Neuroblastoma (paediatric)
Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease.
Overview
Neuroblastoma arises from developing sympathetic nerve cells, usually in the adrenal gland or along the spine, and is the most common cancer of infants and the most common extracranial solid tumour of childhood. It spans the widest clinical range in oncology: some infant tumours (stage MS) regress without treatment, while high-risk disease (about half of patients, defined by INRG stage, age over 18 months, MYCN amplification and other genomic features) is cured in only 50-60% despite the most intensive therapy given to children, which is where anti-GD2 antibodies and CAR-T have made their gains. ALK mutations (~10%) are the main druggable driver; MYCN, though not directly druggable, points to polyamine and ALK biology.
High-risk therapy is a year-long sequence: five to six cycles of induction chemotherapy, surgery, myeloablative chemotherapy with autologous stem-cell rescue (tandem transplant in North America after ANBL0532; busulfan-melphalan single transplant in Europe after HR-NBL1), radiotherapy to the primary site, then anti-GD2 immunotherapy (dinutuximab or dinutuximab beta with GM-CSF; IL-2 abandoned after HR-NBL1) and isotretinoin. Anti-GD2 antibody raised survival by about 20 points (ANBL0032). Since December 2023, two years of oral eflornithine (DFMO) is approved as maintenance on the strength of an externally controlled study; naxitamab and irinotecan-temozolomide-dinutuximab treat relapse; lorlatinib is being added for ALK-aberrant tumours and 131I-MIBG tested in induction (ANBL1531).
Neuroblastoma is also where CAR-T first produced lasting cures in a solid tumour: GD2-CART01 (Bambino Gesù, NEJM 2023) achieved 63% responses and 33% complete remissions in relapsed disease, with some remissions lasting more than a decade. The open questions are whether cell therapy can consolidate first-line remission, how to reduce the lifelong burden of hearing loss, infertility and second cancers in survivors, how to treat MYCN-amplified relapse, and how to bring anti-GD2 therapy to the majority of children in the world who cannot access it.
State of the art
- GD2 CAR-T responses.
- Anti-GD2 immunotherapy after transplant is standard worldwide (ANBL0032), with IL-2 removed after HR-NBL1 showed no benefit.
- Tandem transplant (North America) and busulfan-melphalan (Europe) are the two evidence-based consolidation standards.
- Eflornithine is the first oral maintenance therapy approved (December 2023), on an externally controlled study.
- GD2 CAR-T produced durable complete remissions in relapsed neuroblastoma, the first such result in a childhood solid tumour.
- ALK inhibition with lorlatinib for ALK-aberrant tumours and 131I-MIBG during induction are being tested in ANBL1531.
- Risk-adapted de-escalation: many infants and low-risk patients are observed or cured with surgery alone.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- Anti-GD2 raised high-risk survival to ~60%.
Anatomy and lymph node drainage
- Renal cortex (RCC)
- Renal pelvis and ureter (upper tract urothelial)
- Bladder lining (non-muscle-invasive)
- Bladder muscle wall (muscle-invasive)
- Adrenal cortex
- Adrenal medulla and sympathetic chain (neuroblastoma)
- Developing kidney (Wilms tumour)
- Nodes: renal hilar
- Nodes: para-aortic and paracaval
- Nodes: obturator and iliac (bladder)
Renal cell carcinoma comes from the kidney's filtering cortex, urothelial cancer from the lining of the collecting system and bladder, and the adrenal on top hosts cortical and medullary (neuroblastoma) tumours.
- Renal cortex (RCC)
- Renal pelvis and ureter (upper tract urothelial)
- Bladder lining (non-muscle-invasive)
- Bladder muscle wall (muscle-invasive)
- Adrenal cortex
- Adrenal medulla and sympathetic chain (neuroblastoma)High risk (stage M >18 months, or MYCN-amplified at any age) · ALK-mutated or amplified (~10%; lorlatinib-responsive) · MYCN-amplified (~20%) · Ganglioneuroblastoma / ganglioneuroma (differentiated spectrum)
- Developing kidney (Wilms tumour)
- renal hilar
- para-aortic and paracaval
- obturator and iliac (bladder)
Same organ: Renal cell carcinoma, High-risk neuroblastoma, Pheochromocytoma and paraganglioma (PPGL), Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), Metastatic pheochromocytoma and paraganglioma
Preclinical models
5 cell lines, 2 mouse models and 4 repositories are listed for this cancer.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- ~7-8% of childhood cancers; ~800 US cases a year; median age at diagnosis ~18 months; 5-year survival >90% for low/intermediate risk and ~50-60% for high risk.
- MIBG imaging and 131I-MIBG therapyEstablished
Nothing recorded yet.
Nothing recorded yet.
Also on Nuclide: Roadmaps.
