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Neuroblastoma

A plain-English summary of the published research on Neuroblastoma, reviewed and approved by our editors — not a hand-curated clinical overview.

Research summary · reviewed
Educational only: This page is not medical advice. Coordinate decisions with your oncology team.

Reviewed Jun 2026 · OncoForge editorial · How we review →

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Evidence at a glanceHuman · observationalMixed results⚠ Studies disagree
9 published studies that name Neuroblastoma1 human studies approved & graded (trial, observational, or meta-analysis)125 human clinical studies in the Neuroblastoma corpus1563 source documents in the Neuroblastoma corpus

last checked June 14, 2026

Why this grade?

Human · observational — Human observational evidence only — no trials.

Computed deterministically from the studies’ types and reported outcomes — not written by AI, and not a claim that anything works.

What the guidelines say

NCI PDQESMONCCNASCO

We link the authoritative guidelines rather than reproduce them. Below, the treatments on this page are split into standard care, guideline or regulatory options, supportive care, and studied but not standard so established care is not mixed with experimental or supportive items.

Guideline / FDA options - context-specific
  • vincristine
  • doxorubicin
Studied, not standard - investigational
  • intensive multimodal therapy
  • chemotherapy
  • radiotherapy
  • 3D conformational irradiation
  • intensity modulation radiation therapy
  • stereotaxic irradiation
  • brachytherapy
  • proton therapy
  • anti-GD2 monoclonal antibody therapy
  • high-dose chemotherapy with autologous stem-cell transplant (ASCT)
  • chemotherapy plus anti-GD2 immunotherapy
  • alkylators
  • cisplatin
  • carboplatin
  • anthracyclines
  • camptothecin
  • MIBG
  • arsenic trioxide
  • anti-GD2 antibodies
  • difluoromethylornithine
  • surgical resection
  • Subadventitial tumour resection
  • Gross tumour resection
  • Genistein

Read the guidelines

Cancer-specific deep links aren’t curated yet — these search the authoritative sources for Neuroblastoma.

Treatment map: Neuroblastoma

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Standard care plus every compound studied in the literature (each cited) and graded by evidence, organized by clinical readiness. A category, not a verdict that anything works — confirm anything here with your oncology team.

26
Interventions
0
Standard of care
16
Tested in people
1
Lab / animal
7
Named in lit.
5
Classes
Standard of care (0) Guideline option (2) Tested in people (16) Lab / animal only (1) Named in the literature (7)

Tested in people, by trial phase: phase not reported ×16

Clinical evidence
Preclinical evidence
Standard of care
Guideline option
Tested in people
Lab / animal only
Named in the literature
Surgery & procedures
—
—
4
—
—
Radiotherapy
—
—
2
—
5
Chemotherapy
—
2
5
—
1
Immunotherapy
—
—
2
—
—
Other
—
—
3
1
1

Columns group into clinical evidence (used in, or tested on, people) and preclinical evidence (lab/animal, or only named in the literature). Cell = number of interventions; a dashed cell means none recorded there.

Established care — detail (2)
Chemotherapy
vincristine
FDA-approved for this cancer.
—Guideline option
doxorubicin
FDA-approved for this cancer.
—Guideline option
Investigational & adjunct compounds — detail (24)
Meta-analysis (16)
alkylatorsanthracyclinesanti-GD2 antibodiesanti-GD2 monoclonal antibody therapyarsenic trioxidecamptothecincarboplatinoff-labelchemotherapy plus anti-GD2 immunotherapy· First-line (advanced disease)cisplatinoff-labeldifluoromethylornithineGross tumour resection· Neoadjuvant (before surgery)high-dose chemotherapy with autologous stem-cell transplant (ASCT)MIBGradiotherapySubadventitial tumour resection· Adjuvant (after surgery)surgical resection
Named in the literature
intensive multimodal therapychemotherapy3D conformational irradiationintensity modulation radiation therapystereotaxic irradiationbrachytherapyproton therapy
Lab / animal only
Genistein

"Tested in people" rows show the highest trial phase found in that compound's cited human studies (Phase I–IV; "phase not reported" = a human study with no phase tag). "Studied" = named in the cited literature for this cancer. "FDA ✓" = FDA-approved for this cancer; "off-label" = an FDA-approved drug used outside its approved indications (per openFDA). Not a claim that anything works.

