2026-04-13T13:27:08Zhttps://recercat.cat/oai/request

oai:recercat.cat:11351/135642025-10-24T10:36:20Zcom_2072_378070com_2072_378040col_2072_378092

00925njm 22002777a 4500 dc Moreno, Lucas author Anderson, John author Asgharzadeh, Shahab author Bagatell, Rochelle author Beck Popovic, Maja author DuBois, Steven author 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. http://hdl.handle.net/11351/13564 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 Accelerating Drug Development for Neuroblastoma: Consensus Statement From the Third Neuroblastoma Drug Development Strategy Forum