RARE DISEASE
Diffuse intrinsic pontine glioma
Diffuse intrinsic pontine glioma
Diffuse intrinsic pontine glioma
Synonyms: DIPG
Synonyms: DIPG
Synonyms: DIPG
Drug discovery
5
drugs
With orphan designations
Overview
Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive pediatric brainstem tumor arising in the pons, characterized by diffuse infiltration of glial cells and molecular drivers like H3K27M histone mutations. Diagnosis relies on clinical presentation and MRI findings, with median survival of 8–12 months despite radiotherapy providing temporary symptom relief. No curative therapies exist due to its critical location and resistance to conventional treatments [1][4][11].
Therapies
Radiotherapy: Standard 54–60 Gy fractionated radiation improves symptoms transiently [3][5][16].
Clinical trials: Epigenetic modifiers (e.g., histone deacetylase inhibitors), immunotherapy, and targeted agents (e.g., nimotuzumab) under investigation [7][8][18].
Palliative care: Steroids for edema management and supportive therapies dominate post-progression care [1][12].
Categories: rare neoplastic diseases, rare neurological diseases
Research Papers
1,877 drug discovery papers about Diffuse intrinsic pontine glioma, with 4 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,877 drug discovery papers about Diffuse intrinsic pontine glioma, with 4 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-13 | FECH, a novel metabolic target influencing CAR T-cell phenotype and function.
Recent phase I/II clinical trials have demonstrated that chimeric antigen receptor (CAR) T cells targeting the disialogangliosade GD2 represent a promising therapeutic option for pediatric patients with relapsed or refractory high-risk neuroblastoma (NB). However, incomplete and heterogeneous clinical responses highlight the need to improve CAR T-cell efficacy and persistence. We previously demonstrated the therapeutic benefit of combining the dual insulin-like growth factor 1 receptor/insulin receptor (IGF1R/IR) inhibitor linsitinib (LIN) with third-generation GD2.CAR T cells in diffuse intrinsic pontine glioma, where LIN induced tumor cell death and modulated the CAR T-cell phenotype. Here, we extended these findings to NB and explored the mechanisms of LIN-mediated CAR T-cell modulation. LIN, in combination with CAR T cells, significantly enhanced antitumor activity in LIN-sensitive NB cell lines. Mechanistically, we investigated ferrochelatase (FECH), a mitochondrial enzyme involved in heme biosynthesis, and a known off-target of LIN. LIN treatment or selective FECH inhibition with N-methyl protoporphyrin IX reduced intracellular heme, attenuated activation and exhaustion marker expression and promoted central memory characteristics associated with improved in vivo CAR T-cell persistence and functionality. Both treatments similarly decreased ATP production by reducing glycolysis and mitochondrial respiration in chronical antigen-activated CAR T cells. Collectively, these data reveal a dual mechanism of action for LIN, combining direct tumor cell cytotoxicity with metabolic reprogramming of CAR T cells linked to heme biosynthesis. These findings identify heme metabolism as regulator of CAR T-cell phenotype and function and support further investigation of FECH to enhance therapeutic efficacy in NB and beyond.
2026-08-08 | CAR T-cell therapy in pediatric brain tumors: a narrative review with comparative analysis of clinical trial eligibility criteria.
