Journal
NEUROSURGICAL FOCUS
Volume 41, Issue 6, Pages -Publisher
AMER ASSOC NEUROLOGICAL SURGEONS
DOI: 10.3171/2016.8.FOCUS16307
Keywords
adamantinomatous craniopharyngioma; molecular therapeutics; targeted therapies
Categories
Funding
- Cancer Research UK Clinical Research Training Fellowship through the UCL Cancer Institute
- Children with Cancer UK [W1055]
- Great Ormond Street Hospital Children's Charity (GOSHCC)
- Medical Research Council [164126]
- National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust
- University College London
- Children with Cancer UK [15-190] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [W1055] Funding Source: researchfish
- Medical Research Council [MR/M000125/1] Funding Source: researchfish
- MRC [MR/M000125/1] Funding Source: UKRI
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Since the first identification of CTNNB1 mutations in adamantinomatous craniopharyngioma (ACP), much has been learned about the molecular pathways and processes that are disrupted in ACP pathogenesis. To date this understanding has not translated into tangible patient benefit. The recent development of novel techniques and a range of preclinical models now provides an opportunity to begin to support treatment decisions and develop new therapeutics based on molecular pathology. In this review the authors summarize many of the key findings and pathways implicated in ACP pathogenesis and discuss the challenges that need to be tackled to translate these basic science findings for the benefit of patients.
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