Molecular Targets in Craniopharyngioma

Craniopharyngiomas have been histologically categorized into adamantinomatous (ACP) and papillary (PCP) subtype of craniopharyngioma. However, recent developments in molecular and genetic analysis have identified specific mutations in each, β-catenin in ACP and BRAF mutation in PCP. Furthermore, these developments have provided a deeper insight into the origin and pathology of this tumour and opened a new field of treatment opportunities. Recent findings indicate connection between stem cells and ACP and suggest a paracrine model in which pituitary stem cells drive neoplastic proliferation of nearby epithelial cells through growth factor signalling. Investigation of molecular and genetic alterations in CPs has identified several biomarkers that have paved the way for new possibility to predict the biological behaviour of this tumour as well as early diagnosis of recurrence and new treatment options. Currently, the most promising adjuvant treatment is offered by dual therapy with BRAF and MEK inhibitors in PCPs expressing BRAFV600E mutation. So far this has been reported as case studies, hence, ongoing and upcoming larger clinical trials are highly anticipated to provide more information on this treatment option and its long-term efficacy.It might be possible that in the future, emerging treatments may be applied to reduce the tumour size and facilitate total surgical removal of the tumours potentially improving the outcome. Or even more exciting, simple analysis of tumour markers in serum and/or cerebrospinal fluid in combination with MR imaging would provide sufficient information on diagnosis and available targeted therapy could be applied precluding any surgical intervention