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Disease

Neuroblastoma

Late-stage therapeutic developmentEmerging researchRising momentum
13
Publications
21
Clinical trials
2
Related conditions
2
Related treatments
3
Related proteins
2024
Latest publication
Current focus
Amyloid beta biologyAntibodies biologyTherapeutic developmentInflammation & immunity
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Approval: Phelinun (EMA)

Regulatory2020-11-16EMA

Impaired balance of mitochondrial fission and fusion in Alzheimer's disease.

Research2009-07-01The Journal of neuroscience : the official journal of the Society for Neuroscience

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Clinical Milestones9View all 9
+1 more in the activity timeline below
Activity timeline9

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)

Dinutuximabapproved

Approval — Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 mon… (2017)

Research-associated treatments

Clinical trials

11 sponsors · 1 new · 2 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
21
All trials
6
Active
12
Late-stage
6
Completed
Late-stage studies
Recruiting
Recently completed

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2020emaApprovalMelphalan hydrochloride· High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases. source ↗
2017emaApprovalDinutuximab· Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin 2 (IL 2). source ↗

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

13 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
19992024
Most influential
Recent publications
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

Disease biology

3 matches

Key proteins & gene products studied in this disease. Number shows shared papers.

Related conditions

2 matches

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.

Overview

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.

Causes

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.

Pathophysiology

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.

Risk factors

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.

Current standard of care

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.

Defined in MeSH

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)

References & data sources
  • 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.