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DMGs are high-grade gliomas (HGGs) diagnosed along the brain\u2019s midline. Euchromatin is the hallmark feature of DMG, caused by global hypomethylation of H3K27 either through point mutations in histone H3 genes (H3K27M), or by overexpression of the enhancer of zeste homolog inhibitory protein. In a clinical trial for adults with progressive HGGs, a 22-year-old patient with a thalamic DMG, H3 K27-altered, showed a remarkable clinical and radiological response to dordaviprone (ONC201). This response in an H3 K27-altered HGG patient, coupled with the lack of response of patients harboring wildtype-H3 tumors, has increased the clinical interest in dordaviprone for the treatment of DMG. Additional reports of clinical benefit have emerged, but research defining mechanisms of action (MOA) fall behind dordaviprone\u2019s clinical use, with biomarkers of response unresolved. Here, we summarize dordaviprone\u2019s safety, interrogate its preclinical MOA identifying the mitochondrial protease \u201cClpP\u201d as a biomarker of response, and discuss other ClpP agonists, expanding the arsenal of potential weapons in the fight against DMG. Finally, we discuss combination strategies including ClpP agonists, and their immunomodulatory effects suggestive of a role for the tumor microenvironment in DMG patient response.<\/jats:p>","DOI":"10.1093\/neuonc\/noad144","type":"journal-article","created":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T11:53:28Z","timestamp":1692273208000},"page":"S136-S154","source":"Crossref","is-referenced-by-count":36,"title":["A review of current therapeutics targeting the mitochondrial protease ClpP in diffuse midline glioma, H3 K27-altered"],"prefix":"10.1093","volume":"26","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2245-9213","authenticated-orcid":false,"given":"Evangeline R","family":"Jackson","sequence":"first","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8967-7242","authenticated-orcid":false,"given":"Mika L","family":"Persson","sequence":"additional","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cameron J","family":"Fish","sequence":"additional","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3192-5888","authenticated-orcid":false,"given":"Izac J","family":"Findlay","sequence":"additional","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabine","family":"Mueller","sequence":"additional","affiliation":[{"name":"DIPG\/DMG Center Zurich, University Children\u2019s Hospital Z\u00fcrich , Zurich , Switzerland"},{"name":"Department of Neurology, Neurosurgery and Pediatric, UCSF , San Francisco, California , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1951-9828","authenticated-orcid":false,"given":"Javad","family":"Nazarian","sequence":"additional","affiliation":[{"name":"DIPG\/DMG Center Zurich, University Children\u2019s Hospital Z\u00fcrich , Zurich , Switzerland"},{"name":"Center for Genetic Medicine Research, Children\u2019s National Hospital , Washington, District of Columbia , USA"},{"name":"The George Washington University, School of Medicine and Health Sciences , Washington, District of Columbia , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Esther","family":"Hulleman","sequence":"additional","affiliation":[{"name":"Princess M\u00e1xima Center for Pediatric Oncology , Utrecht, Netherlands, Utrecht , Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jasper","family":"van der Lugt","sequence":"additional","affiliation":[{"name":"Princess M\u00e1xima Center for Pediatric Oncology , Utrecht, Netherlands, Utrecht , Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3651-7672","authenticated-orcid":false,"given":"Ryan J","family":"Duchatel","sequence":"additional","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9063-5370","authenticated-orcid":false,"given":"Matthew D","family":"Dun","sequence":"additional","affiliation":[{"name":"Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"},{"name":"Precision Medicine Research Program, Hunter Medical Research Institute , New Lambton Heights, New South Wales , Australia"},{"name":"Paediatric Program, Mark Hughes Foundation Centre for Brain Cancer Research, College of Health, Medicine and Wellbeing, University of Newcastle , Callaghan, New South Wales , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2023,8,17]]},"reference":[{"issue":"3","key":"2024052010154470800_CIT0001","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1007\/s11060-013-1189-0","article-title":"Clinico-radiologic characteristics of long-term survivors of diffuse intrinsic pontine glioma","volume":"114","author":"Jackson","year":"2013","journal-title":"J Neurooncol."