Reported figures

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Snapshot

The essentials in ~60 seconds — every line is drawn from the cited sources below.

What it is
Neuroblastoma is the most common extracranial solid tumor in children, arising from sympathetic‑nervous‑system precursor cells (most often the adrenal gland or abdominal paravertebral ganglia) and affecting about 10.5/1 million children annually; clinical stage at diagnosis is the single most important prognostic factor. [1][2][3][4][5]
Survival
Outcomes vary widely by risk group; for recently defined high‑risk neuroblastoma, 3‑year event‑free survival was 51.1% (COG ANBL0532) and 44.0% (SIOPEN rapid COJEC), and overall more than half of high‑risk patients die despite intensive multimodal treatment. [6][3][7]
Standard treatment
Treatment for high‑risk disease typically begins with multiagent induction chemotherapy, with surgical resection commonly performed during or after induction and radiotherapy indicated for many patients as part of multimodal care. [6][8]
Key test
Imaging‑based clinical staging using the INRG system (including image‑defined risk factors) together with clinical, histologic, and molecular risk classification is central at diagnosis to guide risk‑adapted treatment; NCCN Guidelines provide diagnostic and risk‑classification recommendations. [5][6][2]
Biggest challenge
The main challenge is the disease's biological heterogeneity — some tumors regress while others are fatal — with clinical stage and high‑risk biology driving poor survival; limited access to care in low‑ and middle‑income countries leads to more advanced stage at presentation and greater need for radiotherapy. [1][3][7][8]

Ask about Neuroblastoma

Answers come only from the cited sources on this page — with the supporting evidence shown. If the sources here don't cover your question, it will say so. Educational information, not medical advice.

Key numbers & factors

Risk factors

  • ▲ increases riskMale sex — Higher male:female ratio reported in Central and South Asia (1.8:1 vs 1.2:1). [9]
  • ▲ increases riskYoung age (under 2 years) — Approximately 60% of cases occur before age 2 and ~97% are diagnosed before age 10. [3]

Biomarkers

  • MYCN amplification · Reported frequency of tumor amplification across induction‑regimen studies [6]
  • Chromosome 1p deletion · Common chromosomal aberration in advanced‑stage disease (reported in about 70% of advanced cases) [3]
  • RASSF7 upregulation · Associated with tumor biology (roles in mitosis, apoptosis, proliferation, differentiation) [10]
  • Vasoactive intestinal peptide (VIP) secretion · Linked to preoperative diarrhoea in reported cases [4]

10 sections — tap any heading to expand its cited detail. Key points are above.

OverviewNeuroblastoma is the most common extracranial solid tumor in children and the most common cancer of infants; it arises from sympathetic nervous system precursor cells and most often occurs in the adrenal gland and abdominal paravertebral ganglia. Disease behavior ranges from spontaneous regression in some patients to progressive, fatal disease in others, and clinical and molecular factors are used at diagnosis to stratify risk.9 points

