OncoSignal

Neuroblastoma

High-risk and relapsed disease: MYCN amplification, ALK mutations and anti-GD2 therapy.

Live from Open Targets Platform, fetched 2026-11-11 15:26 UTC. Every row links to its source record.
Targets linked to neuroblastoma
6,856
the top 50 by overall evidence are shown
With inherited (germline) evidence
11
of the top 50, score above 0.1
Strong genetics, few drugs
18
genetic evidence 0.5 or more, drug evidence under 0.5
18 of the top 50 neuroblastoma targets have strong human genetic evidence but little drug evidence so far

Each dot is one target. Higher means stronger genetic evidence in people (inherited variants or tumour mutations, whichever is stronger). Further right means more evidence from drugs already in trials or approved.

Has inherited (germline) evidence above 0.1Tumour (somatic) or other evidence only
000.250.250.50.50.750.7511Drug evidence (trials and approvals) →Human genetic evidence →Genetics strong, few drugs: open betsGenetics strong, drugs in the clinicDrugs in the clinic, weaker geneticsALK: genetic evidence 0.97 (germline 0.97, somatic 0.81), drug evidence 0.48PHOX2B: genetic evidence 0.30 (germline 0.00, somatic 0.30), drug evidence 0.00BARD1: genetic evidence 0.86 (germline 0.86, somatic 0.53), drug evidence 0.00LMO1: genetic evidence 0.74 (germline 0.74, somatic 0.00), drug evidence 0.00ATRX: genetic evidence 0.60 (germline 0.00, somatic 0.60), drug evidence 0.00SLC6A2: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.76EWSR1: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00KIF1B: genetic evidence 0.56 (germline 0.56, somatic 0.00), drug evidence 0.00RARA: genetic evidence 0.30 (germline 0.00, somatic 0.30), drug evidence 0.66CSF3R: genetic evidence 0.30 (germline 0.00, somatic 0.30), drug evidence 0.66FLI1: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00PTPN11: genetic evidence 0.55 (germline 0.00, somatic 0.55), drug evidence 0.00TOP2B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TOP2A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.69TUBB2B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TOP1: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.67TUBB3: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB4A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB2A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA4A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB1: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB4B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB6: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB8: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA3E: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA3C: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1C: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68NF1: genetic evidence 0.49 (germline 0.00, somatic 0.49), drug evidence 0.00RARG: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.66RARB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.66SMARCA4: genetic evidence 0.53 (germline 0.44, somatic 0.53), drug evidence 0.00TP53: genetic evidence 0.48 (germline 0.27, somatic 0.48), drug evidence 0.11IDH2: genetic evidence 0.61 (germline 0.00, somatic 0.61), drug evidence 0.12CSF2RB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.63CSF2RA: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.63PIK3CA: genetic evidence 0.57 (germline 0.00, somatic 0.57), drug evidence 0.08FBN2: genetic evidence 0.61 (germline 0.00, somatic 0.61), drug evidence 0.00IL2RG: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.61FEV: genetic evidence 0.61 (germline 0.00, somatic 0.61), drug evidence 0.00IL2RB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.61IL2RA: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.61BRCA2: genetic evidence 0.46 (germline 0.19, somatic 0.46), drug evidence 0.00IL15RA: genetic evidence 0.59 (germline 0.59, somatic 0.00), drug evidence 0.06SALL4: genetic evidence 0.58 (germline 0.58, somatic 0.00), drug evidence 0.00RPTN: genetic evidence 0.58 (germline 0.58, somatic 0.00), drug evidence 0.00ARHGEF40: genetic evidence 0.58 (germline 0.58, somatic 0.00), drug evidence 0.00APC: genetic evidence 0.57 (germline 0.00, somatic 0.57), drug evidence 0.00ALKPHOX2BBARD1LMO1RARACSF3RNF1SMARCA4TP53IDH2IL2RGFEV

Scores run from 0 to 1 and are Open Targets' summary of how much evidence exists, not the chance a drug will work. The dividing lines sit at 0.5. Hover a dot for its numbers, click it for the target's page. Source: Open Targets Platform, fetched live.

Where the genetics points and the pipeline has not gone yet

The 18 targets in the top-left of the chart, strongest genetic evidence first. Click one for its drugs, tractability and other cancers.

