Price M, Ballard C, Benedetti J, Neff C, Cioffi G, Waite KA, Kruchko C, Barnholtz-Sloan JS, Ostrom QT (2024) CBTRUS statistical report: primary brain and other central nervous system tumors diagnosed in the united States in 2017–2021. Neurooncology 26(Supplement6):vi1–vi85. https://doi.org/10.1093/neuonc/noae145
Cavalli FMG, Remke M, Rampasek L, Peacock J, Shih DJH, Luu B, Garzia L, Torchia J, Nor C, Morrissy AS, Agnihotri S, Thompson YY, Kuzan-Fischer CM, Farooq H, Isaev K, Daniels C, Cho BK, Kim SK, Wang KC, Lee JY, Taylor MD (2017) Intertumoral heterogeneity within Medulloblastoma subgroups. Cancer Cell 31(6):737–754e6. https://doi.org/10.1016/j.ccell.2017.05.005
Article CAS PubMed PubMed Central Google Scholar
Ramaswamy V, Remke M, Bouffet E, Bailey S, Clifford SC, Doz F, Kool M, Dufour C, Vassal G, Milde T, Witt O, von Hoff K, Pietsch T, Northcott PA, Gajjar A, Robinson GW, Padovani L, André N, Massimino M, Pizer B, Pomeroy SL (2016) Risk stratification of childhood Medulloblastoma in the molecular era: the current consensus. Acta Neuropathol 131(6):821–831. https://doi.org/10.1007/s00401-016-1569-6
Article CAS PubMed PubMed Central Google Scholar
Gajjar A, Robinson GW, Smith KS, Lin T, Merchant TE, Chintagumpala M, Mahajan A, Su J, Bouffet E, Bartels U, Schechter T, Hassall T, Robertson T, Nicholls W, Gururangan S, Schroeder K, Sullivan M, Wheeler G, Hansford JR, Kellie SJ, Northcott PA (2021) Outcomes by clinical and molecular features in children with Medulloblastoma treated with Risk-Adapted therapy: results of an international phase III trial (SJMB03). J Clin Oncology: Official J Am Soc Clin Oncol 39(7):822–835. https://doi.org/10.1200/JCO.20.01372
Northcott PA, Robinson GW, Kratz CP, Mabbott DJ, Pomeroy SL, Clifford SC, Rutkowski S, Ellison DW, Malkin D, Taylor MD, Gajjar A, Pfister SM (2019) Medulloblastoma Nat Reviews Disease Primers 5(1):11. https://doi.org/10.1038/s41572-019-0063-6
Tabori U, Sung L, Hukin J, Laperriere N, Crooks B, Carret AS, Silva M, Odame I, Mpofu C, Strother D, Wilson B, Samson Y, Bouffet E, Canadian Pediatric Brain Tumor Consortium (2006) Distinctive clinical course and pattern of relapse in adolescents with Medulloblastoma. Int J Radiat Oncol Biol Phys 64(2):402–407. https://doi.org/10.1016/j.ijrobp.2005.07.962
Hwang EI, Sayour EJ, Flores CT, Grant G, Wechsler-Reya R, Hoang-Minh LB, Kieran MW, Salcido J, Prins RM, Figg JW, Platten M, Candelario KM, Hale PG, Blatt JE, Governale LS, Okada H, Mitchell DA, Pollack IF (2022) The current landscape of immunotherapy for pediatric brain tumors. Nat Cancer 3(1):11–24. https://doi.org/10.1038/s43018-021-00319-0
Das A, Tabori U, Nahum S, Collins LC, Deyell NB, Dvir R, Faure-Conter R, Hassall C, Minturn TE, Edwards JE, Brookes M, Bianchi E, Levine V, Stone A, Sudhaman SC, Sanchez Ramirez S, Ercan S, Stengs AB, Chung L, Negm J, Morgenstern L, D. A (2023) Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch repair deficiency. Clin Cancer Research: Official J Am Association Cancer Res 29(23):4770–4783. https://doi.org/10.1158/1078-0432.CCR-23-0411
Das A, Sudhaman S, Morgenstern D, Coblentz A, Chung J, Stone SC, Alsafwani N, Liu ZA, Karsaneh OAA, Soleimani S, Ladany H, Chen D, Zatzman M, Cabric V, Nobre L, Bianchi V, Edwards M, Nahum S, Ercan LC, Nabbi AB, Tabori A, U (2022) Genomic predictors of response to PD-1 Inhibition in children with germline DNA replication repair deficiency. Nat Med 28(1):125–135. https://doi.org/10.1038/s41591-021-01581-6
Article CAS PubMed PubMed Central Google Scholar
Lin FY, Stuckert A, Tat C, White M, Ruggieri L, Zhang H, Mehta B, Lapteva N, Mei Z, Major A, Thakkar S, Shum T, Parikh K, Wu MF, Lindsay HB, Scherer L, Shekar M, Baxter P, Wang T, Grilley B, Omer B (2024) Phase I trial of GD2.CART cells augmented with constitutive Interleukin-7 receptor for treatment of High-Grade pediatric CNS tumors. J Clin Oncology: Official J Am Soc Clin Oncol 42(23):2769–2779. https://doi.org/10.1200/JCO.23.02019
Monje M, Mahdi J, Majzner R, Yeom KW, Schultz LM, Richards RM, Barsan V, Song KW, Kamens J, Baggott C, Kunicki M, Rietberg SP, Lim AS, Reschke A, Mavroukakis S, Egeler E, Moon J, Patel S, Chinnasamy H, Erickson C, Mackall C (2025) Intravenous and intracranial GD2-CAR T cells for H3K27M + diffuse midline gliomas. Nature 637(8046):708–715. https://doi.org/10.1038/s41586-024-08171-9
