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Vemurafenib treatment of BRAF V600E-mutated malignant peripheral nerve sheath tumor
1From the Swedish Cancer Institute, Seattle, Washington.
Abstract:
No effective systemic treatment exists for malignant peripheral nerve sheath tumors (MPNSTs). These tumors have been reported to show increased activity in the mitogen-activated protein kinase pathway from the loss of neurofibromatosis-1 regulation and occasionally from BRAF V600E mutation. A patient with sporadic metastatic MPNST and the BRAF V600E mutation was treated with standard doses of sorafenib and later vemurafenib and followed for response. The patient showed a rapid but modest and transient response to sorafenib and a very dramatic response to vemurafenib. This case represents the first report of successful systemic treatment of MPNST with an inhibitor of the BRAF V600E mutation. It will be important to define the general utility of this approach and related therapies in this disease.
Insights
Malignant peripheral nerve sheath tumors (MPNSTs) lack effective systemic treatments. A BRAF V600E-mutated MPNST patient responded dramatically to vemurafenib, suggesting targeted therapy potential.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive cancers with no established systemic treatments.
- MPNSTs often exhibit dysregulation of the mitogen-activated protein kinase (MAPK) pathway, linked to neurofibromatosis-1 loss or BRAF mutations.
Observation:
- A patient with metastatic MPNST and a BRAF V600E mutation was treated with targeted therapies.
- Initial treatment with sorafenib yielded a modest, transient response.
- Subsequent treatment with vemurafenib resulted in a dramatic clinical response.
Findings:
- This case is the first to report successful systemic treatment of MPNST using a BRAF V600E inhibitor.
- Vemurafenib demonstrated significant efficacy in a patient with BRAF V600E-mutated metastatic MPNST.
Implications:
- Targeted inhibition of BRAF V600E may represent a viable therapeutic strategy for a subset of MPNST patients.
- Further clinical trials are warranted to evaluate the efficacy of BRAF inhibitors and related therapies in MPNST treatment.
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