Treatment of BRAF V600E mutated ganglioglioma of the third ventricle with dabrafenib

Abstract

Background: Ganglioglioma (GG) of the third ventricle is rare. Surgical excision of tumors in this location is associated with high morbidity due to nearby eloquent brain centers. Alternative treatments, when available, should be considered to reduce risks of surgical treatment. Case Description: We present the case of a 21-year-old female diagnosed with a BRAF V600E mutated GG of the third ventricle. After an endoscopic biopsy and insertion of a ventriculoperitoneal shunt, the patient was started on the BRAF inhibitor dabrafenib, as an alternative to surgery or radiation. Nearly 2 years after starting dabrafenib, her tumor appearance on serial magnetic resonance imaging is stable, and she has maintained a good quality of life with no new neurological symptoms. Conclusion: The disease control thus far suggests targeted medical therapy of GG of the third ventricle with BRAF inhibitors may have efficacy and should be a considered treatment modality.

Insights Into Vascular Anomalies, Cancer, and Fibroproliferative Conditions: The Role of Stem Cells and the Renin-Angiotensin System

“…Low-dose AKT inhibitor miransertib (ARQ092) reduces the levels of phospho-AKT by approximately half in 83% of tissue samples of patients with Proteus syndrome, caused by gain-of-function mutations in AKT in the PI3KCA/AKT/mTOR pathway ( 117 ). Targeted therapies that inhibit MAPK2K1/ERK for these mutations have been investigated in many cancer types, including metastatic MM ( 118 ), advanced soft tissue sarcoma ( 119 ), and ganglioglioma ( 120 ). Targeting mutations in MEK1/2 (MAP2K1) have also been proposed for VAs.…”