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Translational Orthotopic Models of Glioblastoma Multiforme

Translational Orthotopic Models of Glioblastoma Multiforme

Published on: February 17, 2023

Pilocytic astrocytomas.

Miriam Bornhorst¹, Didier Frappaz², Roger J Packer¹

¹Gilbert Family Neurofibromatosis Institute and Brain Tumor Institute, Children's National Health System, Washington, DC, USA.

Handbook of Clinical Neurology

|March 8, 2016

Summary

This summary is machine-generated.

Pilocytic astrocytoma (PA) is a common pediatric brain tumor. Discoveries reveal key genetic alterations in the Ras/ERK pathway, guiding promising new targeted therapies for better outcomes.

Keywords:

BRAF NF1 glioma low grade pilocytic astrocytoma tumor

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Area of Science:

Pediatric neuro-oncology
Molecular genetics of brain tumors

Background:

Pilocytic astrocytoma (PA) is the most frequent pediatric brain tumor, characterized by slow growth and potential for spontaneous regression.
PAs typically occur in the cerebellum and chiasmatic/hypothalamic regions but can arise elsewhere in the central nervous system, with rare dissemination.
Neurofibromatosis type 1 predisposes children to PAs, particularly in the optic pathway.

Purpose of the Study:

To review the key molecular alterations in pilocytic astrocytoma.
To discuss current and emerging therapeutic strategies for pediatric PAs.
To highlight prognostic factors influencing patient survival.

Main Methods:

Review of existing literature on pilocytic astrocytoma.
Analysis of genetic alterations, focusing on the Ras/ERK pathway.
Examination of treatment modalities and survival data.

Main Results:

KIAA1549-BRAF fusions are the most frequent genetic alteration identified in the Ras/ERK pathway in PAs.
Complete surgical resection is associated with the best survival outcomes.
Pediatric 10-year survival rates exceed 90%, though adult outcomes are poorer.

Conclusions:

Targeted therapies directed at the Ras/ERK pathway represent a promising avenue for future treatment of PAs.
Understanding molecular drivers is crucial for developing effective therapeutic strategies.
Early diagnosis and complete resection significantly improve prognosis for pediatric pilocytic astrocytoma.