{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,5]],"date-time":"2026-02-05T04:03:41Z","timestamp":1770264221385,"version":"3.49.0"},"reference-count":45,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T00:00:00Z","timestamp":1770163200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"NIH\/NCI","award":["U01 CA217613"],"award-info":[{"award-number":["U01 CA217613"]}]},{"name":"NIH\/NCI","award":["2UO1 CA199288-06"],"award-info":[{"award-number":["2UO1 CA199288-06"]}]},{"name":"NIH\/NCI","award":["U24CA263963"],"award-info":[{"award-number":["U24CA263963"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>The poor clinical outcomes of pediatric high-grade glioma (pHGG) highlight the urgent need for new therapies. Oligodendrocyte lineage transcription factor 2 (OLIG2) is a pro-mitotic transcription factor highly expressed in glioma stem cells and may represent a novel therapeutic target. To evaluate the therapeutic efficacy of an OLIG2 inhibitor CT-179 in pHGG, we determined the OLIG2 mRNA expression in 10 patient-derived orthotopic xenograft (PDOX) models. In vitro activities of CT-179 were analyzed in monolayer and neurosphere cells (0\u201310 \u00b5M) with and without radiation (XRT) (0\u20138 Gy), brain penetration was evaluated in tumor-bearing PDOX mice, and in vivo efficacy was determined at 15\u2013240 mg\/kg (oral) alone or combined with XRT (2 Gy\/day \u00d7 5 days). Changes in animal survival times were analyzed using the Kaplan\u2013Meier method, followed by pair-wise comparisons. Increased OLIG2 mRNA expression was detected in seven out of ten PDOX models. CT-179 inhibited cell viability in a time- and dose-dependent manner in all eight pGBM xenograft tumors (IC50 0.03\u201310 \u00b5M) and was potentiated by XRT (0.03\u20131 \u00b5M). Oral gavage (24 mg\/kg) of CT-179 for 5 days led to effective penetration in mouse cerebrum (3232.7 \u00b1 569.2 ng\/g), cerebellum (1563.3 \u00b1 269.6 ng\/g), brain stem (1685.3 \u00b1 309 ng\/g), and PDOX tumors (1814 \u00b1 110.3 ng\/g) vs. 361.3 \u00b1 1.5 ng\/mL in serum. CT-179 alone was not active at 200 mg\/kg in four models, although it was moderately effective at 240 mg\/kg in one model. When combined with XRT, a significant extension of animal survival times was observed in two out of four models. Doses needed to eliminate OLIG2 expression in vitro varied from 0.3 to &gt;1 \u00b5M in pGBM cells. In summary, our data showed that orally administered CT-179 penetrated the blood\u2013brain barrier (BBB) and exhibited potential for inhibiting pGBM growth when combined with XRT.<\/jats:p>","DOI":"10.3390\/ijms27031543","type":"journal-article","created":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T14:55:11Z","timestamp":1770216911000},"page":"1543","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Targeting Pediatric Glioblastomas by Combining OLIG2 Inhibitor CT-179 with Fractionated Radiation in a Panel of Patient-Derived Orthotopic Xenograft Mouse Models"],"prefix":"10.3390","volume":"27","author":[{"given":"Holly","family":"Lindsay","sequence":"first","affiliation":[{"name":"Texas Children\u2019s Cancer Center, Texas Children\u2019s Hospital, Baylor College of Medicine, Houston, TX 77030, USA"},{"name":"School of Medicine, University of Colorado Anschutz Medical Campus, Children\u2019s Hospital Colorado Center for Cancer and Blood Disorders, Aurora, CO 80045, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuchen","family":"Du","sequence":"additional","affiliation":[{"name":"Texas Children\u2019s Cancer Center, Texas Children\u2019s Hospital, Baylor College of Medicine, Houston, TX 77030, USA"},{"name":"Ann & Robert H. Lurie Children\u2019s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2491-2701","authenticated-orcid":false,"given":"Lin","family":"Qi","sequence":"additional","affiliation":[{"name":"Texas Children\u2019s Cancer Center, Texas Children\u2019s Hospital, Baylor College of Medicine, Houston, TX 77030, USA"},{"name":"Ann & Robert H. Lurie Children\u2019s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA"},{"name":"Guangxi Key Laboratory of Tumor Immunology and Microenvironmental Regulation, Guilin Medical University, Guilin 541004, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Huiyuan","family":"Zhang","sequence":"additional","affiliation":[{"name":"Texas Children\u2019s Cancer Center, Texas Children\u2019s Hospital, Baylor College of Medicine, Houston, TX 77030, USA"},{"name":"Ben Towne Center for Childhood Cancer and Blood Disorders, Seattle Children\u2019s Research Institute, Seattle, WA 98101, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-6401-7186","authenticated-orcid":false,"given":"Sibo","family":"Zhao","sequence":"additional","affiliation":[{"name":"Texas Children\u2019s Cancer Center, Texas Children\u2019s Hospital, Baylor College of Medicine, Houston, TX 77030, 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