Medulloblastoma is the most common malignant brain tumor in children and is classified as a grade IV tumor. Previous research has identified four different subgroups of medulloblastomas— wingless (WNT), sonic hedgehog (SHH), Group 3, and Group 4. Today, medulloblastoma in the pediatric population has an overall 70% survival rate at five years from diagnosis but survival rates vary across subtypes. In about 20% of cases, medulloblastoma reoccurs and is very often a fatal event. This project will analyze data about patients and tumors to determine what impacts survival for patients with recurrent medulloblastoma. Guidelines for treatment of relapsed medulloblastoma have not been established due to the broad variety of medulloblastoma treatments that exist. In addition, recent evidence has been found that recurrent medulloblastoma tumors share only 12% of genetic mutations found in patient’s first tumor. This genetic difference suggests that recurrent tumors might be best treated with different targeted therapies than those used at the onset of the disease. Researchers will use data that includes demographic, genetic subtype and other tumor characteristics, treatment modality, and genetic data from the Pediatric Brain Tumor Atlas. Through interrogation of all of this information, it will be possible for researchers to study what factors impact overall and progression-free survival after the recurrence of medulloblastoma in the pediatric population by tumor subtype, paving the way for the development of personalized therapies.
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 is the CBTN request 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 det
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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 clinical feature