Pediatric brain tumors are the leading cause of cancer-related mortality in children, and current standard therapies like surgery, radiotherapy, and chemotherapy offer limited survival benefits and significant long-term morbidity. Chimeric antigen receptor (CAR) T-cell therapy is a transformative treatment for hematologic malignancies and is now being explored for pediatric brain tumors. This review summarizes the latest advances, preclinical and clinical findings, challenges of CAR T-cell therapy, and future directions in pediatric neuro-oncology. 18 studies that met the eligibility criteria were selected, consisting of preclinical models, early-phase clinical trials, and translational studies. A registry search of central nervous system (CNS) tumor trials from Clinicaltrials.gov, ISRCTN, and ANZCTR identified 12 active or completed interventional trials of CAR T-cell therapy in patients with CNS tumors, their eligibility criteria and parameters were compared. Preclinical studies consistently demonstrate that CAR T-cells targeting antigens such as B7-H3, GD2, HER2, IL13Rα2, and EphA2 can induce robust and specific tumor regression in models of medulloblastoma, diffuse intrinsic pontine glioma (DIPG), ependymoma, and high-grade gliomas. On the other hand, B7-H3 is a pan-pediatric target due to its high expression in multiple CNS tumors, including medulloblastoma, ependymoma, and glioma, whereas GD2 is highly relevant for H3K27M-mutant diffuse midline gliomas. Early-phase clinical trials confirm that CAR T-cells can traffic to CNS tumors, infiltrate tumor tissue, and mediate tumor regression. The ICV B7-H3 phase 1 trial in DIPG achieved noteworthy results, with a median survival of 19.8 months across 21 patients and 3 patients surviving more than 40 months. GD2-CAR T-cell therapy in H3K27 M-mutant gliomas showed partial clinical responses, with neurotoxicity and encephalopathy observed, whereas the HER2-targeted locoregional therapy showed no dose-limiting toxicities. Future interventions such as multi-antigen targeting, combinatorial CAR designs, and enhanced cytokine signaling are being developed to improve efficacy and safety. A comparison of 12 registered pediatric CAR T-cell trials showed heterogeneity in eligibility criteria, including age ranges, performance status thresholds, H3K27M mutation requirements, and geographic concentration bias. CAR T-cell therapy holds significant promise for improving outcomes in pediatric brain tumors, but its clinical translation is challenged by tumor heterogeneity, antigen escape, neurotoxicity, and the immunosuppressive tumor microenvironment.
2026-08-07 | Locoregional and systemic adoptive cellular therapies for pediatric brain tumors: a systematic review of CAR‑T, TCR‑engineered T cells, and NK cell strategies.
Adoptive cellular therapies may expand treatment options for pediatric brain tumors by focusing activity on tumor antigens and limiting off-tumor effects. We systematically reviewed preclinical and clinical evidence for CAR T cells, TCR-engineered T cells, and NK or γδ T-cell platforms directed against HER2, B7-H3 (CD276), EGFR806-reactive EGFR, GD2, IL13Rα2, and EphA2 or EphA3, with attention to delivery route, safety, persistence, and combination strategies. Following PRISMA, we searched PubMed, Embase, and Scopus from inception through September 17, 2025, restricted to English. The search yielded 324 records; 103 duplicates were removed; 221 titles and abstracts were screened; 180 full texts were reviewed; and 34 studies were extracted by two independent reviewers. We captured design, tumor and molecular features, product engineering, route and schedule, lymphodepletion, toxicities including cytokine release syndrome, immune effector cell associated neurotoxicity, and tumor inflammation associated neurotoxicity, radiographic or clinical response, survival, and correlatives such as persistence or trafficking in blood, cerebrospinal fluid, or tumor tissue, cytokines, and antigen dynamics. In vivo studies showed reproducible antitumor activity for HER2 in medulloblastoma, GD2 in diffuse midline glioma, and multi-antigen constructs incorporating IL13Rα2 and EphA2 in medulloblastoma and ependymoma, with significant survival advantages compared with controls. γδ T cells targeting the EphA axis selectively killed medulloblastoma with neural sparing; GD2 CAR NK-92 inhibited diffuse intrinsic pontine glioma growth. In early clinical programs, route shaped safety and pharmacodynamics. For GD2, low-dose intravenous induction followed by repeated intraventricular dosing produced objective radiographic regressions and manageable tumor inflammation associated neurotoxicity, while dose-limiting cytokine release syndrome was confined to higher intravenous doses. Intraventricular B7-H3 CAR T cells, given without lymphodepletion, enabled multi-cycle dosing with mainly grade 1 to 2 events and cerebrospinal fluid localized persistence. Weekly intracranial EGFR806 CAR T cells were feasible and well tolerated, with stable disease as the best response in a small cohort. Across trials, persistence and immune activation were most evident in cerebrospinal fluid, supporting cerebrospinal fluid centered pharmacodynamic monitoring. Mechanism-based combinations, including IGF-axis inhibition in diffuse midline glioma and epigenetic priming of GD2 with an integrated safety switch in medulloblastoma, enhanced activity. The evidence supports pediatric-centric antigen selection and a CNS-first, locoregional dosing approach to increase on-tumor exposure and reduce systemic toxicity. Priorities include multi-antigen strategies to prevent escape, incorporation of safety switches, earlier deployment when tumor burden is low, and prospective cerebrospinal fluid pharmacodynamics in multisite phase II studies.
