Evidence map›Paper›PMID 40210723›Full record

ArticleJournal of molecular medicine (Berlin, Germany)2025

ONC201 exerts oncogenic effects beyond its mitochondria-disturbing role in neuroblastoma subsets.

Jyun-Hong Jiang, Yu-Han Lin, Pei-Lin Liao, Ting-Ya Chen, Hui-Ching Chuang, Chao-Cheng Huang, Wen-Ming Hsu, Jiin-Haur Chuang, Wei-Shiung Lian

Abstract read
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In one paragraph

Article in Journal of molecular medicine (Berlin, Germany), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
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0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Jyun-Hong Jiang *Department of Pediatric Surgery, Kaohsiung Chang Gung Memorial Hospital, Graduate Institute of Clinical Medical Sciences, College of Medicine, Chang Gung University, Kaohsiung, Taiwan.
Yu-Han Lin *Center for Mitochondrial Research and Medicine, College of Medicine Chang Gung University, Kaohsiung Chang Gung Memorial Hospital, Kaohsiung, Taiwan.
Pei-Lin LiaoDepartment of Pediatric Surgery, Kaohsiung Chang Gung Memorial Hospital, Graduate Institute of Clinical Medical Sciences, College of Medicine, Chang Gung University, Kaohsiung, Taiwan.
Ting-Ya ChenCenter for Mitochondrial Research and Medicine, College of Medicine Chang Gung University, Kaohsiung Chang Gung Memorial Hospital, Kaohsiung, Taiwan.
Hui-Ching ChuangCenter for Mitochondrial Research and Medicine, College of Medicine Chang Gung University, Kaohsiung Chang Gung Memorial Hospital, Kaohsiung, Taiwan.
Chao-Cheng HuangDepartment of Pathology, Kaohsiung Chang Gung Memorial Hospital, College of Medicine, Chang Gung University, Kaohsiung, Taiwan.
Wen-Ming HsuDepartment of Surgery, National Taiwan University Hospital, National Taiwan University College of Medicine, Taipei, Taiwan.
Jiin-Haur ChuangDepartment of Pediatric Surgery, Kaohsiung Chang Gung Memorial Hospital, Graduate Institute of Clinical Medical Sciences, College of Medicine, Chang Gung University, Kaohsiung, Taiwan. jhchuang@cloud.cgmh.org.tw.
Wei-Shiung LianCenter for Mitochondrial Research and Medicine, College of Medicine Chang Gung University, Kaohsiung Chang Gung Memorial Hospital, Kaohsiung, Taiwan. lianws@gmail.com.

Funding

Chang Gung Memorial Hospital CMRPG8H0181-3Chang Gung Memorial Hospital CRRPG8K0071-3Ministry of Health and Welfare NHRL-EX114-11437SNational Science and Technology Council NSTC 112-2314-B-182A-105-MY3
6 · The paper itself

Abstract

Neuroblastoma (NB) is a formidable challenge in pediatric oncology due to its intricate molecular landscape, necessitating multifaceted therapeutic approaches. ONC201 is an imipridone antibiotic compound with a promising drug candidate leveraging its potent anticancer properties against the mitochondrial proteases ClpP and ClpX. Despite demonstrating early clinical promise, particularly in MYCN-amplified NB, its efficacy in non-MYCN-amplified NB remains a subject worthy of investigation. In this study, we extended the coverage of ONC201 to treat non-MYCN-amplified NB, and our data implicated ONC201's inability to reduce tumor growth in animal models harboring SK-N-AS or SK-N-FI cell lines. Interestingly, ONC201 induced the expression of oncogenic markers c-Myc and LGR5 while downregulating the tumor suppressor ATRX. While it fails to attenuate tumor neovascularization in non-MYCN-amplified NB xenografts, its effectiveness differs from that of its MYCN-amplified counterpart. Rho zero (ρ0)-SK-N-AS cells treated with ONC201 showed comparable observed trends in parental SK-N-AS cells, including LGR5 upregulation and ATRX downregulation, suggesting that ONC201's multifaceted actions extend beyond mitochondrial targets. Our elucidation highlights the need to discern molecular signatures when deploying ONC201 monotherapy against NB, which lacks MYCN-amplification.

Indexed as

Antineoplastic AgentsMitochondriaNeuroblastomaAnimalsCell Line, TumorCell ProliferationGene Expression Regulation, NeoplasticHumansMiceN-Myc Proto-Oncogene ProteinReceptors, G-Protein-CoupledXenograft Model Antitumor AssaysX-linked Nuclear ProteinAntineoplastic AgentsATRX protein, humanN-Myc Proto-Oncogene ProteinReceptors, G-Protein-CoupledX-linked Nuclear ProteinATRXLGR5NeuroblastomaNon-MYCN-amplified cell lineONC201

Identifiers

PMID40210723
PMCPMC12078449

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.