Montelukast as a novel therapeutic approach in metastatic uveal melanoma harboring a CYSLTR2 mutation: a translational case report
Montelukast led to sustained disease stabilization for >12 months, with excellent tolerability and no reported adverse events.
Uveal melanoma (UM), the most common primary intraocular malignancy in adults, has limited systemic treatment options in the metastatic setting. Recent insights into cysteinyl leukotriene receptors (CysLTRs) – particularly CYSLTR2 mutations (prevalence 2%–4%) – suggest new therapeutic approaches for patients who progress despite standard therapies. In this case, a 59‑year‑old male with metastatic UM harboring a CYSLTR2 mutation achieved disease stabilization for >12 months on off‑label montelukast after multiple prior therapies including immune checkpoint inhibitors, chemotherapy, and radiotherapy.
Given the lack of effective treatment options in this population, cysteinyl leukotriene receptor antagonists warrant further preclinical and clinical exploration as a targeted therapeutic strategy in UM.
Evidence level: Megerősített klinikai bizonyíték. Több vagy erősebb humán vizsgálat támogatja.
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