Current clinical trials for craniopharyngiomas: what's on the horizon?

Craniopharyngiomas are histologically low-grade tumors in the sellar/suprasellar region that grow close to critical structures including the hypothalamus, pituitary gland, and optic chiasm. Due to this challenging location, many patients face long-term complications including neuroendocrine, neurologic, and visual deficits. As a result, there is interest in developing risk-optimized treatments that minimize damage to adjacent normal tissue and limit chronic complications patients face. In recent years, numerous multi-omic characterizations of craniopharyngioma have identified potential targetable markers of craniopharyngioma. In adamantinomatous craniopharyngioma, numerous clinical trials to explore MEK, PD-1, WNT, and IL-6 inhibition are currently active. In papillary craniopharyngioma, targeting BRAF-V600E and MEK with monotherapy and combined therapies are currently being investigated. Further combining of these therapies with radiation and surgical techniques have potential to change existing treatment paradigms and improve the long-term outcome for patients with craniopharyngioma. With our advanced understanding, clinical investigations that target identified oncogenic drivers of craniopharyngioma should continue to center on therapy options that minimize complications faced by patients with this chronic, high morbidity disease.