2026-07-31 | Therapy-associated lineage plasticity in DIPG following combined CDK4/6 inhibitor, temozolomide, and radiation.
Diffuse intrinsic pontine glioma (DIPG) is a highly lethal and therapeutically refractory pediatric brain tumor, and the effects of distinct disease backgrounds on treatment response and residual cell-state plasticity remain poorly understood. We evaluated abemaciclib, an FDA-approved CDK4/6 inhibitor, combined with temozolomide (TMZ) and radiation (XRT) in two patient-derived orthotopic xenograft (PDOX) models established from a treatment-naïve biopsy (IBs-9119DIPG) and a previously treated autopsy tumor (IBs-A0317DIPG). Treatment activity was assessed in PDOX-derived 3D tumor organoids and in randomized DIPG PDOX studies, followed by survival analysis, immunohistochemistry, and endpoint single-cell RNA sequencing (scRNA-seq). The triple therapy generated synergistic antitumor effects in PDOX-derived organoids and significantly prolonged survival in both PDOX models (P < 0.05) despite their distinct baseline molecular and cell-state differences. Endpoint scRNA-seq revealed reduced oligodendrocyte-progenitor-like (OPC-like) cells in both models and decreased astrocyte-like cells in the IBs-A0317DIPG model as cell-state changes associated with treatment response. In contrast, neural progenitor-like (NPC-like) cells expanded in IBs-A0317DIPG, whereas mesenchymal-like and Mitotic populations persisted in IBs-9119DIPG as candidate therapy-tolerant states. Pseudotime trajectory analysis uncovered a resistance-associated trajectory characterized by an exit from stemness toward differentiation in OPC-like cells in treatment-naïve IBs-9119DIPG, in contrast to the enrichment of stem-like OPC-like and NPC-like cells in therapy-resistant IBs-A0317DIPG. A transcriptionally defined radiation-resistance-associated subpopulation with candidate radiosensitization target genes (NPAS3, TBC1D15, and INPP4B) was also identified. Overall, the triple therapy improved survival in clinically distinct DIPG PDOX models and revealed therapy-associated residual cell-state changes that may inform future strategies to improve durable DIPG tumor control.
2026-07-27 | Intracerebroventricular B7-H3-targeting CAR T cells for non-pontine DMG and recurrent/refractory pediatric CNS tumors: a phase 1 trial.
High-grade central nervous system (CNS) tumors carry a poor prognosis with limited curative options if first-line therapy fails. B7-H3 is expressed in many of these tumors, and chimeric antigen receptor (CAR) T cell therapy is an emerging immunotherapeutic strategy. BrainChild-03 (NCT04185038) is a single-center, dose-escalation phase 1 study of repeated intracerebroventricular (ICV) B7-H3 CAR T cells in children and young adults with recurrent/refractory CNS tumors (Arms A, B) and diffuse intrinsic pontine glioma (DIPG, Arm C). Here, we report results from Arm B, in which patients with refractory/relapsed CNS tumors or pre- or post-progression non-pontine diffuse midline glioma (DMG) received repeated ICV infusions. Primary objectives were feasibility and safety/tolerability; secondary objectives included CAR T cell detection, disease response, and survival. Of 36 enrolled patients (atypical teratoid rhabdoid tumor n = 5, DMG n = 8, embryonal tumor with multilayer rosettes n = 2, ependymoma n = 4, high-grade glioma n = 6, medulloblastoma n = 8, pineoblastoma n = 3), manufacturing was successful for 35 patients, 26 of whom received therapy. Median age was 10 years (range 1-26). Dose escalation from 1 × 107 to 10 × 107 CAR T cells/dose identified this dose as the maximally tolerated dose regimen, with no dose-limiting toxicities observed. Across 181 total doses (median 7/patient), common adverse events included headache (n = 26), fever (n = 15), and nausea (n = 14). Median survival from first infusion was 11.5 months, ranging from 3.2 months (pineoblastoma, HGG) to 21.4 months (ependymoma); two patients achieved a partial response. Repeated ICV B7-H3 CAR T cell dosing is feasible and tolerable across a spectrum of pediatric CNS tumors, supporting continued investigation in future trials.
