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MEK inhibition in adult patients with pilocytic astrocytomas

Konstantinos Rounis1,2, Anna Falk Delgado3,4, Christopher Illies5

  • 1Karolinska Comprehensive Cancer Center, Solna, Sweden. konstantinos.rounis@ki.se.

NPJ Precision Oncology
|March 17, 2026
PubMed

Insights

Mitogen-activated protein kinase kinase (MEK) inhibitors show promise for adult pilocytic astrocytomas (PAs). This study found trametinib effective in adult PA patients, with partial responses and stable disease observed.

Area of Science:

  • Neuro-oncology
  • Molecular Biology
  • Genetics

Background:

  • Pilocytic astrocytomas (PAs) are rare primary brain tumors with poorer outcomes in adults compared to children.
  • Current treatments like surgery and radiation therapy (RT) have limitations, including toxicity.
  • PAs often show activation of the mitogen-activated protein kinase (MAPK) pathway.

Purpose of the Study:

  • To evaluate the efficacy of trametinib, a MEK1/2 inhibitor, in adult patients with pilocytic astrocytomas.
  • To assess treatment outcomes and progression-free survival in this patient cohort.

Main Methods:

  • Retrospective analysis of five adult patients (ages 30-79) with pilocytic astrocytomas or anaplastic transformation.
  • Patients received trametinib, a MEK1/2 inhibitor.
  • Tumor genetic analysis for NF-1 mutations was performed.

Main Results:

  • Three patients achieved partial responses, and two had stable disease according to RANO 2.0 criteria.
  • Neurological improvement was noted in two patients.
  • Median progression-free survival was 16.65 months.

Conclusions:

  • MEK inhibition with trametinib represents a potential effective treatment strategy for adult pilocytic astrocytomas.
  • Further research is warranted to confirm these findings in larger adult cohorts.

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