Key figures

Survival & outcomes
OutcomeValue95% CI
3-year event-free survival (COG ANBL0532)51.1%—
3-year event-free survival (SIOPEN rapid COJEC)44%—
Median end-induction response rate (PR+)84.4%—
Weighted average end-induction response rate (PR+)79.4%—
Source quotes
  • “with a 3‐year event‐free survival (EFS) rate of 51.1% from the time of diagnosis for patients enrolled in the Children's Oncology Group (COG) trial (ANBL0532)”
  • “and a similar 3‐year EFS rate of 44.0% using the rapid COJEC regimen in a recent International Society of Pediatric Oncology European Neuroblastoma (SIOPEN) group HR‐NBL trial”
  • “The median end induction response rate (EIR) of partial response or better (PR+) across 36 regimens was 84.4% (range: 64.3–100)”
  • “with a weighted average by number of participants of 79.4%.”
  • Sources describe neuroblastoma as the most common extracranial solid tumor in children/early childhood. [1][2][3][6]4 sources
  • Patients are classified as having low-, intermediate-, or high-risk disease using clinical, histological, and molecular features. [6]
  • For patients described as having high-risk disease in recent cooperative-group trials, 3-year event-free survival (EFS) rates of 51.1% and 44.0% have been reported in the COG ANBL0532 and a recent SIOPEN rapid COJEC trial, respectively. [6]
  • Therapy for patients with high-risk neuroblastoma typically begins with multiagent induction chemotherapy. [6]
  • The median end‐induction response rate (partial response or better) across 36 induction regimens was 84.4% (range 64.3–100) with a weighted average of 79.4%. [6]
  • Neuroblastoma is a malignant tumour affecting 10.5/1 million children annually and arises from sympathetic nervous system precursor cells, most frequently in the adrenal gland and abdominal paravertebral ganglia. [4]
  • Neuroblastoma can spontaneously regress in some patients yet progress to a fatal outcome in others. [5]
  • Strong prognostic factors can accurately predict whether children have 'good' or 'bad' disease at diagnosis, and clinical stage is currently the most significant and clinically relevant prognostic factor in neuroblastoma. [5]
  • A decision tree model was developed to calculate the optimal radiotherapy utilization rate (oRUR) for childhood neuroblastoma using indications for radiotherapy and corresponding epidemiological data collected through systematic review and meta-analysis. [8]
EpidemiologyNeuroblastoma has highly variable clinical behavior, ranging from spontaneous regression to fatal outcome despite intensive treatment. Most cases are diagnosed in early childhood (approximately 60% before age 2 and about 97% before age 10), and reported incidence in developed countries is about 11–13 per million in children <15 years (65 per million in children <1 year, and 1 per million in children aged 10–14 years).7 points

Key figures

Survival & outcomes
OutcomeValue95% CI
male:female ratio · no clear difference1.8 vs 1.2—
Source quotes
  • “Tumors also showed higher boys' predominance; neuroblastoma (1.8:1 vs 1.2:1, p < 0.05)”
  • Its clinical behavior is highly variable, ranging from spontaneous regression to fatal outcome despite intensive treatment. [1][7]
  • Approximately 60% of neuroblastoma cases occur before age 2 and about 97% are diagnosed before the age of 10 years; in developed countries incidence has been reported as about 11–13 per million in children aged <15 years, 65 per million in children <1 year, and 1 per million in children aged 10–14 years. [3]
  • In Central and South Asia neuroblastoma showed higher boys' predominance (male:female ratio 1.8:1 vs 1.2:1, p < 0.05), and the authors concluded there was a significant boys' predominance in pediatric surgical care. [9]
  • The oRUR for pediatric neuroblastoma was estimated as 64% (95% CI: 58%-71%) in the global setting, 50% in high-income countries, and 68% in low- and middle-income countries. [8]
  • Patients in low- and middle-income countries have more indications for radiotherapy than those in high-income countries, due to a more adverse tumour stage distribution caused by limited access to healthcare resources. [8]
  • Many low-risk patients experience regression after limited or even no chemotherapy. [7]
  • Final analysis of the systematic review consisted of 16 studies including 779 patients. [4]
Key biomarkersPrecise disease characterization at diagnosis is key for risk-adapted treatment. The NCCN Guidelines provide recommendations for the diagnosis, risk classification, and treatment of neuroblastoma.6 points

