocacy, Relationships, Education, Support) with a dedicated Health and Wellness Counselor. We currently have seven active NCI-CONNECT clinical studies and trials, which altogether have accrued over 350 subjects in 2.25 years (see figure below). These include: a natural history and specimen banking study for adults with rare central nervous system (CNS) cancers (16C0151); an online survey of outcomes and risk for patients with adult CNS cancers (17CN141); a basket trial testing the immune checkpoint inhibitor nivolumab for adults with rare CNS cancers (17C0102); and targeted treatment trials including carboplatin and bevacizumab for patients with ependymoma (16C0009), sunitinib for patients with sarcoma of the central nervous system (18C0137), nivolumab for patients with recurrent IDH mutant gliomas including oligodendrogliomas (19C0006), and marizomib for RELA-fusion positive ependymoma (19C0011). Additionally, we recently activated a study to create a rare CNS cancer tumor and data repository (P194734). Our key accomplishments towards our goals and outcomes include kicking off our program by partnering with the Central Brain Tumor Registry of the United States to determine the incidence and prevalence of the 12 cancers our program focuses on. The data helped us understand these 12 CNS cancers, develop educational content and establish the need for tumor specific clinical trials and studies, workshops, and clinics. Our disease-specific scientific workshops and clinical outcomes assessment workshop noted above have successfully brought together neuro-oncology experts to collaborate to advance research and care as exemplified by the publishing of papers resulting from these workshops. Two proceedings papers in Neuro-Oncology Advances, with two more in progress, are meeting summaries and provide recommendations and action plans with the goal to discover new therapies and methods to improve adult patient outcomes. A third paper in Lancet Oncology used meeting input to show how including specific data on how patients function and their symptoms on clinical trials can help determine the benefit of a trial. Furthermore, each workshop had a Tumor Board highlighting discussions of management of patients with the respective cancers. These were recorded and are available in the Educational Center on the Society for Neuro-Oncology website. Additional manuscripts are in development as a result of the collaborations fostered by our scientific workshops, including a paper on medulloblastoma, and preclinical work describing the mechanism of action of a new class of drugs showing unique activity in the histone mutated gliomas, one of the NCI-CONNECT cancers.