All 50 targets, by type of evidence

Ranked by overall score for neuroblastoma on the Open Targets Platform. Darker cells mean stronger evidence of that type.

Peds RMTLmarks the 6 of these targets on the FDA's Pediatric Relevant Molecular Target List: a drug against one of them can be required to be studied in children under the RACE for Children Act.

TargetOverallGermline geneticsSomaticClinicalgenetic literaturePathwaysLiteratureAnimal models
ALKPeds RMTL
ALK receptor tyrosine kinase
0.840.970.81
PHOX2B
paired like homeobox 2B
0.63–0.30
BARD1
BRCA1 associated RING domain 1
0.610.860.53
LMO1
LIM domain only 1
0.480.74–
ATRXPeds RMTL
ATRX chromatin remodeler
0.47–0.60
SLC6A2
solute carrier family 6 member 2
0.47––
EWSR1
EWS RNA binding protein 1
0.46–0.72
KIF1B
kinesin family member 1B
0.460.56–
RARA
retinoic acid receptor alpha
0.46–0.30
CSF3R
colony stimulating factor 3 receptor
0.45–0.30
FLI1
Fli-1 proto-oncogene, ETS transcription factor
0.45–0.72
PTPN11Peds RMTL
protein tyrosine phosphatase non-receptor type 11
0.44–0.55
TOP2B
DNA topoisomerase II beta
0.43––
TOP2A
DNA topoisomerase II alpha
0.42––
TUBB2B
tubulin beta 2B class IIb
0.42––
TUBB
tubulin beta class I
0.42––
TUBA1A
tubulin alpha 1a
0.42––
TUBA1B
tubulin alpha 1b
0.42––
TOP1
DNA topoisomerase I
0.42––
TUBB3
tubulin beta 3 class III
0.41––
TUBB4A
tubulin beta 4A class IVa
0.41––
TUBB2A
tubulin beta 2A class IIa
0.41––
TUBA4A
tubulin alpha 4a
0.41––
TUBB1
tubulin beta 1 class VI
0.41––
TUBB4B
tubulin beta 4B class IVb
0.41––
TUBB6
tubulin beta 6 class V
0.41––
TUBB8
tubulin beta 8 class VIII
0.41––
TUBA3E
tubulin alpha 3e
0.41––
TUBA3C
tubulin alpha 3c
0.41––
TUBA1C
tubulin alpha 1c
0.41––
NF1
neurofibromin 1
0.41–0.49
RARG
retinoic acid receptor gamma
0.41––
RARB
retinoic acid receptor beta
0.41––
SMARCA4
SWI/SNF related BAF chromatin remodeling complex subunit ATPase 4
0.400.440.53
TP53Peds RMTL
tumor protein p53
0.390.270.48
IDH2Peds RMTL
isocitrate dehydrogenase (NADP(+)) 2
0.39–0.61
CSF2RB
colony stimulating factor 2 receptor subunit beta
0.39––
CSF2RA
colony stimulating factor 2 receptor subunit alpha
0.38––
PIK3CAPeds RMTL
phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha
0.38–0.57
FBN2
fibrillin 2
0.38–0.61
IL2RG
interleukin 2 receptor subunit gamma
0.37––
FEV
FEV transcription factor, ETS family member
0.37–0.61
IL2RB
interleukin 2 receptor subunit beta
0.37––
IL2RA
interleukin 2 receptor subunit alpha
0.37––
BRCA2
BRCA2 DNA repair associated
0.370.190.46
IL15RA
interleukin 15 receptor subunit alpha
0.370.59–
SALL4
spalt like transcription factor 4
0.360.58–
RPTN
repetin
0.350.58–
ARHGEF40
Rho guanine nucleotide exchange factor 40
0.350.58–
APC
APC regulator of Wnt signaling pathway
0.35–0.57

4 biomarkers in Neuroblastoma have validated or clinical evidence in CIViC

Accepted evidence from CIViC, the open (CC0) expert-curated knowledgebase, searched by the disease name “Neuroblastoma”. Level A is validated (for example in guidelines or an approval), level B comes from clinical trials or studies. 28 accepted items exist; the first 28 were read. This is the published evidence, not treatment advice.