Medulloblastoma is the most common malignant brain tumor in children under 18 years of age. It is classified as World Health Organization (WHO) grade IV. Four different subgroups of medulloblastomas—WNT, SHH, Group 3, and Group 4—have been identified based on molecular characteristics. Today, medulloblastoma in the pediatric population (<18 years old) has an overall 70% survival rate at five years from diagnosis, with survival rates varying based on subtype.
What are the goals of this project?
This project will analyze patient and tumor factors to determine what impacts survival in this patient population.
What is the impact of this project?
The recurrence of medulloblastoma, which occurs in approximately 20% of cases, is considered an almost completely fatal event; survival rates at 3 years have been reported at a mere 18%. Further, guidelines for treatment of relapsed medulloblastoma have not been established due to the heterogeneity of treatment that exists.In addition, recent evidence that recurrent medulloblastoma tumors share only 12% of genetic mutations found in the dominant clonal tumor cell at initial diagnosis suggests that recurrent tumors might be best treated with different targeted therapies than those used at the onset of the disease.
Why the CBTN request is important to this project?
Using data that includes demographic, genetic subtype and other tumor characteristics, treatment modality, and whole-genome sequencing information from the Children’s Brain Tumor Network (CBTN) databank, it will be possible to study the factors that impact overall and progression-free survival after the recurrence of medulloblastoma in the pediatric population by tumor subtype.
The Children's Brain Tumor Network contributed to this project by providing access to the Pediatric Brain Tumor Atlas.
- Maureen Rakovec
Chetan Bettegowda, MD, PhD
Identify key genetic pathways that are altered in brain tumors; Developing clinical trials to selectively target these pathways; Developing targeted therapies for patients with primary and metastatic tumors of the brain and spine; Ongoing clinical protocol to develop a simple blood test that can
Medulloblastomas comprises the vast majority of pediatric embryonal tumors and by definition arise in the posterior fossa, where they constitute approximately 40% of all posterior fossa tumors. Other forms of embryonal tumors each make up 2% or less of all childhood brain tumors.The clinic