OnCo
cancersCancer

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.

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) kills roughly 40-50% despite the most intensive therapy given to children. 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 today

  • 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%.
Who it affects
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.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. Neuroblastoma is grouped under 'other specified cancers'; GLOBOCAN has no paediatric tumour-type breakdown. See IARC's International Incidence of Childhood Cancer instead.

Standard of care

9top
High-risk

Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant.

NCI PDQ: Neuroblastoma Treatment (COG risk groups)
LorlatinibMonoclonal antibodies
Very-low / low risk (L1, MS)

Observation with serial imaging for asymptomatic L1/MS (many regress); surgery alone for resectable L1; short chemotherapy only for symptoms or progression.

NCCN · COG/SIOPEN low-risk protocols (observation …
INRG staging and risk groupsUltrasound
Intermediate risk

2-8 cycles of moderate chemotherapy (carboplatin, etoposide, cyclophosphamide, doxorubicin) guided by response and biology; surgery; isotretinoin in some protocols.

DoxorubicinCytotoxic chemotherapyINRG staging and risk groups
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
COG ANBL1531131I-MIBG (iobenguane I-131) therapyLorlatinibDinutuximab (ch14.18) / dinutuximab betaDoxorubicin
High risk: local control

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.

IMRT / IGRT (modern external beam)Proton therapy
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 (BuM…
Tandem autologous transplantAutologous stem cell transplant (high-dose therapy)COG ANBL0532SIOPEN HR-NBL1
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; eflorni…
Dinutuximab (ch14.18) / dinutuximab betaCOG ANBL0032Eflornithine (DFMO)NMTRC003/003B (DFMO maintenance)Caution: IL-2 added to anti-GD2 therapy
Relapsed / refractory

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.

Dinutuximab (ch14.18) / dinutuximab betaNaxitamabNaxitamab Study 201131I-MIBG (iobenguane I-131) therapyLorlatinibGD2-CART01 (Bambino Gesù phase 1/2)Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy)
Survivorship

Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic.

Cardio-oncology

Subtypes & biomarkers

top
Subtypes
  • 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)
Biomarkers clinicians test

Target prevalence in this cancer

Target / alterationPrevalenceSource
ALK
Higher at relapse
8-14%
Wikipedia

How common each drug target or alteration is in this cancer. Population-level and approximate; see the target page for detail. Full matrix.

History

17top
  1. 1910James Homer Wright describes neuroblastoma and its rosettes
  2. 1971Spontaneous regression of stage IV-S (now MS) disease recognised (Evans staging)
    INRG staging and risk groups
  3. 1983MYCN amplification linked to aggressive disease (Brodeur, Schwab)
    MYCN amplification
  4. 1985131I-MIBG therapy first used in relapsed neuroblastoma
    MIBG imaging and 131I-MIBG therapy
  5. 1999CCG-3891: myeloablative therapy with autologous rescue and isotretinoin improve survival
    Autologous stem cell transplant (high-dose therapy)
  6. 2008ALK mutations identified as a hereditary and somatic driver
    ALK
  7. 2009INRG classification unifies international risk grouping
    INRG staging and risk groups
  8. 2010Anti-GD2 (ch14.18) improves EFS
  9. 2010ANBL0032: anti-GD2 immunotherapy raises survival ~20 points (NEJM)
    COG ANBL0032Dinutuximab (ch14.18) / dinutuximab beta
  10. 2015Dinutuximab approved (US); dinutuximab beta in EU 2017
    Dinutuximab (ch14.18) / dinutuximab beta
  11. 2017HR-NBL1: busulfan-melphalan beats CEM; IL-2 adds no benefit (2018)
    SIOPEN HR-NBL1
  12. 2019ANBL0532: tandem transplant improves EFS (JAMA)
    COG ANBL0532Tandem autologous transplant
  13. 2020Naxitamab approved for relapsed disease (accelerated)
    NaxitamabNaxitamab Study 201
  14. 2023GD2 CAR-T phase 1/2 in NEJM
  15. 2023GD2-CART01 in NEJM: durable CAR-T remissions in a solid tumour; eflornithine approved (13 Dec)
    GD2-CART01 (Bambino Gesù phase 1/2)Eflornithine (DFMO)NMTRC003/003B (DFMO maintenance)
  16. 2025Long-term GD2 CAR-T follow-up (Nature Medicine); naxitamab primary-refractory phase 2 (75% CR); eflornithine EU filing
    GD2-CART01 (Bambino Gesù phase 1/2)NaxitamabEflornithine (DFMO)
  17. 2026ANBL1531 MIBG randomisation maturing; ALK arm reports
    COG ANBL1531