Advanced diseaseShow Hide where advanced disease can reach, and what treats itThe sites decide which scans are done at staging and follow-up, and every site listed has a treatment approach.
Most people reading this do not have advanced disease. The map describes what can happen over the whole course of the illness, across autopsy and registry series; today's staging scans find spread earlier, and each site has treatments, from focused radiotherapy for a few spots to drugs that reach the brain.
What helpsInduction chemotherapy, transplant, radiotherapy and anti-GD2 antibodies clear marrow and bone disease in most children; GD2 CAR-T has produced lasting remissions at relapse; stage MS liver disease in infants can regress on its own.
Nothing recorded yet.
Nothing recorded yet.
Also on Nuclide: Indications.
- Bone marrowmost common
Marrow involvement defines stage M; MIBG scans and marrow biopsies are part of staging.
- Bone (skull, orbit, long bones)most common
- Lymph nodescommon
- Livercommon
Massive liver involvement in infants (stage MS) can regress on its own.
- Orbit and skinless common
Periorbital bruising (raccoon eyes) and blue skin nodules in infants.
Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant.
Observation with serial imaging for asymptomatic L1/MS (many regress); surgery alone for resectable L1; short chemotherapy only for symptoms or progression.
2-8 cycles of moderate chemotherapy (carboplatin, etoposide, cyclophosphamide, doxorubicin) guided by response and biology; surgery; isotretinoin in some protocols.
5-6 cycles (COG: topotecan-cyclophosphamide × 2 then cisplatin-etoposide, cyclophosphamide-doxorubicin-vincristine; SIOPEN: rapid COJEC); stem-cell harvest; ANBL1531 adds 131I-MIBG (randomised) or lorlatinib (ALK); ANBL17P1 adds dinutuximab to induction.
Surgical resection of primary after induction (gross total where safe); external-beam radiotherapy 21.6 Gy to primary site (boost to residual) and MIBG-avid metastatic sites; proton therapy where available.
Tandem autologous transplant (thiotepa-cyclophosphamide, then CEM) in North America (ANBL0532); single busulfan-melphalan transplant in Europe (HR-NBL1).
Anti-GD2 antibody (dinutuximab + GM-CSF + isotretinoin; dinutuximab beta in Europe, no IL-2) × 5-6 cycles; then eflornithine maintenance 2 years (US, 2023).
Irinotecan-temozolomide + dinutuximab or naxitamab (ANBL1221); naxitamab + GM-CSF for marrow/bone disease; 131I-MIBG for MIBG-avid disease; lorlatinib for ALK; GD2 CAR-T (Italy, trials); DFMO-based maintenance; palliative radiotherapy.
Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic.
Subtypes & biomarkers
top- Very-low and low risk (L1, MS in infants; often observation or surgery alone)
- Intermediate risk (L2, M in infants; moderate chemotherapy)
- High risk (stage M >18 months, or MYCN-amplified at any age)
- ALK-mutated or amplified (~10%; lorlatinib-responsive)
- MYCN-amplified (~20%)
- Relapsed/refractory (MIBG-avid vs non-avid; marrow vs soft-tissue)
- Ganglioneuroblastoma / ganglioneuroma (differentiated spectrum)
- Opsoclonus-myoclonus-associated (paraneoplastic)
- MYCN amplification
- ALK mutation
- Age, stage, ploidy
- Segmental chromosome aberrations
- INRG stage and image-defined risk factors
- Age (<18 months)
- MYCN amplification (FISH)
- ALK mutation/amplification
- 11q aberration, 1p deletion, ploidy, segmental chromosomal aberrations
- INPC histology
- Urinary catecholamines (VMA/HVA)
- 123I-MIBG Curie score / 18F-MFBG PET
- Bone marrow minimal residual disease (PHOX2B, TH qPCR)
- GD2 expression (near-universal)
How often this target appears
- 1910James Homer Wright describes neuroblastoma and its rosettes
- 1971Spontaneous regression of stage IV-S (now MS) disease recognised (Evans staging)
- 1983MYCN amplification linked to aggressive disease (Brodeur, Schwab)
- 1985131I-MIBG therapy first used in relapsed neuroblastoma
- 1999CCG-3891: myeloablative therapy with autologous rescue and isotretinoin improve survival
- 2008ALK mutations identified as a hereditary and somatic driver
- 2009INRG classification unifies international risk grouping
- 2010Anti-GD2 (ch14.18) improves EFS
- 2010ANBL0032: anti-GD2 immunotherapy raises survival ~20 points (NEJM)
- 2015Dinutuximab approved (US); dinutuximab beta in EU 2017
- 2017HR-NBL1: busulfan-melphalan beats CEM; IL-2 adds no benefit (2018)
- 2019ANBL0532: tandem transplant improves EFS (JAMA)
- 2020Naxitamab approved for relapsed disease (accelerated)
- 2023GD2 CAR-T phase 1/2 in NEJM
- 2023GD2-CART01 in NEJM: durable CAR-T remissions in a solid tumour; eflornithine approved (13 Dec)
- 2025Long-term GD2 CAR-T follow-up (Nature Medicine); naxitamab primary-refractory phase 2 (75% CR); eflornithine EU filing
- 2026ANBL1531 MIBG randomisation maturing; ALK arm reports
Dated changes read from the records linked to this indication: approvals, regulatory steps, reported trials, guideline versions and milestones. Newest first; no date is inferred.
