ArticleThe Journal of experimental medicine2020
Ganglioneuromas are driven by activated AKT and can be therapeutically targeted with mTOR inhibitors.
Article in The Journal of experimental medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed, 18 citations in OpenAlex.
- Stereotactic Body Radiation Therapy in the Management of Recurrent Unresectable Retroperitoneal Ganglioneuroma.Cureus · 2026Article
- Article
- Colonic Ganglioneuroma: A Combined Single-Institution Experience and Review of the Literature of Forty-Three Patients.Diseases (Basel, Switzerland) · 2023Article
- Retroperitoneal ganglioneuroma presenting as lower back pain.Journal of surgical case reports · 2022Article
- Adrenal ganglioneuroma: Prognostic factors (Review).Experimental and therapeutic medicine · 2021Review
- Targeting ganglioneuromas with mTOR inhibitors.Molecular & cellular oncology · 2021Article
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Peripheral sympathetic nervous system tumors are the most common extracranial solid tumors of childhood and include neuroblastoma, ganglioneuroblastoma, and ganglioneuroma. Surgery is the only effective therapy for ganglioneuroma, which may be challenging due to the location of the tumor and involvement of surrounding structures. Thus, there is a need for well-tolerated presurgical therapies that could reduce the size and extent of ganglioneuroma and therefore limit surgical morbidity. Here, we found that an AKT-mTOR-S6 pathway was active in human ganglioneuroma but not neuroblastoma samples. Zebrafish transgenic for constitutively activated myr-Akt2 in the sympathetic nervous system were found to develop ganglioneuroma without progression to neuroblastoma. Inhibition of the downstream AKT target, mTOR, in zebrafish with ganglioneuroma effectively reduced the tumor burden. Our results implicate activated AKT as a tumorigenic driver in ganglioneuroma. We propose a clinical trial of mTOR inhibitors as a means to shrink large ganglioneuromas before resection in order to reduce surgical morbidity.
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