Recurrent ganglioglioma in adults treated with BRAF inhibitors

Area of Science:

• Neuro-oncology
• Molecular diagnostics
• Targeted cancer therapy

Background:

• Ganglioglioma is a rare brain tumor.
• Temozolomide, surgery, and radiation are standard treatments but often fail in recurrent or refractory cases.
• BRAF V600E mutations are implicated in various cancers, including some gliomas.

Purpose of the Study:

• To screen for BRAF V600E mutations in patients with refractory ganglioglioma.
• To evaluate the efficacy and safety of dabrafenib, a BRAF inhibitor, in patients with BRAF V600E-positive ganglioglioma.

Main Methods:

• Screening of 13 adult patients with refractory ganglioglioma for BRAF V600E mutation.
• Treatment of mutation-positive patients with dabrafenib.
• Assessment of treatment toxicity, response, and progression-free survival.

Main Results:

• BRAF V600E mutation detected in 3 out of 13 patients (23%).
• Dabrafenib was well-tolerated, with no grade 3 or higher toxicity observed.
• Best response included stable disease in two patients and partial response in one patient.
• Median progression-free survival was 7 months (range: 4-10 months).

Conclusions:

• BRAF V600E mutation is present in a subset of refractory gangliogliomas.
• Dabrafenib demonstrates acceptable safety and clinical activity in this patient population.
• Targeted therapy with BRAF inhibitors may represent a viable treatment option for BRAF V600E-positive ganglioglioma.