ONC206 demonstrates potent antitumorigenic activity and is a potential novel therapeutic strategy for high-risk medulloblastoma.
Overview
- Cancer Genome and Epigenetics Program, NCI-Designated Cancer Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, California, USA
- Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA
- Department of Pediatrics, Emory University,Atlanta, Georgia, USA
- Department of Pathology, Children’s Healthcare of Atlanta, Atlanta, Georgia, USA
- Division of Regenerative Medicine, Department of Medicine, Moores Cancer Center and Sanford Stem Cell Institute, UC San Diego, La Jolla, California, USA
- Chimerix (a Jazz Pharmaceuticals Company), Durham, North Carolina, USA
- Department of Neurology and Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center, New York, New York, USA
- Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Atlanta, Georgia, USA
Abstract
Background: Medulloblastoma (MB) is the most common malignant pediatric brain tumor, and has an urgent need for novel treatment approaches. Dordaviprone (ONC201) and its chemical derivative with nanomolar potency, ONC206, induce apoptosis of cancer cells by activation of the mitochondrial caseinolytic protease P (ClpP). Dordaviprone is currently in phase I clinical trials for pediatric patients with primary brain tumors.
Methods: In this study, we evaluated the preclinical therapeutic effects of ONC206 in MB and investigated its mechanism of action.
Results: We found evidence for high expression of ClpP at both the RNA and protein level in MB tumors, compared to very low expression in normal brain tissue. In addition, we saw a pronounced reduction in cell viability of human group 3 and group 4 and murine Sonic-Hedgehog-activated
Conclusion: Our results highlight ONC206 as a novel therapeutic option for patients with high-risk MB and provide strong rationale for testing the efficacy of ONC206 in the treatment of these patients.
Reproduced under the paper's license (CC BY-NC), from the paper cited above.
Code
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Data
Datasets cited
- geo:GSE85217, at NCBI GEO; found in the text, “Molecular Data Mining”
Data Availability
All data that support the findings of the study are available from the corresponding author upon request.
Reproduced under the paper's license (CC BY-NC), from the paper cited above.
Versions
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Version 1, 27 September 2026: the first record
Recorded: type, language, journal, volume, issue, pages, dates, 15 authors, 5 keywords, 16 MeSH terms, 14 funders, 43 references, 1 integrity notice.
Cite
This paper
Tzaridis, T., Liu, J., Chien, F. L., Malhotra, A., Zhu, D., Gershon, I., Zhang, H., Velazquez Vega, J. E., Schniederjan, M., Sposito, T., Adams, P. D., Allen, J. E., Prabhu, V. V., Wechsler-Reya, R. J., & MacDonald, T. J. (2026). ONC206 demonstrates potent antitumorigenic activity and is a potential novel therapeutic strategy for high-risk medulloblastoma. Neuro-oncology, 28(7), 1753-1768. https://
BibTeX
@article{tzaridis2026onc
author = {Tzaridis, Theophilos and Liu, Jingbo and Chien, Franklin Lee and Malhotra, Anshu and Zhu, Dan and Gershon, Isabella and Zhang, Hongying and Velazquez Vega, Jose E and Schniederjan, Matthew and Sposito, Teresa and Adams, Peter D and Allen, Joshua E and Prabhu, Varun V and Wechsler-Reya, Robert J and MacDonald, Tobey J},
title = {{ONC206 demonstrates potent antitumorigenic activity and is a potential novel therapeutic strategy for high-risk medulloblastoma}},
journal = {Neuro-oncology},
year = {2026},
month = jul,
volume = {28},
number = {7},
pages = {1753--1768},
publisher = {Oxford University Press},
issn = {1522-8517},
doi = {10.1093/
url = {https://
pmid = {41818696},
pmcid = {PMC13338324}
}
RIS
TY - JOUR
AU - Tzaridis, Theophilos
AU - Liu, Jingbo
AU - Chien, Franklin Lee
AU - Malhotra, Anshu
AU - Zhu, Dan
AU - Gershon, Isabella
AU - Zhang, Hongying
AU - Velazquez Vega, Jose E
AU - Schniederjan, Matthew
AU - Sposito, Teresa
AU - Adams, Peter D
AU - Allen, Joshua E
AU - Prabhu, Varun V
AU - Wechsler-Reya, Robert J
AU - MacDonald, Tobey J
TI - ONC206 demonstrates potent antitumorigenic activity and is a potential novel therapeutic strategy for high-risk medulloblastoma
T2 - Neuro-oncology
J2 - Neuro Oncol
PY - 2026
DA - 2026/
VL - 28
IS - 7
SP - 1753
EP - 1768
SN - 1522-8517
PB - Oxford University Press
DO - 10.1093/
UR - https://
LA - en
ER -
CSL-JSON
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