Targeted Treatment Approaches for Children and Young Adults with Malignant Brain Tumors (High-Grade Gliomas)

High-grade gliomas are malignant brain tumors that spread rapidly and are difficult to treat. Especially in children and young adults, they often result from random changes in the genetic material of brain cells, causing cell growth to spiral out of control. Such tumors are typically treated with a combination of surgery, radiation therapy, and chemotherapy (often with the drug temozolomide). These treatments can slow tumor growth but often do not lead to a permanent cure. In this study, the patients’ tumor tissue will be comprehensively examined using modern molecular biology methods (“molecular profiling”). This includes analyses of the genetic material (DNA), gene activity (RNA), and other molecular characteristics.

The goal of this Phase 2 study is to examine the tumor tissue using state-of-the-art molecular biology methods in order to enable targeted treatments tailored to the specific tumor type. For these analyses, a tissue sample from the tumor is required, e.g., from surgery or a biopsy (tissue sample collection). During participation, in addition to the tumor tissue, blood or cerebrospinal fluid (CSF) samples will also be analyzed, and brain imaging will be performed to identify potential biomarkers that can provide information about the course of the disease or the response to therapies. Depending on which genetic alterations are found in the tumor, patients may later be enrolled in a suitable follow-up study that investigates drugs targeting these alterations.

The study is being initiated by Nationwide Children’s Hospital (USA) and is being conducted at several specialized pediatric cancer centers worldwide, including in the USA, Canada, Australia, Germany, the Netherlands, and the United Kingdom. A total of approximately 300 to 350 young people are expected to participate.

Children, adolescents, and young adults aged 1 to 39 with a newly diagnosed high-grade glioma or DIPG are eligible to participate. A tissue sample from a biopsy or surgery must be available. In addition, parental consent is required for minors. Patients may have previously undergone surgery, radiation therapy, or treatment with temozolomide or bevacizumab.