Study participants must have a diagnosis of SHH-MB that is recurrent and confirmed both histologically and by Clinical Laboratory Improvement Amendments (CLIA)-certified methylation-based subgroup testing. a program that creates partnerships among patients, health care professionals, researchers, and advocacy businesses. 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. is usually another resource providing patient information by email and phone about varied topics such as financial assistance and second opinions. From 2001 to 2019, 31 individuals inquired about adult MB with most contacts initiated by caregivers. Resources and information were provided depending on individual need and included online support networks or local support groups, financial resources, and printed educational materials, such as the ABTA Medulloblastoma Brochure. Through social media outreach, NCI-CONNECT also gathers input on the experience of adults with MB. Twitter and Facebook users were encouraged to describe obtaining care, how they felt during treatment, quality-of-life (QoL) issues and challenges, and their experience trying to find information about adult MB. Similar to the ABTA findings, patients expressed confusion about the treatment decisions and a need for support to process information. Both groups expressed a consensus that connecting adults with Pavinetant MB with each other would be beneficial as this populace feels isolated and in need of support. Introduction to Adult MB: Challenges and Opportunities Marta Penas-Prado, MD MB is usually a malignant primary CNS tumor that accounts for less than 1% of brain tumors in adults. In patients age 15 and older, less than 150 new cases are diagnosed each year in the United States and an estimated 450 in Europe.5,6 While MB is more common in children age 14 and younger, patients between ages 15 and 39 represent about 30% of all cases.6 For the purpose of this workshop, adult MB refers to those diagnosed at age 18 or older, although the biology of the disease in late adolescents seems similar to that of adults, and MB is recognized as one of the cancers occurring Itga2 in the adolescent and young adult (AYA) populace.7 Patients age 40 and older account for less than 8% of all MB cases, but represent an important therapeutic challenge. Research priorities have been skewed toward pediatric MB and treatment recommendations in adults are frequently based on retrospective data and the pediatric experience due to limited clinical trial data and lack of randomized studies.8,9 However, the molecular features of MB in adults10,11 and their tolerance to treatment12 differ from pediatric patients, warranting research focused on adults. Despite advances in molecular classification there has been little change in treatment and, as expected, no significant improvement in survival for adult MB over the last 2 decades. Currently, approximately 30% of cases recur after therapy and survival rates are dismal.13,14 As discussed later, the introduction of smoothened (SMO) inhibitors in clinical trials for patients with recurrent MB who have the Sonic Hedgehog (SHH) subgroup (SHH-MB) has produced some modest responses but, when given as a single agent in the recurrent setting, has failed to improve survival.15 Risk stratification Pavinetant in adults still depends exclusively on clinical factors and is adapted from pediatric data, classifying patients in 2 disease categories: or (gross total resection defined as 1.5 cm2 postoperative residual tumor and absence of metastatic dissemination inside or outside the CNS), and ( 1.5 cm2 residual and/or presence of metastatic dissemination at Pavinetant diagnosis).8 Strikingly, most adults present with standard-risk disease, but some still experience recurrence (often with extraneural dissemination) and succumb to the disease. Although the understanding of molecular markers that can determine prognosis in pediatric MB has advanced considerably,11,16 these markers are either infrequent in adults (eg, mutations, and amplification), or their association with specific molecular subgroups and prognosis is usually unclear. For example, monosomy 6 is almost exclusively seen in pediatric WNT MB, but is present in both SHH and Group 4 tumors in adults; furthermore, monosomy 6 was found not to be a positive prognostic marker in adult MB. Unlike in children, 50% of adult MBs with loss of 6q and nuclear beta catenin positivity had no mutations.17mutations seem to be highly enriched in childhood SHH MB (SHH-) and confer prognostic significance within this subtype, but are infrequent in adults (SHH-) and were not.