All 22 changes by month →- 2026-09-07This recordNeuroblastoma (paediatric)Facts on this page last checked
When this page itself was last checked or edited.
- 2026GuidelineNeuroblastoma (paediatric)Guideline 2026: High risk: consolidation
Tandem autologous transplant (thiotepa-cyclophosphamide, then CEM) in North America (ANBL0532); single busulfan-melphalan transplant in Europe (HR-NBL1). (NCCN COG standard (tandem); SIOPEN standard (BuMel))
- 2026GuidelineNeuroblastoma (paediatric)Guideline 2026: High risk: post-consolidation
Anti-GD2 antibody (dinutuximab + GM-CSF + isotretinoin; dinutuximab beta in Europe, no IL-2) × 5-6 cycles; then eflornithine maintenance 2 years (US, 2023). (NCCN Dinutuximab: FDA-approved standard; eflornithine: FDA-approved maintenance)
- 2026GuidelineNeuroblastoma (paediatric)Guideline COG 2026: High risk: induction
5-6 cycles (COG: topotecan-cyclophosphamide × 2 then cisplatin-etoposide, cyclophosphamide-doxorubicin-vincristine; SIOPEN: rapid COJEC); stem-cell harvest; ANBL1531 adds 131I-MIBG (randomised) or lorlatinib (ALK); ANBL17P1 adds dinutuximab to induction. (NCCN COG ANBL1531 backbone)
- 2026MilestoneCOG ANBL1531ANBL1531 MIBG randomisation maturing; ALK arm reports
A milestone in how this cancer is treated.
- 2025MilestoneNeuroblastoma (paediatric)Long-term GD2 CAR-T follow-up (Nature Medicine); naxitamab primary-refractory phase 2 (75% CR); eflornithine EU filing
A milestone in how this cancer is treated.
What is in development for Neuroblastoma (paediatric), drawn from the whole corpus: 3 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Trials under way · 2
- COG ANBL1531 · phase 3 · Children's Oncology Group
- Open-Label Study of 18F-mFBG for Imaging Neuroblastoma · phase 3 · Innervate Radiopharmaceuticals LLC (Formerly: Illumina Radiopharmaceuticals LLC)
Ideas not yet in a trial · 1
Open problems and what is being done
Relapsed high-risk disease.
What is being done about thisRecurrence and residual diseaseAvailable now- 131I-MIBG (iobenguane I-131) therapyEstablished
In trialsNothing recorded yet.
Ideas and roadmapsNothing recorded yet.
Also on Nuclide: Indications.
Long-term toxicity of intensive therapy.
Relapsed high-risk neuroblastoma is rarely curable; MYCN-amplified relapse worst of all.
What is being done about thisRecurrence and residual diseaseAvailable now- 131I-MIBG (iobenguane I-131) therapyEstablished
In trialsNothing recorded yet.
Ideas and roadmapsNothing recorded yet.
Also on Nuclide: Indications.
Anti-GD2 therapy causes severe neuropathic pain; less painful antibody formats (e.g., humanised, Fc-engineered) and CAR-T are needed.
Long-term toxicity of tandem transplant, cisplatin (hearing loss), and radiation in children who will live 70 years.
Eflornithine's approval rests on an external control; a randomised trial is unlikely, so uncertainty persists.
Access: anti-GD2 antibodies are expensive and unavailable in most low- and middle-income countries where most children with cancer live.
MYCN remains undruggable directly; polyamine and Aurora/BET strategies are indirect.
GD2 CAR-T needs randomised evidence and manufacturing at scale; only a few centres can deliver it.
Imaging burden: repeated MIBG scans with sedation; MFBG PET adoption is slow.
Trials
topTrials recruiting now
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Landmark trials
Direct links plus the targets, companies, and technologies of this cancer's products.
isotopes
2technologies
4targets
1drugs
2companies
3institutions
3terms
1trials
2ideas
1people
2Latest papers
topQuery for this indication: (TITLE:"Neuroblastoma" OR ABSTRACT:"Neuroblastoma" OR TITLE:"paediatric" OR ABSTRACT:"paediatric") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Neuroblastoma (paediatric), not a curated reading list.
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