Yue C, Zhang Q, Sun F, Pan Q. Neoplasia. 2025;60:101122. https://doi.org/10.1016/j.neo.2025.101122. Global, regional and national burden of neuroblastoma and other peripheral nervous system tumors, 1990 to 2021 and predictions to 2035: visualizing epidemiological characteristics based on GBD 2021.

Campbell K, Siegel DA, Umaretiya PJ, Dai S, Heczey A, Lupo PJ, et al. A comprehensive analysis of neuroblastoma incidence, survival, and racial and ethnic disparities from 2001 to 2019. Pediatr Blood Cancer. 2024;71:e30732. https://doi.org/10.1002/pbc.30732.

Article  CAS  PubMed  Google Scholar 

Yan P, Qi F, Bian L, Xu Y, Zhou J, Hu J, et al. Comparison of Incidence and Outcomes of Neuroblastoma in Children, Adolescents, and Adults in the United States: A Surveillance, Epidemiology, and End Results (SEER) Program Population Study. Med Sci Monit. 2020;26:e927218. https://doi.org/10.12659/MSM.927218.

Article  PubMed  PubMed Central  Google Scholar 

van Heerden J, Abraham N, Schoeman J, Reynders D, Singh E, Kruger M. Reporting Incidences of Neuroblastoma in Various Resource Settings. JCO Glob Oncol. 2021;7:947–64. https://doi.org/10.1200/GO.21.00054.

Article  PubMed  PubMed Central  Google Scholar 

Ploessl C, Pan A, Maples KT, Lowe DK. Dinutuximab: An Anti-GD2 Monoclonal Antibody for High-Risk Neuroblastoma. Ann Pharmacother. 2016;50:416–22. https://doi.org/10.1177/1060028016632013.

Article  CAS  PubMed  Google Scholar 

Markham A, Naxitamab. First Approval Drugs. 2021;81:291–6. https://doi.org/10.1007/s40265-021-01467-4.

Article  CAS  PubMed  Google Scholar 

Wu ZL, Schwartz E, Seeger R, Ladisch S. Expression of GD2 ganglioside by untreated primary human neuroblastomas. Cancer Res janv. 1986;46:440–3.

CAS  Google Scholar 

Svennerholm L, Boström K, Fredman P, Jungbjer B, Lekman A, Månsson JE, et al. Gangliosides and allied glycosphingolipids in human peripheral nerve and spinal cord. Biochim Biophys Acta. 1994;1214:115–23. https://doi.org/10.1016/0005-2760(94)90034-5.

Article  CAS  PubMed  Google Scholar 

Yuki N, Yamada M, Tagawa Y, Takahashi H, Handa S. Pathogenesis of the neurotoxicity caused by anti-GD2 antibody therapy. J Neurol Sci. 1997;149:127–30.

Article  CAS  PubMed  Google Scholar 

Alvarez-Rueda N, Desselle A, Cochonneau D, Chaumette T, Clemenceau B, Leprieur S, et al. A monoclonal antibody to O-acetyl-GD2 ganglioside and not to GD2 shows potent anti-tumor activity without peripheral nervous system cross-reactivity. PLoS ONE. 2011;6:e25220. https://doi.org/10.1371/journal.pone.0025220.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Machy P, Mortier E, Birklé S. Biology of GD2 ganglioside: implications for cancer immunotherapy. Front Pharmacol. 2023;14:1249929. https://doi.org/10.3389/fphar.2023.1249929.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Di Matteo S, Bilotta MT, Pelosi A, Haas D, Theinert T, Weber G, et al. Transition to a mesenchymal state in neuroblastoma may be characterized by a high expression of GD2 and by the acquisition of immune escape from NK cells. Front Immunol. 2024;15:1382931. https://doi.org/10.3389/fimmu.2024.1382931.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Valentino L, Moss T, Olson E, Wang HJ, Elashoff R, Ladisch S. Shed tumor gangliosides and progression of human neuroblastoma. Blood. 1990;75:1564–7.

Article  CAS  PubMed  Google Scholar 

Li R, Gage D, McKallip R, Ladisch S. Structural characterization and in vivo immunosuppressive activity of neuroblastoma GD2. Glycoconj J. 1996;13:385–9. https://doi.org/10.1007/BF00731471.

