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Targeted Therapy in the Management of Modern Craniopharyngiomas
Maikerly Reyes1, Mohammad Taghvaei1, Siyuan Yu1
1Department of Neurological Surgery, Thomas Jefferson University Hospital, Philadelphia, PA 19107, USA.
Background:
The proximity of craniopharyngiomas (CPs) to critical neurovascular structures can lead to a host of neurologic and endocrine complications that lead to difficulty with surgical management. In this review, we examine the molecular and genetic markers implicated in CP, their involvement in tumorigenic pathways, and their impact on CP prognosis and treatment.
Methods:
We undertook a focused review of relevant articles, clinical trials, and molecular summaries regarding CP.
Results:
Genetic and immunological markers show variable expression in different types of CP. BRAF is implicated in tumorigenesis in papillary CP (pCP), whereas CTNNB1 and EGFR are often overexpressed in adamantinomatous CP (aCP) and VEGF is overexpressed in aCP and recurrent CP. Targeted treatment modalities inhibiting these pathways can shrink or halt progression of CP. In addition, EGFR inhibitors may sensitize tumors to radiation therapy. These drugs show promise in medical management and neoadjuvant therapy for CP. Immunotherapy, including anti-interleukin-6 (IL-6) drugs and interferon treatment, are also effective in managing tumor growth. Ongoing clinical trials in CP are limited but are testing BRAF/MET inhibitors and IL-6 monoclonal antibodies.
Conclusions:
Genetic and immunological markers show variable expression in different subtypes of CP. Several current molecular treatments have shown some success in the management of this disease. Additional clinical trials and targeted therapies will be important to improve CP patient outcomes.
Insights
Molecular and genetic markers are key to understanding craniopharyngioma (CP) subtypes and developing targeted treatments. Investigating these markers improves prognosis and guides novel therapies for better patient outcomes.
Area of Science:
- Neuro-oncology
- Molecular Oncology
- Endocrinology
Background:
- Craniopharyngiomas (CPs) pose surgical challenges due to their location near critical neurovascular structures.
- Surgical management of CPs is complicated by potential neurologic and endocrine deficits.
Purpose of the Study:
- To review molecular and genetic markers implicated in craniopharyngioma (CP).
- To examine the role of these markers in tumorigenic pathways, prognosis, and treatment of CP.
Main Methods:
- Focused review of relevant literature, clinical trials, and molecular summaries.
- Analysis of genetic and immunological markers in different CP subtypes.
Main Results:
- Variable expression of genetic markers (BRAF, CTNNB1, EGFR, VEGF) observed across CP subtypes (pCP, aCP).
- Targeted therapies inhibiting implicated pathways show promise in halting CP progression and sensitizing tumors to radiation.
- Immunotherapies (anti-IL-6, interferon) demonstrate efficacy in managing tumor growth.
Conclusions:
- Genetic and immunological markers are crucial for understanding CP heterogeneity.
- Current molecular treatments offer some success in CP management.
- Further clinical trials and targeted therapies are essential for improving patient outcomes.