Pipeline

13top
EstablishedRadioligand therapy (beta, norepinephrine transporter)
131I-MIBG (iobenguane I-131) therapy
ApprovedSmall-molecule kinase inhibitor (ALK/ROS1)
Lorlatinib · Lorbrena
ApprovedSmall-molecule ornithine decarboxylase inhibitor
Eflornithine (DFMO) · Iwilfin
ApprovedMonoclonal antibody (anti-GD2, humanised)
Naxitamab · Danyelza
Armoured, logic-gated & next-gen CARsCOG ANBL1531GD2-CART01 (Bambino Gesù phase 1/2)GD2 CAR-T as consolidation in high-risk neuroblastoma18F-MFBG PET replacing 123I-MIBG scintigraphyMIBG imaging and 131I-MIBG therapyAnti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy)Tandem autologous transplantY-mAbs Therapeutics

Open problems

  • Relapsed high-risk disease.
  • Long-term toxicity of intensive therapy.
  • Relapsed high-risk neuroblastoma is rarely curable; MYCN-amplified relapse worst of all.
  • 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

top

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Neuroblastoma (paediatric)
condition: neuroblastoma
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Landmark trials in OnCo

COG ANBL0032COG ANBL0532COG ANBL1531GD2-CART01 (Bambino Gesù phase 1/2)Naxitamab Study 201NMTRC003/003B (DFMO maintenance)SIOPEN HR-NBL1

Expert centres

top
Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

top
Bring to your appointment

Questions to ask your oncologist about Neuroblastoma

Generated from this cancer's standard of care, biomarkers, and pipeline · 27 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. Which biomarkers have been tested on my tumour (for example MYCN amplification, ALK mutation, Age, stage, ploidy, Segmental chromosome aberrations, INRG stage and image-defined risk factors), and what were the results?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include Very-low and low risk, Intermediate risk, High risk.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

High-risk

  1. For my situation (high-risk), which of the standard options do you recommend and why?
    Why: Guideline options include: Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant.
  2. Am I a candidate for Lorlatinib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Very-low / low risk (L1, MS)

  1. For my situation (very-low / low risk (l1, ms)), which of the standard options do you recommend and why?
    Why: Guideline options include: Observation with serial imaging for asymptomatic L1/MS (many regress); surgery alone for resectable L1; short chemotherapy only for symptoms or progression.

Intermediate risk

  1. For my situation (intermediate risk), which of the standard options do you recommend and why?
    Why: Guideline options include: 2-8 cycles of moderate chemotherapy (carboplatin, etoposide, cyclophosphamide, doxorubicin) guided by response and biology; surgery; isotretinoin in some protocols.
  2. Am I a candidate for Doxorubicin, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

High risk: induction

  1. For my situation (high risk: induction), which of the standard options do you recommend and why?
    Why: Guideline options include: 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.
  2. Am I a candidate for 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, Dinutuximab (ch14.18) / dinutuximab beta or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of COG ANBL1531 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

High risk: local control

  1. For my situation (high risk: local control), which of the standard options do you recommend and why?
    Why: Guideline options include: 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.

High risk: consolidation

  1. For my situation (high risk: consolidation), which of the standard options do you recommend and why?
    Why: Guideline options include: Tandem autologous transplant (thiotepa-cyclophosphamide, then CEM) in North America (ANBL0532); single busulfan-melphalan transplant in Europe (HR-NBL1).
  2. How do the results of COG ANBL0532 and SIOPEN HR-NBL1 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

High risk: post-consolidation

  1. For my situation (high risk: post-consolidation), which of the standard options do you recommend and why?
    Why: Guideline options include: Anti-GD2 antibody (dinutuximab + GM-CSF + isotretinoin; dinutuximab beta in Europe, no IL-2) × 5-6 cycles; then eflornithine maintenance 2 years (US, 2023).
  2. Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Eflornithine (DFMO), and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of COG ANBL0032 and NMTRC003/003B (DFMO maintenance) apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Relapsed / refractory

  1. For my situation (relapsed / refractory), which of the standard options do you recommend and why?
    Why: Guideline options include: 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.
  2. Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Naxitamab, 131I-MIBG (iobenguane I-131) therapy or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of Naxitamab Study 201 and GD2-CART01 (Bambino Gesù phase 1/2) apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Survivorship

  1. For my situation (survivorship), which of the standard options do you recommend and why?
    Why: Guideline options include: Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic.

Any stage

  1. Are there clinical trials I could join, for example of Armoured, logic-gated & next-gen CARs, COG ANBL1531, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. Would a second opinion at a high-volume centre change anything, and can you help arrange it?
    Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
  3. What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
    Why: Supportive care improves quality of life and helps patients complete treatment.
  4. I read that “Relapsed high-risk disease”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Long-term toxicity of intensive therapy”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.