2026-08-13 | FECH, a novel metabolic target influencing CAR T-cell phenotype and function.
Recent phase I/II clinical trials have demonstrated that chimeric antigen receptor (CAR) T cells targeting the disialogangliosade GD2 represent a promising therapeutic option for pediatric patients with relapsed or refractory high-risk neuroblastoma (NB). However, incomplete and heterogeneous clinical responses highlight the need to improve CAR T-cell efficacy and persistence. We previously demonstrated the therapeutic benefit of combining the dual insulin-like growth factor 1 receptor/insulin receptor (IGF1R/IR) inhibitor linsitinib (LIN) with third-generation GD2.CAR T cells in diffuse intrinsic pontine glioma, where LIN induced tumor cell death and modulated the CAR T-cell phenotype. Here, we extended these findings to NB and explored the mechanisms of LIN-mediated CAR T-cell modulation. LIN, in combination with CAR T cells, significantly enhanced antitumor activity in LIN-sensitive NB cell lines. Mechanistically, we investigated ferrochelatase (FECH), a mitochondrial enzyme involved in heme biosynthesis, and a known off-target of LIN. LIN treatment or selective FECH inhibition with N-methyl protoporphyrin IX reduced intracellular heme, attenuated activation and exhaustion marker expression and promoted central memory characteristics associated with improved in vivo CAR T-cell persistence and functionality. Both treatments similarly decreased ATP production by reducing glycolysis and mitochondrial respiration in chronical antigen-activated CAR T cells. Collectively, these data reveal a dual mechanism of action for LIN, combining direct tumor cell cytotoxicity with metabolic reprogramming of CAR T cells linked to heme biosynthesis. These findings identify heme metabolism as regulator of CAR T-cell phenotype and function and support further investigation of FECH to enhance therapeutic efficacy in NB and beyond.
2026-08-08 | CAR T-cell therapy in pediatric brain tumors: a narrative review with comparative analysis of clinical trial eligibility criteria.
Pediatric brain tumors are the leading cause of cancer-related mortality in children, and current standard therapies like surgery, radiotherapy, and chemotherapy offer limited survival benefits and significant long-term morbidity. Chimeric antigen receptor (CAR) T-cell therapy is a transformative treatment for hematologic malignancies and is now being explored for pediatric brain tumors. This review summarizes the latest advances, preclinical and clinical findings, challenges of CAR T-cell therapy, and future directions in pediatric neuro-oncology. 18 studies that met the eligibility criteria were selected, consisting of preclinical models, early-phase clinical trials, and translational studies. A registry search of central nervous system (CNS) tumor trials from Clinicaltrials.gov, ISRCTN, and ANZCTR identified 12 active or completed interventional trials of CAR T-cell therapy in patients with CNS tumors, their eligibility criteria and parameters were compared. Preclinical studies consistently demonstrate that CAR T-cells targeting antigens such as B7-H3, GD2, HER2, IL13Rα2, and EphA2 can induce robust and specific tumor regression in models of medulloblastoma, diffuse intrinsic pontine glioma (DIPG), ependymoma, and high-grade gliomas. On the other hand, B7-H3 is a pan-pediatric target due to its high expression in multiple CNS tumors, including medulloblastoma, ependymoma, and glioma, whereas GD2 is highly relevant for H3K27M-mutant diffuse midline gliomas. Early-phase clinical trials confirm that CAR T-cells can traffic to CNS tumors, infiltrate tumor tissue, and mediate tumor regression. The ICV B7-H3 phase 1 trial in DIPG achieved noteworthy results, with a median survival of 19.8 months across 21 patients and 3 patients surviving more than 40 months. GD2-CAR T-cell therapy in H3K27 M-mutant gliomas showed partial clinical responses, with neurotoxicity and encephalopathy observed, whereas the HER2-targeted locoregional therapy showed no dose-limiting toxicities. Future interventions such as multi-antigen targeting, combinatorial CAR designs, and enhanced cytokine signaling are being developed to improve efficacy and safety. A comparison of 12 registered pediatric CAR T-cell trials showed heterogeneity in eligibility criteria, including age ranges, performance status thresholds, H3K27M mutation requirements, and geographic concentration bias. CAR T-cell therapy holds significant promise for improving outcomes in pediatric brain tumors, but its clinical translation is challenged by tumor heterogeneity, antigen escape, neurotoxicity, and the immunosuppressive tumor microenvironment.