Article CAS PubMed Google Scholar
Vitanza NA, Ronsley R, Choe M, Seidel K, Huang W, Rawlings-Rhea SD, Beam M, Steinmetzer L, Wilson AL, Brown C, Beebe A, Lindgren C, Gustafson JA, Wein A, Holtzclaw S, Hoeppner C, Goldstein HE, Browd SR, Hauptman JS, Lee A, Jensen MC (2025) Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic Pontine glioma: a phase 1 trial. Nat Med 31(3):861–868. https://doi.org/10.1038/s41591-024-03451-3
Article CAS PubMed PubMed Central Google Scholar
Louis DN, Perry A, Wesseling P, Brat DJ, Cree IA, Figarella-Branger D, Hawkins C, Ng HK, Pfister SM, Reifenberger G, Soffietti R, von Deimling A, Ellison DW (2021) The 2021 WHO classification of tumors of the central nervous system: a summary. Neurooncology 23(8):1231–1251. https://doi.org/10.1093/neuonc/noab106
Hovestadt V, Ayrault O, Swartling FJ, Robinson GW, Pfister SM, Northcott PA (2020) Medulloblastomics revisited: biological and clinical insights from thousands of patients. Nat Rev Cancer 20(1):42–56. https://doi.org/10.1038/s41568-019-0223-8
Article CAS PubMed Google Scholar
Liu Y, Su Z, Tavana O, Gu W (2024) Understanding the complexity of p53 in a new era of tumor suppression. Cancer Cell 42(6):946–967. https://doi.org/10.1016/j.ccell.2024.04.009
Article CAS PubMed PubMed Central Google Scholar
Suzuki H, Kumar SA, Shuai S, Diaz-Navarro A, Gutierrez-Fernandez A, De Antonellis P, Cavalli FMG, Juraschka K, Farooq H, Shibahara I, Vladoiu MC, Zhang J, Abeysundara N, Przelicki D, Skowron P, Gauer N, Luu B, Daniels C, Wu X, Forget A, Taylor MD (2019) Recurrent noncoding U1 SnRNA mutations drive cryptic splicing in SHH Medulloblastoma. Nature 574(7780):707–711. https://doi.org/10.1038/s41586-019-1650-0
Article CAS PubMed PubMed Central Google Scholar
Wu Q, Jiang L, Li SC, He QJ, Yang B, Cao J (2021) Small molecule inhibitors targeting the PD-1/PD-L1 signaling pathway. Acta Pharmacol Sin 42(1):1–9. https://doi.org/10.1038/s41401-020-0366-x
Article CAS PubMed Google Scholar
Dunkel IJ, Doz F, Foreman NK, Hargrave D, Lassaletta A, André N, Hansford JR, Hassall T, Eyrich M, Gururangan S, Bartels U, Gajjar A, Howell L, Warad D, Pacius M, Tam R, Wang Y, Zhu L, Cohen K (2023) Nivolumab with or without ipilimumab in pediatric patients with high-grade CNS malignancies: Safety, efficacy, biomarker, and pharmacokinetics-CheckMate 908. Neurooncology 25(8):1530–1545. https://doi.org/10.1093/neuonc/noad031
Ramaswamy V, Remke M, Bouffet E, Faria CC, Perreault S, Cho YJ, Shih DJ, Luu B, Dubuc AM, Northcott PA, Schüller U, Gururangan S, McLendon R, Bigner D, Fouladi M, Ligon KL, Pomeroy SL, Dunn S, Triscott J, Jabado N, Taylor MD (2013) Recurrence patterns across Medulloblastoma subgroups: an integrated clinical and molecular analysis. Lancet Oncol 14(12):1200–1207. https://doi.org/10.1016/S1470-2045(13)70449-2
Article CAS PubMed PubMed Central Google Scholar
Das A, Fernandez N, Levine A, Smith K, Wang E, Galati M, Aamir Z, Chung J, Negm L, Friedman H, O’Flaherty K, Crump O, Trinh Q, Nunes N, Bianchi V, Stengs L, Edwards M, Stein L, Bouffet E, Taylor M, Northcott P, Ramaswamy V, Hawkins C, Tabori U (2024) IMMU–14. Trans–species analysis of replication–repair deficient (RRD) Medulloblastoma and response to immune checkpoint inhibition: an IRRDC report. Neuro–Oncology 26(Suppl 8):viii154–viii155. https://doi.org/10.1093/neuonc/noae165.0607
Article PubMed Central Google Scholar
Machy P, Mortier E, Birklé S (2023) Biology of GD2 ganglioside: implications for cancer immunotherapy. Front Pharmacol 14:1249929. https://doi.org/10.3389/fphar.2023.1249929
Article CAS PubMed PubMed Central Google Scholar
Filbin M, Monje M (2019) Developmental origins and emerging therapeutic opportunities for childhood cancer. Nat Med 25(3):367–376. https://doi.org/10.1038/s41591-019-0383-9
Article CAS PubMed PubMed Central Google Scholar
Svennerholm L, Rynmark BM, Vilbergsson G, Fredman P, Gottfries J, Månsson JE, Percy A (1991) Gangliosides in human fetal brain. J Neurochem 56(5):1763–1768. https://doi.org/10.1111/j.1471-4159.1991.tb02078.x
Article CAS PubMed Google Scholar
Svennerholm L, Boström K, Fredman P, Jungbjer B, Lekman A, Månsson JE, Rynmark BM (1994) Gangliosides and allied glycosphingolipids in human peripheral nerve and spinal cord. Biochim Biophys Acta 1214(2):115–123. https://doi.org/10.1016/0005-2760(94)90034-5
Comments (0)