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🗺️ Disease Atlas · Nervous system disorder

neuroblastoma

Neuroblastoma (NB) is the most common solid, extracranial childhood tumor. It is an aggressive pediatric cancer that originates from neural crest tissues of the sympathetic nervous system.

🔔 Follow weekly
🧬6856genes / proteins with evidence
💊173drugs and clinical candidates
🧪12+recruiting trials worldwide
✅5approved
📈27phase 3
🧬

Understand neuroblastoma

Cancer · Cells that grow out of control
🎓 Learn more

What it is

Neuroblastoma is a type of cancer that forms in nerve cells called neuroblasts. Neuroblasts are immature nerve tissue. They normally turn into working nerve cells. But in neuroblastoma, they form a tumor.

Neuroblastoma usually begins in the adrenal glands. You have two adrenal glands, one on top of each kidney. The adrenal glands make important hormones that help control heart rate, blood pressure, blood glucose (blood sugar), and the way the body reacts to stress. Neuroblastoma may also begin in the neck, chest or spinal cord.

What causes neuroblastoma?

From MedlinePlus: Neuroblastoma (US National Library of Medicine) ↗

🧬 Cancer: Cancer is a group of diseases in which some of the body's cells grow and divide without control, and can spread to other parts of the body.

❓ Why it happens

  • Changes (mutations) build up in the genes that tell a cell when to grow, divide and die.
  • Most of these changes happen during life: tobacco, too much sun, alcohol, some infections (such as HPV and hepatitis B and C), radiation, obesity and ageing all raise the risk.
  • Some people inherit a faulty gene that raises their risk, such as BRCA1 or BRCA2.

🫀 What it does to the body

  • Cancer cells form a lump (tumour) that can press on or invade nearby organs.
  • They can travel through the blood or lymph to start new tumours elsewhere (metastasis).
  • Blood cancers crowd out healthy blood cells in the bone marrow.

🧱 Why it is hard to cure

  • Every cancer is different, even between two people with the same type - and inside one tumour.
  • Cancer cells keep changing, so they can learn to resist a medicine that worked at first.
  • Cancer cells are the body's own cells, so killing them without harming healthy cells is hard.
  • Many cancers cause no symptoms until they have spread, and a new medicine often takes more than ten years to go from the lab to patients.

🚀 What research is trying now

  • Targeted medicines against the exact gene change in a tumour (for example EGFR, ALK or KRAS).
  • Immunotherapy that helps the immune system recognise cancer, including CAR-T cell therapy.
  • Personalised cancer vaccines built from each tumour's mutations, now in late trials.
  • Blood tests that look for tumour DNA to find cancer earlier, and AI to read scans and slides.

General information about cancer, reviewed by people; it does not describe any one person. Below: the evidence for neuroblastoma itself.

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AI briefing

Where the science on neuroblastoma stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens neuroblastoma.

🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
ALK ALK receptor tyrosine kinasePROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.84
PHOX2B paired like homeobox 2B🧊 AlphaFold structure ↗ 0.63
BARD1 BRCA1 associated RING domain 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.61
LMO1 LIM domain only 1🧊 AlphaFold structure ↗ 0.48
ATRX ATRX chromatin remodelerPROTAC🧊 AlphaFold structure ↗ 0.47
SLC6A2 solute carrier family 6 member 2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.47
EWSR1 EWS RNA binding protein 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.46
KIF1B kinesin family member 1BPROTAC🧊 AlphaFold structure ↗ 0.46
RARA retinoic acid receptor alphaPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.46
CSF3R colony stimulating factor 3 receptorPROTACantibodyother🧊 AlphaFold structure ↗ 0.45
FLI1 Fli-1 proto-oncogene, ETS transcription factorPROTACsmall molecule🧊 AlphaFold structure ↗ 0.45
PTPN11 protein tyrosine phosphatase non-receptor type 11PROTACsmall molecule🧊 AlphaFold structure ↗ 0.44
TOP2B DNA topoisomerase II betaPROTACsmall molecule🧊 AlphaFold structure ↗ 0.43
TOP2A DNA topoisomerase II alphaPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.43
TUBB2B tubulin beta 2B class IIbPROTACothersmall molecule🧊 AlphaFold structure ↗ 0.42

