Marta Penas-Prado1, Brett J Theeler1,2, Brittany Cordeiro1, Ira J Dunkel3, Peter Hau4, Anita Mahajan5, Giles W Robinson6, Nicole Willmarth7, Orwa Aboud1,8, Kenneth Aldape9, John A Butman10, Amar Gajjar6, William Kelly1, Ganesh Rao11, Margarita Raygada1, Christine Siegel1, Carlos G Romo1,8, Terri S Armstrong1, Mark R Gilbert1. 1. Neuro-Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA. 2. Department of Neurology, Walter Reed National Military Medical Center, Bethesda, Maryland, USA. 3. Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA. 4. Wilhelm Sander NeuroOncology Unit and Department of Neurology, University Hospital Regensburg, Regensburg, Germany. 5. Radiation Oncology, Mayo Clinic, Rochester, Minnesota, USA. 6. Department of Oncology, St Jude Children's Research Hospital, Memphis, Tennessee, USA. 7. American Brain Tumor Association, Chicago, Illinois, USA. 8. Brain Tumor Program, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA. 9. Laboratory of Pathology, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, Maryland, USA. 10. Radiology and Imaging Sciences, NIH Clinical Center, Bethesda, Maryland, USA. 11. Department of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Abstract
BACKGROUND: Medulloblastoma (MB) is a rare brain tumor occurring more frequently in children in whom research has been primarily focused. Treatment recommendations in adults are mainly based on retrospective data and pediatric experience; however, molecular features and treatment tolerance differ between the 2 age groups. In adults, prognostic tools are suboptimal, late recurrences are typical, and long-term sequelae remain understudied. Treatment has not adapted to molecular classification advances; thus, the survival rate of adult MB has not improved. METHODS: In 2017, the National Cancer Institute (NCI) received support from the Cancer Moonshot℠ to address the challenges and unmet needs of adults with rare central nervous system tumors through NCI-CONNECT, a program that creates partnerships among patients, health care professionals, researchers, and advocacy organizations. On November 25, 2019, NCI-CONNECT convened leading clinicians and scientists in a workshop to review advances in research, share scientific insights, and discuss clinical challenges in adult MB. RESULTS: Working groups identified unmet needs in clinical trial design, tissue acquisition and testing, tumor modeling, and measurement of clinical outcomes. CONCLUSIONS: Participants identified opportunities for collaboration; discussed plans to create a working group of clinicians, researchers, and patient advocates; and developed specific action items to expedite progress in adult MB. Published by Oxford University Press on behalf of the Society for Neuro-Oncology and the European Association of Neuro-Oncology 2020.
BACKGROUND: Medulloblastoma (MB) is a rare brain tumor occurring more frequently in children in whom research has been primarily focused. Treatment recommendations in adults are mainly based on retrospective data and pediatric experience; however, molecular features and treatment tolerance differ between the 2 age groups. In adults, prognostic tools are suboptimal, late recurrences are typical, and long-term sequelae remain understudied. Treatment has not adapted to molecular classification advances; thus, the survival rate of adult MB has not improved. METHODS: In 2017, the National Cancer Institute (NCI) received support from the Cancer Moonshot℠ to address the challenges and unmet needs of adults with rare central nervous system tumors through NCI-CONNECT, a program that creates partnerships among patients, health care professionals, researchers, and advocacy organizations. On November 25, 2019, NCI-CONNECT convened leading clinicians and scientists in a workshop to review advances in research, share scientific insights, and discuss clinical challenges in adult MB. RESULTS: Working groups identified unmet needs in clinical trial design, tissue acquisition and testing, tumor modeling, and measurement of clinical outcomes. CONCLUSIONS: Participants identified opportunities for collaboration; discussed plans to create a working group of clinicians, researchers, and patient advocates; and developed specific action items to expedite progress in adult MB. Published by Oxford University Press on behalf of the Society for Neuro-Oncology and the European Association of Neuro-Oncology 2020.
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