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 Moonshotto 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.
Original language | English (US) |
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Article number | vdaa097 |
Journal | Neuro-Oncology Advances |
Volume | 2 |
Issue number | 1 |
DOIs | |
State | Published - Jan 1 2020 |
Externally published | Yes |
Keywords
- NCI-CONNECT
- adult medulloblastoma
- clinical trials
- rare CNS tumors
- workshop
ASJC Scopus subject areas
- Surgery
- Oncology
- Clinical Neurology