2026-08-07 | Locoregional and systemic adoptive cellular therapies for pediatric brain tumors: a systematic review of CAR‑T, TCR‑engineered T cells, and NK cell strategies.
Adoptive cellular therapies may expand treatment options for pediatric brain tumors by focusing activity on tumor antigens and limiting off-tumor effects. We systematically reviewed preclinical and clinical evidence for CAR T cells, TCR-engineered T cells, and NK or γδ T-cell platforms directed against HER2, B7-H3 (CD276), EGFR806-reactive EGFR, GD2, IL13Rα2, and EphA2 or EphA3, with attention to delivery route, safety, persistence, and combination strategies. Following PRISMA, we searched PubMed, Embase, and Scopus from inception through September 17, 2025, restricted to English. The search yielded 324 records; 103 duplicates were removed; 221 titles and abstracts were screened; 180 full texts were reviewed; and 34 studies were extracted by two independent reviewers. We captured design, tumor and molecular features, product engineering, route and schedule, lymphodepletion, toxicities including cytokine release syndrome, immune effector cell associated neurotoxicity, and tumor inflammation associated neurotoxicity, radiographic or clinical response, survival, and correlatives such as persistence or trafficking in blood, cerebrospinal fluid, or tumor tissue, cytokines, and antigen dynamics. In vivo studies showed reproducible antitumor activity for HER2 in medulloblastoma, GD2 in diffuse midline glioma, and multi-antigen constructs incorporating IL13Rα2 and EphA2 in medulloblastoma and ependymoma, with significant survival advantages compared with controls. γδ T cells targeting the EphA axis selectively killed medulloblastoma with neural sparing; GD2 CAR NK-92 inhibited diffuse intrinsic pontine glioma growth. In early clinical programs, route shaped safety and pharmacodynamics. For GD2, low-dose intravenous induction followed by repeated intraventricular dosing produced objective radiographic regressions and manageable tumor inflammation associated neurotoxicity, while dose-limiting cytokine release syndrome was confined to higher intravenous doses. Intraventricular B7-H3 CAR T cells, given without lymphodepletion, enabled multi-cycle dosing with mainly grade 1 to 2 events and cerebrospinal fluid localized persistence. Weekly intracranial EGFR806 CAR T cells were feasible and well tolerated, with stable disease as the best response in a small cohort. Across trials, persistence and immune activation were most evident in cerebrospinal fluid, supporting cerebrospinal fluid centered pharmacodynamic monitoring. Mechanism-based combinations, including IGF-axis inhibition in diffuse midline glioma and epigenetic priming of GD2 with an integrated safety switch in medulloblastoma, enhanced activity. The evidence supports pediatric-centric antigen selection and a CNS-first, locoregional dosing approach to increase on-tumor exposure and reduce systemic toxicity. Priorities include multi-antigen strategies to prevent escape, incorporation of safety switches, earlier deployment when tumor burden is low, and prospective cerebrospinal fluid pharmacodynamics in multisite phase II studies.
2026-07-31 | Therapy-associated lineage plasticity in DIPG following combined CDK4/6 inhibitor, temozolomide, and radiation.
