[Recent Advances in the Treatment of Craniopharyngioma:A Breakthrough in Molecular Targeted Therapy]

Area of Science:

• Neuro-oncology
• Molecular biology
• Endocrinology

Background:

• Craniopharyngiomas are benign tumors near critical brain structures, posing surgical and treatment challenges.
• Recent discoveries identified specific mutations (CTNNB1, BRAF V600E) driving tumor development.
• Understanding tumor biology is key to improving patient outcomes.

Purpose of the Study:

• To review current understanding of craniopharyngioma biology and treatment.
• To highlight advances in surgical and molecular therapies.
• To discuss future directions in personalized treatment approaches.

Main Methods:

• Review of recent molecular studies on craniopharyngioma driver mutations.
• Analysis of advancements in endoscopic endonasal transsphenoidal surgery.
• Evaluation of radiotherapy's role in managing residual or recurrent disease.
• Assessment of molecular targeted therapies, including BRAF and MEK inhibitors.

Main Results:

• CTNNB1 mutations are found in adamantinomatous craniopharyngiomas; BRAF V600E mutations in papillary craniopharyngiomas.
• Endoscopic surgery offers improved safety and minimally invasive resection.
• Targeted therapy with BRAF/MEK inhibitors shows significant tumor shrinkage in BRAF V600E-mutated papillary craniopharyngiomas.
• These targeted agents are effective for recurrent/refractory disease and show potential for neoadjuvant and function-preserving treatment.

Conclusions:

• Craniopharyngioma treatment is evolving with a deeper understanding of molecular drivers.
• Minimally invasive surgery and targeted therapies are transforming management.
• Future strategies will integrate molecular subtypes, patient factors, and functional outcomes for individualized care.
• Preoperative molecular diagnostics, like liquid biopsy, may enhance targeted therapy integration.