Key figures

Survival & outcomes
OutcomeValue95% CI
percent_1p_deletion_in_advanced_stage70%—
Source quotes
  • “Chromosomal aberration is frequent in NB. For example, deletions of the short arm of chromosome 1 (1p) occur in about 70% of advanced stage.”
  • Chromosomal aberration is frequent in neuroblastoma; deletions of the short arm of chromosome 1 (1p) occur in about 70% of advanced-stage disease. [3]
  • Precise disease characterization for each patient at diagnosis is key for risk-adapted treatment. [7]
  • The NCCN Guidelines provide recommendations for the diagnosis, risk classification, and treatment of neuroblastoma. [2]
  • Among the included induction-regimen studies, the reported median rate of tumor MYCN amplification was 43% (range 10–100). [6]
Show 2 lab & early-research findings
  • RASSF7 has been reported to play roles in mitosis, microtubule growth, apoptosis, proliferation, and differentiation, and to be upregulated in multiple malignancies, including neuroblastoma. [10]
  • Preoperative diarrhoea in reported cases was strongly linked to elevated VIP secretion. [4]
Standard managementManagement of neuroblastoma is multimodal and commonly involves systemic induction regimens, surgery, and radiotherapy; induction approaches vary internationally and many patients have an indication for radiotherapy. Consensus-based imaging recommendations exist for high‑risk disease, and a Latin American expert panel has recommended anti‑GD2 immunotherapy for newly diagnosed high‑risk patients and for early integration at relapse in salvage treatment.6 points

Key figures

Survival & outcomes
OutcomeValue95% CI
paediatric irradiations per year in France800—
percent with indication for radiotherapy64%—
Source quotes
  • “A third of children with cancer receive radiotherapy as part of their initial treatment, which represents 800 paediatric irradiations per year in France carried out in 15 specialized centres approved on the recommendations of the French national cancer institute in decreasing order of frequency, the types of cancer that require irradiation are: brain tumours, neuroblastomas, Ewing's sarcomas, Hodgkin's lymphomas, soft tissue sarcomas including rhabdomyosarcomas, and nephroblastomas.”
  • “Based on our results, 64&#xa0;% of children with neuroblastoma have an indication for radiotherapy.”
  • About one third of children with cancer receive radiotherapy as part of their initial treatment, representing 800 paediatric irradiations per year in France; neuroblastomas are listed among the types of childhood cancers that require irradiation; paediatric radiotherapy methods include 3D conformational irradiation, intensity modulation radiation therapy, stereotaxic irradiation (normal or hypofractionated), brachytherapy, and proton therapy; and treatment guidelines for paediatric radiotherapy follow recommendations of the French society for childhood cancers (SFCE) or French and European prospective protocols. [11]
  • Based on the study results, 64% of children with neuroblastoma have an indication for radiotherapy. [8]
  • Consensus-based imaging recommendations for high‑risk neuroblastoma were developed to improve reproducibility and diagnostic performance at first presentation and throughout follow-up. [1]
  • A Latin American expert panel strongly recommended anti‑GD2 immunotherapy as maintenance treatment for all newly diagnosed high‑risk neuroblastoma patients to maximize event‑free survival and reduce morbid and costly salvage therapies, and the same panel recommended early integration (at first relapse) of anti‑GD2 immunotherapy in salvage treatment. [12]
  • Distinct induction regimens are used internationally and vary by agents, duration, cycle number, and dose intensity; few randomized controlled trials have compared induction regimens, producing a lack of data regarding comparative efficacy and toxicity; surgical resection was reported to be performed after induction in 22 (61.1%) regimens and during induction in 14 (38.9%) regimens; and only eight (22.2%) of the included trials contained a randomized induction question. [6]
  • The authors state that subadventitial tumour resection should be avoided when undertaking surgery for neuroblastoma to minimize the risk(s) of persistent postoperative diarrhoea. [4]
Treatments & compounds studied26 distinct therapeutics and procedures across modalities — chemotherapy, radiotherapy, immunotherapy, surgical/procedural interventions and other agents — are reported in the cited sources for neuroblastoma.25 treatments