Article  CAS  PubMed  Google Scholar 

Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010;363:1324–34. https://doi.org/10.1056/NEJMoa0911123.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ladenstein R, Pötschger U, Valteau-Couanet D, Luksch R, Castel V, Yaniv I. Interleukin 2 with anti-GD2 antibody ch14.18/CHO (dinutuximab beta) in patients with high-risk neuroblastoma (HR-NBL1/SIOPEN): a multicentre, randomised, phase 3 trial. Lancet Oncol déc. 2018;19:1617–29.

Article  CAS  Google Scholar 

Cheever MA, Allison JP, Ferris AS, Finn OJ, Hastings BM, Hecht TT, et al. The prioritization of cancer antigens: a national cancer institute pilot project for the acceleration of translational research. Clin Cancer Res. 2009;15:5323–37. https://doi.org/10.1158/1078-0432.CCR-09-0737.

Article  PubMed  PubMed Central  Google Scholar 

Mujoo K, Cheresh DA, Yang HM, Reisfeld RA. Disialoganglioside GD2 on human neuroblastoma cells: target antigen for monoclonal antibody-mediated cytolysis and suppression of tumor growth. Cancer Res. 1987;47:1098–104.

CAS  PubMed  Google Scholar 

Modak S, Cheung N-KV. Disialoganglioside directed immunotherapy of neuroblastoma. Cancer Invest. 2007;25:67–77. https://doi.org/10.1080/07357900601130763.

Article  CAS  PubMed  Google Scholar 

Quintarelli C, Orlando D, Boffa I, Guercio M, Polito VA, Petretto A, et al. Choice of costimulatory domains and of cytokines determines CAR T-cell activity in neuroblastoma. Oncoimmunology. 2018;7:e1433518. https://doi.org/10.1080/2162402X.2018.1433518.

Article  PubMed  PubMed Central  Google Scholar 

Long AH, Haso WM, Shern JF, Wanhainen KM, Murgai M, Ingaramo M, et al. 4-1BB costimulation ameliorates T cell exhaustion induced by tonic signaling of chimeric antigen receptors. Nat Med juin. 2015;21:581–90.

Article  CAS  Google Scholar 

Weber EW, Parker KR, Sotillo E, Lynn RC, Anbunathan H, Lattin J, et al. Transient rest restores functionality in exhausted CAR-T cells through epigenetic remodeling. Science. 2021;372:eaba1786. https://doi.org/10.1126/science.aba1786.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Lynn RC, Weber EW, Sotillo E, Gennert D, Xu P, Good Z, et al. c-Jun overexpression in CAR T cells induces exhaustion resistance. Nature. 2019;576:293–300. https://doi.org/10.1038/s41586-019-1805-z.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Richman SA, Nunez-Cruz S, Moghimi B, Li LZ, Gershenson ZT, Mourelatos Z. High-Affinity GD2-Specific CAR T Cells Induce Fatal Encephalitis in a Preclinical Neuroblastoma Model. Cancer Immunol Res janv. 2018;6:36–46.

Article  CAS  Google Scholar 

Majzner RG, Weber EW, Lynn RC, Xu P, Mackall CL. Neurotoxicity Associated with a High-Affinity GD2 CAR-Letter. Cancer Immunol Res. 2018;6:494–5. https://doi.org/10.1158/2326-6066.CIR-18-0089.

Article  PubMed  Google Scholar 

Hoseini SS, Dobrenkov K, Pankov D, Xu XL, Cheung N-KV. Bispecific antibody does not induce T-cell death mediated by chimeric antigen receptor against disialoganglioside GD2. Oncoimmunology. 2017;6:e1320625. https://doi.org/10.1080/2162402X.2017.1320625.

Article  PubMed  PubMed Central  Google Scholar 

Monje M, Mahdi J, Majzner R, Yeom KW, Schultz LM, Richards RM, et al. Intravenous and intracranial GD2-CAR T cells for H3K27M+ diffuse midline gliomas. Nature. 2025;637:708–15. https://doi.org/10.1038/s41586-024-08171-9.

Article  CAS  PubMed  Google Scholar 

Louis CU, Savoldo B, Dotti G, Pule M, Yvon E, Myers GD. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood. 2011;118:6050–6.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Pule MA, Savoldo B, Myers GD, Rossig C, Russell HV, Dotti G, et al. Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma. Nat Med. 2008;14:1264–70. https://doi.org/10.1038/nm.1882.

Article  CAS  PubMed