biostudies-literatureUnknown28(18)Kline C<h4>Purpose</h4>PNOC003 is a multicenter precision medicine trial for children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG).<h4>Patients and methods</h4>Patients (3-25 years) were enrolled on the basis of imaging consistent with DIPG. Biopsy tissue was collected for whole-exome and mRNA sequencing. After radiotherapy (RT), patients were assigned up to four FDA-approved drugs based on molecular tumor board recommendations. H3K27M-mutant circulating tumor DNA (ctDNA) was longitudinally measured. Tumor tissue and matched primary cell lines were characterized using whole-genome sequencing and DNA methylation profiling. When applicable, results were verified in an independent cohort from the Children's Brain Tumor Network (CBTN).<h4>Results</h4>Of 38 patients enClinical cancer research : an official journal of the American Association for Cancer Research3965-3978https://www.ebi.ac.uk/biostudies/studies/S-EPMC9475246biostudies-literatureUpfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003.PMC9475246Kilburn LLiang WZhang JPrados MBanerjee AYadavilli SNazarian JLuks TKline CGupta NZhang BBonner ERVillanueva-Meyer JResnick AMolinaro AJain PPacker RJBerens MKuhn JKraya AWaszak SMCrawford JRZhang YGaonkar KSMueller SByron SKambhampati MRokita JLfalseUpfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003.<h4>Purpose</h4>PNOC003 is a multicenter precision medicine trial for children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG).<h4>Patients and methods</h4>Patients (3-25 years) were enrolled on the basis of imaging consistent with DIPG. Biopsy tissue was collected for whole-exome and mRNA sequencing. After radiotherapy (RT), patients were assigned up to four FDA-approved drugs based on molecular tumor board recommendations. H3K27M-mutant circulating tumor DNA (ctDNA) was longitudinally measured. Tumor tissue and matched primary cell lines were characterized using whole-genome sequencing and DNA methylation profiling. When applicable, results were verified in an independent cohort from the Children's Brain Tumor Network (CBTN).<h4>Results</h4>Of 38 patients en2022-01-01T00:00:00Z2022 Sep2026-05-27T22:31:16.392Z2025-02-19T02:38:02.452ZS-EPMC94752463585279510.1158/1078-0432.CCR-22-0803