Emerging treatments

BRAF inhibitors

The squamous papillary variant of craniopharyngioma is often associated with a mutation in the BRAF V600E gene, which may be used as a specific target for pharmacological treatments with BRAF inhibitor monotherapy (e.g., dabrafenib, vemurafenib), or the combination of a BRAF inhibitor with a MEK inhibitor (e.g., trametinib, cobimetinib).[20][72]​​​ BRAF inhibitor monotherapy and combination therapy with a BRAF inhibitor and an MEK inhibitor have shown some promise in the treatment of BRAF-V600E positive papillary craniopharyngiomas.[73][74][75]​ However, the appropriate timing of use in the treatment of newly diagnosed or recurrent tumours remains to be determined.​​ Studies have also shown positive responses to treatment with the BRAF inhibitor vemurafenib and the MEK inhibitor binimetinib in the adamantinomatous subtype of craniopharyngioma. Due to the limited number of studies and the heterogeneity of tumours in this variant, more research is needed to determine the best sequence and combinations of treatment.[72]

Other immunomodulatory therapies

There continues to be ongoing interest in targeted treatment for adamantinomatous variants of craniopharyngioma, particularly given it is the more common subtype.[76]​ Some of the targeted treatments used in prior studies include tocilizumab, pegylated interferon, and bevacizumab, with inconsistency in the clinical outcomes measured across different studies making it difficult to assess the evidence for tumour control.[72]​

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