Targeting ganglioneuromas with mTOR inhibitors

Mol Cell Oncol. 2021 Jan 10;8(1):1856621. doi: 10.1080/23723556.2020.1856621.

Abstract

We recently identified activated protein kinase B (PKB/AKT) as a tumorigenic driver in childhood ganglioneuroma. Inhibition of the mechanistic target of rapamycin (mTOR), a serine/threonine kinase downstream of AKT, effectively reduced the tumor burden in zebrafish with ganglioneuroma. We propose a clinical trial of mTOR inhibitors as a means to shrink large ganglioneuromas prior to surgical resection.

Keywords: AKT; Ganglioneuroma; mTOR inhibitor; zebrafish model.