Advances in Targeted Therapies Offer Hope for Pediatric Glioma Treatment

Recent findings from a collaborative study between Dana-Farber Cancer Institute and the Broad Institute have shed light on promising targeted treatment options for pediatric gliomas, the most common type of brain tumor in children. The research revealed that approximately 8.9% of pediatric glioma cases feature alterations in the fibroblast growth factor receptor (FGFR) family of proteins, particularly FGFR1 and FGFR2. These genetic changes, which include point mutations and structural variants, suggest that many of these tumors may be susceptible to existing FDA-approved FGFR inhibitors.

The study involved comprehensive genomic analyses of 11,635 gliomas across various age groups, uncovering that FGFR alterations are notably prevalent in low-grade gliomas, especially in children. To investigate further, researchers developed innovative models using neural stem cells with FGFR alterations, demonstrating that these genetic changes can drive tumor development. Importantly, preclinical testing of FGFR inhibitors showed that these drugs could effectively inhibit tumor growth.

Retrospective clinical observations also indicated that some children with FGFR-altered gliomas experienced disease stabilization upon treatment with FGFR-targeting medicines. Although no current FDA-approved therapies or clinical trials specifically target FGFR alterations in pediatric gliomas, this research points toward personalized treatment strategies that could be less toxic and more effective than conventional therapies.

Dr. Pratiti (Mimi) Bandopadhayay, a senior author of the study, emphasized the clinical relevance, stating, "This research provides essential insights that could impact treatment options for children diagnosed with FGFR-altered gliomas worldwide." The team plans to refine these targeted therapies, enhance drug delivery to the brain, and initiate clinical trials to evaluate their safety and efficacy further.

This groundbreaking work builds on prior successes, such as the approval of tovorafenib for BRAF-altered childhood gliomas, and represents a significant step toward precision medicine in pediatric neuro-oncology.

Source: https://medicalxpress.com/news/2025-07-therapies-pediatric-gliomas.html