Adamantinomatous craniopharyngioma in the molecular age and the potential of targeted therapies: a review.

Pediatric adamantinomatous craniopharyngiomas (ACPs) are histologically benign brain tumors that often follow an aggressive clinical course. Arising in the sellar/suprasellar region, they grow in close proximity to critical neurological and vascular structures and can result in significant neuroendocrine morbidity. First-line treatment often involves surgical resection with or without radiotherapy and has been associated with significant morbidity and poor quality of life outcomes. As a result, the discovery of alternative effective and safe treatments is clearly desirable. In recent years, laboratory studies have harnessed sophisticated techniques to identify the upregulation of several markers that may represent potential therapeutic targets. These targets include IL-6, PD1/PD-L1, MEK, IDO-1, and others. Agents that target these pathways exist, and there is an opportunity to investigate their potential efficacy in the treatment of ACP. Trials investigating some of these agents as monotherapy and in combination for the treatment of pediatric ACP are underway or in development. If positive, these trials may result in a paradigm shift in treatment that will hopefully result in reduced morbidity and better outcomes for patients.