Diffuse intrinsic pontine glioma (DIPG) is a highly lethal and therapeutically refractory pediatric brain tumor, and the effects of distinct disease backgrounds on treatment response and residual cell-state plasticity remain poorly understood. We evaluated abemaciclib, an FDA-approved CDK4/6 inhibitor, combined with temozolomide (TMZ) and radiation (XRT) in two patient-derived orthotopic xenograft (PDOX) models established from a treatment-naïve biopsy (IBs-9119DIPG) and a previously treated autopsy tumor (IBs-A0317DIPG). Treatment activity was assessed in PDOX-derived 3D tumor organoids and in randomized DIPG PDOX studies, followed by survival analysis, immunohistochemistry, and endpoint single-cell RNA sequencing (scRNA-seq). The triple therapy generated synergistic antitumor effects in PDOX-derived organoids and significantly prolonged survival in both PDOX models (P < 0.05) despite their distinct baseline molecular and cell-state differences. Endpoint scRNA-seq revealed reduced oligodendrocyte-progenitor-like (OPC-like) cells in both models and decreased astrocyte-like cells in the IBs-A0317DIPG model as cell-state changes associated with treatment response. In contrast, neural progenitor-like (NPC-like) cells expanded in IBs-A0317DIPG, whereas mesenchymal-like and Mitotic populations persisted in IBs-9119DIPG as candidate therapy-tolerant states. Pseudotime trajectory analysis uncovered a resistance-associated trajectory characterized by an exit from stemness toward differentiation in OPC-like cells in treatment-naïve IBs-9119DIPG, in contrast to the enrichment of stem-like OPC-like and NPC-like cells in therapy-resistant IBs-A0317DIPG. A transcriptionally defined radiation-resistance-associated subpopulation with candidate radiosensitization target genes (NPAS3, TBC1D15, and INPP4B) was also identified. Overall, the triple therapy improved survival in clinically distinct DIPG PDOX models and revealed therapy-associated residual cell-state changes that may inform future strategies to improve durable DIPG tumor control.
2026-07-27 | Intracerebroventricular B7-H3-targeting CAR T cells for non-pontine DMG and recurrent/refractory pediatric CNS tumors: a phase 1 trial.
High-grade central nervous system (CNS) tumors carry a poor prognosis with limited curative options if first-line therapy fails. B7-H3 is expressed in many of these tumors, and chimeric antigen receptor (CAR) T cell therapy is an emerging immunotherapeutic strategy. BrainChild-03 (NCT04185038) is a single-center, dose-escalation phase 1 study of repeated intracerebroventricular (ICV) B7-H3 CAR T cells in children and young adults with recurrent/refractory CNS tumors (Arms A, B) and diffuse intrinsic pontine glioma (DIPG, Arm C). Here, we report results from Arm B, in which patients with refractory/relapsed CNS tumors or pre- or post-progression non-pontine diffuse midline glioma (DMG) received repeated ICV infusions. Primary objectives were feasibility and safety/tolerability; secondary objectives included CAR T cell detection, disease response, and survival. Of 36 enrolled patients (atypical teratoid rhabdoid tumor n = 5, DMG n = 8, embryonal tumor with multilayer rosettes n = 2, ependymoma n = 4, high-grade glioma n = 6, medulloblastoma n = 8, pineoblastoma n = 3), manufacturing was successful for 35 patients, 26 of whom received therapy. Median age was 10 years (range 1-26). Dose escalation from 1 × 107 to 10 × 107 CAR T cells/dose identified this dose as the maximally tolerated dose regimen, with no dose-limiting toxicities observed. Across 181 total doses (median 7/patient), common adverse events included headache (n = 26), fever (n = 15), and nausea (n = 14). Median survival from first infusion was 11.5 months, ranging from 3.2 months (pineoblastoma, HGG) to 21.4 months (ependymoma); two patients achieved a partial response. Repeated ICV B7-H3 CAR T cell dosing is feasible and tolerable across a spectrum of pediatric CNS tumors, supporting continued investigation in future trials.
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Drug Discovery Landscape
5 orphan drug designations for Diffuse intrinsic pontine glioma.
5 orphan drug designations for Diffuse intrinsic pontine glioma.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Humanised IgG1 kappa monoclonal antibody against erythropoietin isoform EV-3 | antibodies | EMA | 2026-02-23 | — | Andremacon S.r.l. |
Iniparib | small molecules | EMA | 2025-12-09 | — | Raremoon Consulting Esp S.L. |
1,2:5,6-dianhydrogalactitol | small molecules | FDA | 2022-12-14 | — | Kintara Therapeutics, Inc. |
dimethylaminomicheliolide Fumarate | small molecules | FDA | 2022-11-03 | — | Accendatech AU Pty Ltd. |
A10 & AS2-1 Antineoplaston | peptides | FDA | 2004-09-03 | — | Burzynski Research Institute, Inc. |
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