Everything relevant

106top

Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

18
Allogeneic stem cell transplantationArmoured, logic-gated & next-gen CARsAutologous stem cell transplant (high-dose therapy)CAR-T cell therapyCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Cardio-oncologyCytotoxic chemotherapyIMRT / IGRT (modern external beam)MIBG imaging and 131I-MIBG therapyMonoclonal antibodiesPlatinum agentsProton therapyRadioligand therapy (beta emitters)Small-molecule kinase inhibitorsSurvivorship care and late-effects surveillanceTandem autologous transplantTopoisomerase-I inhibitors (and ADC payloads)Ultrasound

targets

2
ALKGD2 (disialoganglioside)

drugs

10
EstablishedRadioligand therapy (beta, norepinephrine transporter)
131I-MIBG (iobenguane I-131) therapy
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
ApprovedMonoclonal antibody (anti-GD2, chimeric)
Dinutuximab (ch14.18) / dinutuximab beta · Unituxin / Qarziba (EU)
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
ApprovedSmall-molecule ornithine decarboxylase inhibitor
Eflornithine (DFMO) · Iwilfin
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
ApprovedSmall-molecule kinase inhibitor (ALK/ROS1)
Lorlatinib · Lorbrena
ApprovedMonoclonal antibody (anti-GD2, humanised)
Naxitamab · Danyelza
Not mapped hereSmall-molecule chemoprotectant
Sodium thiosulfate (otoprotectant) · Pedmark
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo

companies

4
IpsenPfizer (incl. Seagen)US WorldMedsY-mAbs Therapeutics

institutions

25
Centre Oscar LambretChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Hospital of PhiladelphiaChildren's Oncology Group (COG)City of HopeDan L Duncan Comprehensive Cancer Center, Baylor College of MedicineDana-Farber Brigham Cancer CenterGhent University Hospital / Cancer Research Institute GhentGreat Ormond Street Hospital for ChildrenHacettepe University Cancer InstituteIstanbul University Institute of OncologyMemorial Sloan Kettering Cancer CenterNewcastle Cancer Centre / Northern Centre for Cancer CarePrincess Máxima Center for Pediatric OncologySahlgrenska University Hospital / Sahlgrenska Center for Cancer ResearchSIOP Europe – European Society for Paediatric OncologySt. Jude Children's Research HospitalStanford Health Care / Stanford Cancer InstituteSydney Children's Hospitals Network / Children's Cancer InstituteTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)UC San Diego Moores Cancer CenterUniversity of Chicago Medicine Comprehensive Cancer CenterUniversity of Wisconsin Carbone Cancer Center

pathways

1
MYC

terms

6
ADCC (antibody-dependent cellular cytotoxicity)Autologous stem cell transplant (ASCT)Cytokine release syndrome (CRS)Event-free / disease-free survival (EFS, DFS, iDFS, RFS)INRG staging and risk groupsMYCN amplification

trials

7
COG ANBL0032COG ANBL0532COG ANBL1531GD2-CART01 (Bambino Gesù phase 1/2)Naxitamab Study 201NMTRC003/003B (DFMO maintenance)SIOPEN HR-NBL1

pairings

2
Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy)Caution: IL-2 added to anti-GD2 therapy

ideas

17
18F-MFBG PET replacing 123I-MIBG scintigraphyA non-profit pharmaceutical company for the cancers markets ignoreA paediatric palliative care team in every childhood cancer unitAdvance market commitments for paediatric and rare cancer drugsAn international consortium pooling the outcome of every treated child with cancerAn open organoid bank for cancers too rare to have modelsCan MYC be drugged directly, and will patients tolerate it?Develop drugs in children first when the target is a children's targetEscrow a share of adult revenue until the paediatric study is doneGD2 CAR-T as consolidation in high-risk neuroblastomaGuaranteed-quality childhood cancer medicines free of charge in 50 countriesMake paediatric combination studies part of every relevant adult cancer drug approvalMilestone-based venture philanthropy with royalties recycled into the pipelineOne global paediatric cancer development plan instead of separate FDA and EMA plansOpen-source drug discovery to clinical proof of concept for neglected cancersPay a prize for rare cancer drugs instead of hoping for a marketSwitch off an undruggable oncogene permanently with epigenetic editing

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation

people

9
Alexandra "Alex" ScottAlice L. YuAngelika EggertBrigitte C. WidemannCrystal L. MackallJulie R. ParkKatherine K. MatthaySusan L. CohnTrevor J. Pugh

bottlenecks

3
No incentive to repurpose cheap drugsRare and paediatric cancers without marketsSurvivorship and late effects are neglected

Latest papers

top
Literature trend6,022 papers in the last 12 months+10% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (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.

