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Ref Type Journal Article
PMID (39036436)
Authors Wagle N, Sharma A, Nguyen M, Truong J, Juarez TM, Kesari S
Title Neoadjuvant combination treatment with checkpoint inhibitors, chemotherapy, and BRAF/MEK inhibitors for BRAFV600E glioblastoma results in sustained response: A case report.
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Abstract Text Radiation's confounding and adverse effects on tumor microenvironment and normal brain could potentially be delayed by upfront combination treatment. We present a patient with newly diagnosed BRAFV600E-mutant, PD-L1-positive glioblastoma treated with off-label RAF/MEK inhibitors encorafenib/binimetinib after progressing on postoperative immune checkpoint blockade and temozolomide (no radiation administered: NCT03425292). Complete response occurred 6 months after adding encorafenib/binimetinib, and clinical benefit was sustained for over 20 months. Treatment was well tolerated with manageable toxicities, with quality of life and cognitive function maintained throughout treatment. Adding encorafenib/binimetinib to immunotherapy and temozolomide conferred favorable and lasting efficacy for our BRAFV600E -mutant glioblastoma patient, justifying future studies.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E CD274 pos glioblastoma predicted - sensitive Binimetinib + Encorafenib + Nivolumab + Temozolomide Case Reports/Case Series Actionable In a clinical case study, the addition of Mektovi (binimetinib) and Braftovi (encorafenib) to treatment with the combination of Opdivo (nivolumab) and Temodar (temozolomide) resulted in a complete response after 6 months in a patient with CD274 (PD-L1), PDCD1 (PD-1)-positive glioblastoma harboring BRAF V600E, and the patient remained progression-free for at least 20 months while on treatment (PMID: 39036436). 39036436