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

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Drugs and candidates

Every medicine linked to neuroblastoma, by how far it has got
Approved · 5
DinutuximabApproved
Dinutuximab BetaApproved
Iobenguane I 131Norepinephrine transporter substrateApproved
MelphalanApproved
NaxitamabApproved
In phase 2 / 3 · 20
AldesleukinInterleukin-2 receptor agonistPhase 3
BusulfanPhase 3
CarboplatinPhase 3
CisplatinPhase 3
CyclophosphamidePhase 3
DacarbazinePhase 3
Show all 20
DoxorubicinDNA topoisomerase II alpha inhibitorPhase 3
Doxorubicin HydrochlorideDNA topoisomerase II alpha inhibitorPhase 3
EtoposideDNA topoisomerase II inhibitorPhase 3
Etoposide PhosphateDNA topoisomerase II alpha inhibitorPhase 3
FilgrastimGranulocyte colony stimulating factor receptor agonistPhase 3
Human Immunoglobulin GPhase 3
IfosfamidePhase 3
IobenguaneNorepinephrine transporter substratePhase 3
IrinotecanDNA topoisomerase I inhibitorPhase 3
IsotretinoinRetinoic acid receptor agonistPhase 3
LorlatinibALK tyrosine kinase receptor inhibitor; EML4-ALK inhibitorPhase 3
MesnaPhase 3
PrednisoneGlucocorticoid receptor agonistPhase 3
SargramostimGranulocyte-macrophage colony-stimulating factor receptor agonistPhase 3
Early trials · 20
AbemaciclibCyclin-dependent kinase 4 inhibitor; Cyclin-dependent kinase 6 inhibitorPhase 1/2
AdavosertibSerine/threonine-protein kinase WEE1 inhibitorPhase 1/2
AlrizomadlinTumour suppressor p53/oncoprotein Mdm2 inhibitorPhase 1/2
Amxt-1501 DicapratePhase 1/2
Apg-2575Apoptosis regulator Bcl-2 inhibitorPhase 1/2
Bortezomib26S proteasome inhibitorPhase 1/2
Show all 20
CapecitabineThymidylate synthase inhibitorPhase 1/2
CirmtuzumabInactive tyrosine-protein kinase transmembrane receptor ROR1 inhibitorPhase 1/2
CytarabineDNA polymerase (alpha/delta/epsilon) inhibitorPhase 1/2
DabrafenibSerine/threonine-protein kinase B-raf inhibitorPhase 1/2
EntinostatHistone deacetylase inhibitorPhase 1/2
IberdomideCRL4(CRBN) E3 ubiquitin ligase modulatorPhase 1/2
IdasanutlinTumour suppressor p53/oncoprotein Mdm2 inhibitorPhase 1/2
Interleukin-7Phase 1/2
IpilimumabCytotoxic T-lymphocyte protein 4 inhibitorPhase 1/2
NivolumabProgrammed cell death protein 1 inhibitorPhase 1/2
RepotrectinibNerve growth factor receptor Trk-A inhibitor; Neurotrophic tyrosine kinase receptor type …Phase 1/2
Rivoceranib MesylateVascular endothelial growth factor receptor 2 inhibitorPhase 1/2
VenetoclaxApoptosis regulator Bcl-2 inhibitorPhase 1/2
Ak-01Phase 1
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Recruiting trials

Worldwide, open to new patients now
Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib fo…NCT06607692 · Phase 1, Phase 2 · 📍 Boadilla del MonteAn International Study on Pediatric Patients With Rare Tumors.NCT07072143 · n/a · 📍 PadovaDinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly…NCT06172296 · Phase 3 · 📍 BirminghamPhase I Trial of rhIL-15 Plus Dinutuximab Plus Irinotecan/Temozolomide for Children and Young Adults With Rel…NCT06995872 · Phase 1 · 📍 BethesdaNANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) TrialNCT06450041 · Phase 2 · 📍 Los AngelesA Study of a Vaccine in Combination With β-glucan and GM-CSF in People With NeuroblastomaNCT04936529 · Phase 2 · 📍 New YorkNKG2D.Zeta-NK Cell Conditioning With C7R.GD2.CAR-T Cells for Patients With Relapsed or Refractory Osteosarcom…NCT07211737 · Phase 1 · 📍 HoustonInternational Congenital Central Hypoventilation Syndrome (CCHS) Registry and CCHS SHARENCT03088020 · n/a · 📍 ChicagoTesting the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded …NCT07437963 · Phase 1, Phase 2 · 📍 OaklandImmunotherapy With Dinutuximab Beta in Combination With Chemotherapy for the Treatment of Patients With Prima…NCT05272371 · Phase 1 · 📍 KrakowAPG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid TumorsNCT05701306 · Phase 1, Phase 2 · 📍 GuangzhouNeuroblastoma Biology StudyNCT01587300 · n/a · 📍 Los Angeles Search more trials →
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