Recent progress and novel approaches to treating atypical teratoid rhabdoid tumor

Elizabeth Alva; Jeffrey Rubens; Susan Chi; Tom Rosenberg; Alyssa Reddy; Eric H Raabe; Ashley Margol

doi:10.1016/j.neo.2023.100880

Recent progress and novel approaches to treating atypical teratoid rhabdoid tumor

Neoplasia. 2023 Mar:37:100880. doi: 10.1016/j.neo.2023.100880. Epub 2023 Feb 9.

Authors

Elizabeth Alva¹, Jeffrey Rubens², Susan Chi³, Tom Rosenberg³, Alyssa Reddy⁴, Eric H Raabe⁵, Ashley Margol⁶

Affiliations

¹ Division of Pediatric Hematology-Oncology, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama, USA.
² Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
³ Dana-Farber Cancer Institute, Children's Hospital Boston, Boston, MA, USA.
⁴ Departments of Neurology and Pediatrics, University of California San Francisco, San Francisco, CA, USA.
⁵ Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA. Electronic address: eraabe2@jhmi.edu.
⁶ Children's Hospital Los Angeles, Los Angeles, CA, USA; Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. Electronic address: amargol@chla.usc.edu.

Abstract

Atypical teratoid rhabdoid tumors (AT/RT) are malignant central nervous system (CNS) tumors that occur mostly in young children and have historically carried a very poor prognosis. While recent clinical trial results show that this tumor is curable, outcomes are still poor compared to other central nervous system embryonal tumors. We here review prior AT/RT clinical trials and highlight promising pre-clinical results that may inform novel clinical approaches to this aggressive cancer.

Keywords: INI1; MEK; MYC; adaptive; clinical trial; epigenetics; mTOR.

Publication types

Clinical Trial
Research Support, N.I.H., Extramural

MeSH terms

Central Nervous System Neoplasms*
Child
Child, Preschool
Humans
Infant
Rhabdoid Tumor* / pathology
Rhabdoid Tumor* / therapy
SMARCB1 Protein
Teratoma* / pathology
Teratoma* / therapy

Substances

SMARCB1 Protein

Supplementary concepts

Typical Teratoid Rhabdoid Tumor

Grants and funding

P30 CA006973/CA/NCI NIH HHS/United States