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Recent advances in targeted therapies offer new hope, particularly those addressing key molecular characteristics and newly identified cancer dependencies. Among these are histone deacetylase inhibitors (HDACis), receptor tyrosine kinase inhibitors, and novel agents such as ONC201 and unesbulin that target metabolic and epigenetic pathways respectively. In addition, emerging therapies like FACT inhibitors and polyamine pathway inhibitors are showing promise by disrupting critical cancer cell processes. Immunotherapies, including CAR-T cells targeting surface antigens such as GD2 and B7-H3, cancer vaccines, monoclonal antibodies, and oncolytic viruses, are also gaining traction, offering a new approach by harnessing the immune system to attack tumor cells. Despite these advances, challenges such as drug delivery across the blood\u2013brain barrier and therapeutic resistance persist, necessitating the development of combination therapies and innovative delivery methods. Ongoing research is focused on refining these strategies and exploring additional molecular and immunological targets to improve outcomes for children with DMG.<\/jats:p>","DOI":"10.1093\/noajnl\/vdaf008","type":"journal-article","created":{"date-parts":[[2025,1,10]],"date-time":"2025-01-10T23:17:55Z","timestamp":1736551075000},"page":"ii41-ii58","source":"Crossref","is-referenced-by-count":0,"title":["A new hope: Clinical advances in targeted therapies for pediatric diffuse midline glioma"],"prefix":"10.1093","volume":"8","author":[{"given":"Joshua","family":"Zhu","sequence":"first","affiliation":[{"name":"School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]},{"name":"Children\u2019s Cancer Institute, Lowy Cancer Centre, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1155-0510","authenticated-orcid":false,"given":"Maria","family":"Tsoli","sequence":"additional","affiliation":[{"name":"School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]},{"name":"Children\u2019s Cancer Institute, Lowy Cancer Centre, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jean","family":"Bertoldo","sequence":"additional","affiliation":[{"name":"School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]},{"name":"Children\u2019s Cancer Institute, Lowy Cancer Centre, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sabine","family":"Mueller","sequence":"additional","affiliation":[{"name":"Department of Neurology, Neurosurgery and Pediatrics, University of California, San Francisco , San Francisco, California ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7451-7916","authenticated-orcid":false,"given":"David S","family":"Ziegler","sequence":"additional","affiliation":[{"name":"Kids Cancer Centre, Sydney Children\u2019s Hospital , Randwick, New South Wales ,","place":["Australia"]},{"name":"School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney , Kensington, New South Wales ,","place":["Australia"]},{"name":"Children\u2019s Cancer Institute, Lowy Cancer Centre, UNSW Sydney , Kensington, New South Wales 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