Penas-Prado Marta, Theeler Brett J, Cordeiro Brittany, Dunkel Ira J, Hau Peter, Mahajan Anita, Robinson Giles W, Willmarth Nicole, Aboud Orwa, Aldape Kenneth, Butman John A, Gajjar Amar, Kelly William, Rao Ganesh, Raygada Margarita, Siegel Christine, Romo Carlos G, Armstrong Terri S, Gilbert Mark R
Neuro-Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
Department of Neurology, Walter Reed National Military Medical Center, Bethesda, Maryland, USA.
Neurooncol Adv. 2020 Aug 17;2(1):vdaa097. doi: 10.1093/noajnl/vdaa097. eCollection 2020 Jan-Dec.
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.
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.
Working groups identified unmet needs in clinical trial design, tissue acquisition and testing, tumor modeling, and measurement of clinical outcomes.
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.
髓母细胞瘤（MB）是一种罕见的脑肿瘤，在儿童中更为常见，研究主要集中于此。成人的治疗建议主要基于回顾性数据和儿科经验；然而，这两个年龄组的分子特征和治疗耐受性有所不同。在成人中，预后工具并不理想，晚期复发很常见，长期后遗症仍未得到充分研究。治疗尚未适应分子分类的进展；因此，成人MB的生存率并未提高。
2017年，美国国立癌症研究所（NCI）获得了“癌症登月计划”（Cancer Moonshot℠）的支持，通过NCI-CONNECT项目应对患有罕见中枢神经系统肿瘤的成人所面临的挑战和未满足的需求，该项目在患者、医疗保健专业人员、研究人员和倡导组织之间建立了合作关系。2019年11月25日，NCI-CONNECT召集了顶尖临床医生和科学家参加一个研讨会，以回顾研究进展、分享科学见解并讨论成人MB的临床挑战。
工作组确定了临床试验设计、组织采集与检测、肿瘤建模以及临床结果测量等方面未满足的需求。
参与者确定了合作机会；讨论了创建一个由临床医生、研究人员和患者倡导者组成的工作组的计划；并制定了具体行动项目以加快成人MB的研究进展。
