Neuroblastoma
Recent clinical, regulatory, research and industry developments relating to this disease.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q4 2028.
- 2 industry developments reported.
- Q4 2028Phase II Study of Ex-Vivo Expanded Allogeneic Universal Donor TGFβi NK Cell Infusions in Combination With Temozolomide, Irinotecan, Dinutuximab, and Sargramostim in Patients With Relapsed or Refractory Neuroblastoma The STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial
- Q3 2029A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk Neuroblastoma
- Q4 2029A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma
- Q1 2031A Phase 2/3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination With Chemotherapy in Participants With High-Risk Neuroblastoma
Clinical MilestonesView all 12Hide
- 2026-06-24A Comprehensive Safety Trial of Chimeric Antibody 14.18 (Ch14.18) With GM-CSF, IL-2 and Isotretinoin in High-Risk Neuroblastoma Patients Following Myeloablative TherapyResults posted
- 2026-06-02A Pilot Induction Regimen Incorporating Chimeric 14.18 Antibody (ch14.18, Dinutuximab) (NSC# 764038) and Sargramostim (GM-CSF) for the Treatment of Newly Diagnosed High-Risk NeuroblastomaResults posted
- 2026-03-05Phase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell RescueResults posted
- 2026-10-08A Phase 2/3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination With Chemotherapy in Participants With High-Risk NeuroblastomaResults expected Q1 2031
- 2026-08-19A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk NeuroblastomaResults expected Q4 2029
- 2026-08-12A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk NeuroblastomaResults expected Q3 2029
- 2026-07-20Phase II Study of Ex-Vivo Expanded Allogeneic Universal Donor TGFβi NK Cell Infusions in Combination With Temozolomide, Irinotecan, Dinutuximab, and Sargramostim in Patients With Relapsed or Refractory Neuroblastoma The STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) TrialResults expected Q4 2028
- 2026-09-30Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk NeuroblastomaPrimary completion
- 2026-09-01Introduction of Induction Chemoimmunotherapy Regimen for the Treatment of Pediatric Patients With Stage 4 High-risk Neuroblastoma and Ganglioneuroblastoma Older 18 MonthsPrimary completion
- 2026-08-30A Prospective Phase 3 Multi-center Study to Assess the Efficacy and Safety of 18F-mFBG PET Imaging in Subjects With NeuroblastomaCompleted
- 2027-05-31Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy in Patients With High-Risk Neuroblastoma Receiving Tandem TransplantsWithdrawn
- 2027-04-01Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission. An International, Open-Label,Uncontrolled, Single-Arm, Multicenter, Phase 2 TrialWithdrawn
Industry & MarketViewHide
- 2026-11-03AAV-STITCH shows activity across neuroblastoma modelsIndustry · BioWorld Gene Therapy
- 2026-10-08Prognostic Impact of Telomere Maintenance Mechanisms in NeuroblastomaIndustry · Journal of Clinical Oncology
- 2027-05-31ClinicalDefibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy in Patients With High-Risk Neuroblastoma Receiving Tandem TransplantsWithdrawn
- 2027-04-01ClinicalNaxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission. An International, Open-Label,Uncontrolled, Single-Arm, Multicenter, Phase 2 TrialWithdrawn
- 2026-11-03IndustryAAV-STITCH shows activity across neuroblastoma modelsIndustry · BioWorld Gene Therapy
- 2026-10-08IndustryPrognostic Impact of Telomere Maintenance Mechanisms in NeuroblastomaIndustry · Journal of Clinical Oncology
- 2026-10-08ClinicalA Phase 2/3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination With Chemotherapy in Participants With High-Risk NeuroblastomaResults expected Q1 2031
- 2026-09-30ClinicalUtilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk NeuroblastomaPrimary completion
- 2026-09-01ClinicalIntroduction of Induction Chemoimmunotherapy Regimen for the Treatment of Pediatric Patients With Stage 4 High-risk Neuroblastoma and Ganglioneuroblastoma Older 18 MonthsPrimary completion
- 2026-08-30ClinicalA Prospective Phase 3 Multi-center Study to Assess the Efficacy and Safety of 18F-mFBG PET Imaging in Subjects With NeuroblastomaCompleted
- 2026-08-19ClinicalA Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk NeuroblastomaResults expected Q4 2029
- 2026-08-12ClinicalA Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk NeuroblastomaResults expected Q3 2029
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Original application approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)
Original application approval — Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 mon… (2017)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Neuroblastoma8
- Neurotoxicity Syndromes2
- Alzheimer Disease1
- Bone Marrow Transplantation1
- Brain-Derived Neurotrophic Factor1
- Cell Differentiation1
- Environmental Pollutants1
- Exosomes1
Leading journals6
- The New England journal of medicine2
- Blood1
- Cell and tissue research1
- Cell communication and signaling : CCS1
- Ecotoxicology and environmental safety1
- In vitro cellular & developmental biology. Animal1
Leading researchers8
- Cohn SL2
- Grupp SA2
- Kreissman SG2
- London WB2
- Maris JM2
- Matthay KK2
- Reynolds CP2
- Shimada H2
Affiliations (unnormalised)6
- Boston Children's Hospital1
- Case Western Reserve University1
- Center for Cell and Gene Therapy1
- Centre for Research in Biosciences1
- Children's Hospital at Westmead1
- Chonnam Notational University1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Neuroblastoma is a common childhood neoplasm that arises from neural crest cells in the sympathetic nervous system. It is the most common intraabdominal malignancy of childhood, but it can also originate in the thorax, neck, or rarely the central nervous system. Its clinical behavior is highly variable, ranging from spontaneous remission to rapidly progressive metastatic disease.
The grounding supports a genetic contribution to neuroblastoma, with literature focusing on molecular signatures and genetic variations involved in its pathogenesis. It also notes that numerous genetic and genomic alterations and dysfunctional pathways drive disease onset, growth, progression, and resistance to therapy. No single causal exposure or definitive aetiology is specified in the supplied material.
Neuroblastoma develops from neural crest-derived cells in the sympathetic nervous system and shows characteristic histology of uniform round cells with hyperchromatic nuclei arranged in nests separated by fibrovascular septa. The literature grounding emphasizes genetic and genomic alterations, dysfunctional pathways, and phosphorylation-related mechanisms as contributors to tumor initiation, progression, and treatment resistance. The disease is also associated with opsoclonus-myoclonus syndrome.
The supplied grounding does not identify specific epidemiologic or environmental risk factors. It does indicate that genetic and genomic alterations are associated with the disease, but does not define these as patient-level risk factors. No additional risk factors are supported by the provided material.
The grounding supports treatment at the modality level as drug therapy and molecular targeting therapies, reflecting efforts to use targeted approaches against disease-driving pathways. Isotretinoin is co-studied, indicating a role for retinoid-based therapy in the literature, and monoclonal antibodies are also represented among co-studied entities. The supplied material does not support more specific standard treatment details beyond these broad categories.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
A common neoplasm of early childhood arising from neural crest cells in the sympathetic nervous system, and characterized by diverse clinical behavior, ranging from spontaneous remission to rapid metastatic progression and death. This tumor is the most common intraabdominal malignancy of childhood, but it may also arise from thorax, neck, or rarely occur in the central nervous system. Histologic features include uniform round cells with hyperchromatic nuclei arranged in nests and separated by fibrovascular septa. Neuroblastomas may be associated with the opsoclonus-myoclonus syndrome. (From DeVita et al., Cancer: Principles and Practice of Oncology, 5th ed, pp2099-2101; Curr Opin Oncol 1998 Jan;10(1):43-51)
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.