},{"issue":"19","key":"2024052010154470800_CIT0002","doi-asserted-by":"crossref","first-page":"1963","DOI":"10.1200\/JCO.2017.75.9308","article-title":"Clinical, Radiologic, Pathologic, and Molecular 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Oncol"},{"issue":"4","key":"2024052010154470800_CIT0005","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1038\/s41388-021-02102-y","article-title":"Pharmaco-proteogenomic profiling of pediatric diffuse midline glioma to inform future treatment strategies","volume":"41","author":"Findlay","year":"2022","journal-title":"Oncogene."},{"issue":"9","key":"2024052010154470800_CIT0006","doi-asserted-by":"crossref","first-page":"1408","DOI":"10.1093\/neuonc\/noac117","article-title":"The intrinsic and microenvironmental features of diffuse midline glioma: Implications for the development of effective immunotherapeutic treatment strategies","volume":"24","author":"Persson","year":"2022","journal-title":"Neuro Oncol"},{"issue":"6134","key":"2024052010154470800_CIT0007","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1126\/science.1232245","article-title":"Inhibition of PRC2 activity by a gain-of-function H3 mutation found in pediatric 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gliomas","volume":"124","author":"Khuong-Quang","year":"2012","journal-title":"Acta Neuropathol."},{"issue":"5","key":"2024052010154470800_CIT0011","doi-asserted-by":"crossref","first-page":"660","DOI":"10.1016\/j.ccr.2013.10.006","article-title":"Reduced H3K27me3 and DNA hypomethylation are major drivers of gene expression in K27M mutant pediatric high-grade gliomas","volume":"24","author":"Bender","year":"2013","journal-title":"Cancer Cell"},{"issue":"1","key":"2024052010154470800_CIT0012","doi-asserted-by":"crossref","first-page":"3775","DOI":"10.1038\/s41598-023-30395-4","article-title":"Histone H3-wild type diffuse midline gliomas with H3K27me3 loss are a distinct entity with exclusive EGFR or ACVR1 mutation and differential methylation of homeobox genes","volume":"13","author":"Ajuyah","year":"2023","journal-title":"Sci Rep."},{"issue":"1","key":"2024052010154470800_CIT0013","doi-asserted-by":"crossref","first-page":"11316","DOI":"10.1038\/ncomms11316","article-title":"Structural 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glioma","volume":"4","author":"Stafford","year":"2018","journal-title":"Sci Adv."},{"issue":"1","key":"2024052010154470800_CIT0017","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1038\/s41467-019-09140-x","article-title":"H3K27M induces defective chromatin spread of PRC2-mediated repressive H3K27me2\/me3 and is essential for glioma tumorigenesis","volume":"10","author":"Harutyunyan","year":"2019","journal-title":"Nat Commun."},{"issue":"4","key":"2024052010154470800_CIT0018","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1038\/nm.4293","article-title":"EZH2 is a potential therapeutic target for H3K27M-mutant pediatric gliomas","volume":"23","author":"Mohammad","year":"2017","journal-title":"Nat Med."},{"issue":"6216","key":"2024052010154470800_CIT0019","doi-asserted-by":"crossref","first-page":"1529","DOI":"10.1126\/science.1253799","article-title":"Use of human embryonic stem cells to model pediatric gliomas with H3.3K27M histone 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glioma","volume":"3","author":"Duchatel","year":"2021","journal-title":"Neuro Oncol Adv"},{"issue":"3","key":"2024052010154470800_CIT0023","doi-asserted-by":"crossref","first-page":"565","DOI":"10.1007\/s11060-023-04347-x","article-title":"Preliminary findings of German-sourced ONC201 treatment in H3K27 altered pediatric pontine diffuse midline gliomas","volume":"163","author":"Tanrikulu","year":"2023","journal-title":"J Neurooncol."},{"issue":"3","key":"2024052010154470800_CIT0024","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1016\/S1470-2045(23)00070-0","article-title":"Access to new drugs in paediatric oncology: Can we learn from the ongoing ONC201 saga","volume":"24","author":"Andr\u00e9","year":"2023","journal-title":"Lancet Oncol."},{"issue":"45","key":"2024052010154470800_CIT0025","doi-asserted-by":"crossref","first-page":"74380","DOI":"10.18632\/oncotarget.11814","article-title":"Discovery and clinical introduction of first-in-class imipridone 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