Sistema de Salut de Catalunya
Institut Català de la Salut
[DuBois SG] Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, Massachusetts, USA. [Moreno L] Servei de Pediatria, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Anderson J] University College London Great Ormond Street Institute of Child Health and Zayed Centre for Research in Rare Diseases of Childhood, London, UK. [Asgharzadeh S] Division of Hematology-Oncology, Department of Pediatrics, Children’s Hospital Los Angeles and University of Southern California, Los Angeles, California, USA. [Bagatell R] Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia and Perelman School of Medicine, Philadelphia, Pennsylvania, USA. [Beck-Popovic M] Pediatric Hematology Oncology Unit, University Hospital CHUV, Lausanne, Switzerland
Vall d'Hebron Barcelona Hospital Campus
2025-08-21T11:45:18Z
2025-08-21T11:45:18Z
2025-09
Drug development; Neuroblastoma; Relapse
Desarrollo de medicamentos; Neuroblastoma; Recaída
Desenvolupament de medicaments; Neuroblastoma; Recaiguda
High-risk neuroblastoma is a poor prognosis cancer of the sympathetic nervous system that accounts for a disproportionate number of childhood cancer deaths. Many viable biological targets have been identified, and the number of potential combinations is even larger. Several products have attained marketing authorization for treatment of patients with neuroblastoma. Patient outcomes remain poor, with approximately 50% of children with newly diagnosed high-risk neuroblastoma cured of their disease. International, multistakeholder Neuroblastoma Drug Development Strategy (NDDS) meetings were established more than a decade ago. This third NDDS meeting included academia, industry, regulatory, and patient advocacy representatives to prioritize agents and to address key challenges in drug development in this disease. Given the central role that anti-GD2 therapy plays, novel GD2-directed combinations were a key focus, including epigenetic enzymes such as EZH2 and immunologic targets such as IL15 and TIGIT as potential combination partners. GD2-directed chimeric antigen receptor (CAR)-T cells were a top priority, along with emerging CAR-T targets such as B7-H3 and GPC2. Recognizing that combination therapies are likely to be most impactful for patients and for advancing therapies to frontline, another key focus was on high priority combinations of targeted therapies, including Aurora A kinase plus BCL2 or ATR inhibitors. Additional targets and agents were prioritized or deprioritized based upon current data. Access to drugs for clinical trials was viewed as a major barrier to progress. Strategies to overcome this challenge focused on united efforts by the international scientific and advocacy community and early engagement by industry with regulatory authorities.
This work was supported by Accelerate the Future Fund; Alex's Lemonade Stand Foundation; Dana-Farber Cancer Institute; and Friends for Life.
Article
Published version
English
Decisió, Presa de; Infants; Neuroblastoma - Tractament; Medicaments antineoplàstics - Ús terapèutic; Medicaments - Desenvolupament; CHEMICALS AND DRUGS::Chemical Actions and Uses::Pharmacologic Actions::Therapeutic Uses::Antineoplastic Agents; Other subheadings::Other subheadings::/therapeutic use; PSYCHIATRY AND PSYCHOLOGY::Behavior and Behavior Mechanisms::Psychology, Social::Group Processes::Consensus; ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development; DISEASES::Neoplasms::Neoplasms by Histologic Type::Neoplasms, Germ Cell and Embryonal::Neuroectodermal Tumors::Neoplasms, Neuroepithelial::Neuroectodermal Tumors, Primitive::Neuroectodermal Tumors, Primitive, Peripheral::Neuroblastoma; Other subheadings::Other subheadings::Other subheadings::/drug therapy; NAMED GROUPS::Persons::Age Groups::Child; COMPUESTOS QUÍMICOS Y DROGAS::acciones y usos químicos::acciones farmacológicas::usos terapéuticos::antineoplásicos; Otros calificadores::Otros calificadores::/uso terapéutico; PSIQUIATRÍA Y PSICOLOGÍA::conducta y mecanismos de la conducta::psicología social::procesos de grupo::consenso; TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos; ENFERMEDADES::neoplasias::neoplasias por tipo histológico::neoplasias de células germinales y embrionarias::tumores neuroectodérmicos::neoplasias neuroepiteliales::tumores neuroectodérmicos primitivos::tumores neuroectodérmicos primitivos periféricos::neuroblastoma; Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia; DENOMINACIONES DE GRUPOS::personas::Grupos de Edad::niño
Wiley
Pediatric Blood & Cancer;72(9)
https://doi.org/10.1002/pbc.31831
Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
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