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Recent Advancements in Ependymoma: Challenges and Therapeutic Opportunities
Kelsey C Bertrand1, Paul Klimo2,3,4
1Division of Neuro-oncology, Department of Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA, Kelsey.Bertrand@STJUDE.org.
Background:
Ependymoma is one of the most common malignant pediatric brain tumors and can be difficult to treat. Over the last decade, much progress has been made in the understanding of the underlying molecular drivers within this group of tumors, but clinical outcomes remain unchanged.
Summary:
Here, we review the most recent molecular advances in pediatric ependymoma, evaluate results of recent clinical trials and discuss the ongoing challenges in the field and questions that remain.
Key Messages:
The field of ependymoma has vastly changed over the last several decades with ten distinct molecular subgroups now described, but much progress needs to be made in developing new therapeutic strategies and targets.
Insights
Pediatric ependymoma, a common brain tumor, has seen molecular advances but unchanged outcomes. New therapeutic strategies are needed to improve treatment for this challenging disease.
Area of Science:
- Neuro-oncology
- Pediatric oncology
- Molecular pathology
Background:
- Ependymoma is a frequent and challenging malignant pediatric brain tumor.
- Despite molecular understanding advances, clinical outcomes have not improved.
Purpose of the Study:
- To review recent molecular discoveries in pediatric ependymoma.
- To evaluate current clinical trial results and identify challenges.
Main Methods:
- Literature review of molecular advances.
- Analysis of recent pediatric ependymoma clinical trials.
Main Results:
- Ten distinct molecular subgroups of ependymoma are now recognized.
- Progress in molecular understanding has not yet translated to improved patient outcomes.
Conclusions:
- Significant advancements in molecular subtyping of ependymoma have been made.
- New therapeutic strategies and molecular targets are crucial for improving treatment efficacy.

