Targeting pediatric adrenocortical carcinoma: Molecular insights and emerging therapeutic strategies

Rákkutatás · Adrenocortical carcinoma · KRAS

Targeting metabolic vulnerabilities, such as steroidogenesis and lipid metabolism, offers additional avenues for therapeutic innovation.

Pediatric adrenocortical carcinoma (pACC) is an exceptionally rare and aggressive malignancy with limited data and most protocols adapted from adult treatment. Molecular profiling reveals actionable targets in the Wnt/β‑catenin and MAP/ERK pathways, IGF2 overexpression and epigenetic dysregulation. Emerging strategies including immune‑checkpoint inhibitors, CAR T‑cell therapy, radiopharmaceuticals and metabolic‑targeted approaches show promise but remain largely untested in children. Diagnostic advances such as liquid biopsy and steroid profiling may improve monitoring. Collaborative, pediatric‑specific clinical trials are urgently needed to translate these insights into effective therapy.

pACC carries a high mortality rate due to its aggressive biology and the absence of established pediatric treatment protocols; therefore, identifying actionable molecular targets, novel therapeutics, and improved diagnostic tools is critical to improve outcomes for affected children.

Bizonyítékszint: Sejtvonalas. Laboratóriumi sejtekben vizsgálták.

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