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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.<\/jats:p>","DOI":"10.1007\/s11060-024-04899-6","type":"journal-article","created":{"date-parts":[[2025,3,5]],"date-time":"2025-03-05T04:20:15Z","timestamp":1741148415000},"page":"281-288","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Current clinical trials for craniopharyngiomas: what\u2019s on the horizon?"],"prefix":"10.1007","volume":"172","author":[{"given":"Nikhil","family":"Joshi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabine","family":"Mueller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cassie","family":"Kline","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,3,5]]},"reference":[{"issue":"3","key":"4899_CR1","doi-asserted-by":"publisher","first-page":"513","DOI":"10.1210\/er.2013-1115","volume":"35","author":"HL M\u00fcller","year":"2014","unstructured":"M\u00fcller HL, Craniopharyngioma (2014) Endocr Rev 35(3):513\u2013543. https:\/\/doi.org\/10.1210\/er.2013-1115","journal-title":"Endocr Rev"},{"key":"4899_CR2","doi-asserted-by":"publisher","first-page":"235","DOI":"10.1016\/B978-0-444-59602-4.00016-2","volume":"124","author":"HL M\u00fcller","year":"2014","unstructured":"M\u00fcller HL, Craniopharyngioma (2014) Handb Clin Neurol 124:235\u2013253. https:\/\/doi.org\/10.1016\/B978-0-444-59602-4.00016-2","journal-title":"Handb Clin Neurol"},{"issue":"6","key":"4899_CR3","doi-asserted-by":"publisher","first-page":"589","DOI":"10.1007\/s00401-005-1004-x","volume":"109","author":"R Buslei","year":"2005","unstructured":"Buslei R, Nolde M, Hofmann B et al (2005) Common mutations of beta-catenin in adamantinomatous craniopharyngiomas but not in other tumours originating from the sellar region. 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