Connected

99top

Pages like this

not linked directly; found by shared links

technologies

13
Armoured, logic-gated & next-gen CARsAutologous stem cell transplant (high-dose therapy)CAR-T cell therapyCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Cardio-oncologyCytotoxic chemotherapyIMRT / IGRT (modern external beam)MIBG imaging and 131I-MIBG therapyMonoclonal antibodiesProton therapyRadioligand therapy (beta emitters)Tandem autologous transplantUltrasound

targets

2
ALKGD2 (disialoganglioside)

drugs

10
EstablishedRadioligand therapy (beta, norepinephrine transporter)
131I-MIBG (iobenguane I-131) therapy
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
ApprovedMonoclonal antibody (anti-GD2, chimeric)
Dinutuximab (ch14.18) / dinutuximab beta · Unituxin / Qarziba (EU)
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
ApprovedSmall-molecule ornithine decarboxylase inhibitor
Eflornithine (DFMO) · Iwilfin
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
ApprovedSmall-molecule kinase inhibitor (ALK/ROS1)
Lorlatinib · Lorbrena
ApprovedMonoclonal antibody (anti-GD2, humanised)
Naxitamab · Danyelza
Not mapped hereSmall-molecule chemoprotectant
Sodium thiosulfate (otoprotectant) · Pedmark
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo

companies

2
US WorldMedsY-mAbs Therapeutics

institutions

25
Centre Oscar LambretChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Hospital of PhiladelphiaChildren's Oncology Group (COG)City of HopeDan L Duncan Comprehensive Cancer Center, Baylor College of MedicineDana-Farber Brigham Cancer CenterGhent University Hospital / Cancer Research Institute GhentGreat Ormond Street Hospital for ChildrenHacettepe University Cancer InstituteIstanbul University Institute of OncologyMemorial Sloan Kettering Cancer CenterNewcastle Cancer Centre / Northern Centre for Cancer CarePrincess Máxima Center for Pediatric OncologySahlgrenska University Hospital / Sahlgrenska Center for Cancer ResearchSIOP Europe – European Society for Paediatric OncologySt. Jude Children's Research HospitalStanford Health Care / Stanford Cancer InstituteSydney Children's Hospitals Network / Children's Cancer InstituteTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)UC San Diego Moores Cancer CenterUniversity of Chicago Medicine Comprehensive Cancer CenterUniversity of Wisconsin Carbone Cancer Center

pathways

1
MYC

terms

6
ADCC (antibody-dependent cellular cytotoxicity)Autologous stem cell transplant (ASCT)Cytokine release syndrome (CRS)Event-free / disease-free survival (EFS, DFS, iDFS, RFS)INRG staging and risk groupsMYCN amplification

trials

7
COG ANBL0032COG ANBL0532COG ANBL1531GD2-CART01 (Bambino Gesù phase 1/2)Naxitamab Study 201NMTRC003/003B (DFMO maintenance)SIOPEN HR-NBL1

pairings

2
Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy)Caution: IL-2 added to anti-GD2 therapy

ideas

17
18F-MFBG PET replacing 123I-MIBG scintigraphyA non-profit pharmaceutical company for the cancers markets ignoreA paediatric palliative care team in every childhood cancer unitAdvance market commitments for paediatric and rare cancer drugsAn international consortium pooling the outcome of every treated child with cancerAn open organoid bank for cancers too rare to have modelsCan MYC be drugged directly, and will patients tolerate it?Develop drugs in children first when the target is a children's targetEscrow a share of adult revenue until the paediatric study is doneGD2 CAR-T as consolidation in high-risk neuroblastomaGuaranteed-quality childhood cancer medicines free of charge in 50 countriesMake paediatric combination studies part of every relevant adult cancer drug approvalMilestone-based venture philanthropy with royalties recycled into the pipelineOne global paediatric cancer development plan instead of separate FDA and EMA plansOpen-source drug discovery to clinical proof of concept for neglected cancersPay a prize for rare cancer drugs instead of hoping for a marketSwitch off an undruggable oncogene permanently with epigenetic editing

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation

people

9
Alexandra "Alex" ScottAlice L. YuAngelika EggertBrigitte C. WidemannCrystal L. MackallJulie R. ParkKatherine K. MatthaySusan L. CohnTrevor J. Pugh

bottlenecks

3
No incentive to repurpose cheap drugsRare and paediatric cancers without marketsSurvivorship and late effects are neglected