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Prolonged disease control with MEK inhibitor in neurofibromatosis type I-associated glioblastoma
M Ameratunga1, G McArthur2,3,4, H Gan1
1Department of Medical Oncology, Austin Health, Heidelberg, Vic, Australia.
What Is Known And Objective:
Neurofibromatosis is associated with overactivation of the RAS-MAPK pathway. MEK inhibitors have been shown to be an effective treatment modality in other malignancies.
Case Summary:
We present a 24-year-old male with treatment-refractory neurofibromatosis-associated glioblastoma, who experienced clinical and radiological benefit from the MEK inhibitor, trametinib.
What Is New And Conclusion:
This case highlights the therapeutic success of a MEK inhibitor in neurofibromatosis-associated glioblastoma. As a corollary, this should prompt evaluation of MEK inhibitors in tumours associated with neurofibromatosis. It remains to be elucidated if tumours with somatic NF1 mutations may also benefit from therapy targeting the RAS-MAPK pathway.
Insights
Neurofibromatosis-associated glioblastoma responded well to trametinib, a MEK inhibitor. This suggests MEK inhibitors may be effective for neurofibromatosis-related tumors.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Neurofibromatosis is linked to RAS-MAPK pathway overactivation.
- MEK inhibitors are effective in other cancers.
Observation:
- A 24-year-old male with refractory neurofibromatosis-associated glioblastoma was treated.
- The patient received the MEK inhibitor trametinib.
Findings:
- The patient showed clinical and radiological improvement.
- Trametinib demonstrated therapeutic success in this case.
Implications:
- MEK inhibitors warrant evaluation for neurofibromatosis-associated tumors.
- Further research is needed for tumors with somatic NF1 mutations.