Chemotherapy

  • chemotherapy: Some low-risk patients experience regression after limited or even no chemotherapy. [7]
    cure rate 50%
    Source quote
    • “Bei Hochrisiko-Neuroblastom können dagegen nur ca. 50% der Patienten durch eine intensive multimodale Therapie geheilt werden.”
  • alkylators: Alkylator agents were used in all 36 induction regimens included in the review. [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • cisplatin: Cisplatin was used in 31 of the 36 induction regimens (86.1%). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • carboplatin: Carboplatin was used in 14 of the 36 induction regimens (38.9%). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • vincristine: Vincristine was used in 26 of the 36 induction regimens (72.2%). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • anthracyclines: Anthracyclines (including doxorubicin) were used in 27 of the 36 induction regimens (75%). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • doxorubicin: Doxorubicin planned dose intensity during induction was reported with a median planned dose intensity of 5.0 mg/m2/week (range 2.8–16.7). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • camptothecin: Ten regimens (27.9%) included at least one camptothecin. [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”

Immunotherapy

  • anti-GD2 monoclonal antibody therapy: Anti‑GD2 monoclonal antibody immunotherapy has been incorporated into treatment for high‑risk neuroblastoma; the consensus notes that survival rates for high‑risk neuroblastoma have improved with its incorporation. [12]
  • anti-GD2 antibodies: Anti‑GD2 antibodies were included as a novel agent in a minority of induction regimens (reported among the agents evaluated in 16.7% of regimens). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”

Radiotherapy

  • radiotherapy: Irradiation methods used in paediatric radiotherapy include intensity-modulated radiotherapy, stereotactic irradiation (standard or hypofractionated), brachytherapy and proton therapy. [13][8]
    optimal radiotherapy utilization rate (global) 64% (95% CI 58–71)
    Source quote
    • “The oRUR for pediatric neuroblastoma was 64&#xa0;% (95&#xa0;% CI: 58&#xa0;%-71&#xa0;%) in the global setting, 50&#xa0;% in high-income countries, and 68&#xa0;% in low- and middle-income countries.”
  • 3D conformational irradiation: 3D conformational irradiation is listed among the irradiation methods used in paediatric radiotherapy for cancers that include neuroblastomas. [11]
  • intensity modulation radiation therapy: Intensity modulation radiation therapy (IMRT) is listed among the irradiation techniques available for paediatric cancers including neuroblastomas. [11]
  • stereotaxic irradiation: Stereotaxic irradiation, delivered under normal or hypofractionated conditions, is listed among the irradiation options for paediatric cancers including neuroblastomas. [11]
  • brachytherapy: Brachytherapy is listed as one of the available irradiation methods for paediatric cancers including neuroblastomas. [11]
  • proton therapy: Proton therapy is included among the irradiation techniques available for paediatric cancers including neuroblastomas. [11]
  • MIBG: Only six regimens (16.7%) included a novel agent. [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”

Procedures & devices

  • high-dose chemotherapy with autologous stem-cell transplant (ASCT): High‑dose chemotherapy with autologous stem‑cell transplant (ASCT) remains recommended by the panel until randomized trials omitting ASCT are available. [12]
  • surgical resection: Surgical resection was reported as part of care either after induction (in 22 regimens) or during induction (in 14 regimens). [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • Subadventitial tumour resection: Adjuvant (after surgery)Subadventitial tumour resection was associated with significantly increased postoperative diarrhoea compared to non subadventitial resection. [4]
  • Gross tumour resection: Neoadjuvant (before surgery)In the majority of neuroblastoma patients, preoperative diarrhoea ameliorated after gross tumour resection with elevated VIP normalized. [4]

Other

  • intensive multimodal therapy: Intensive multimodal therapy is used in high-risk neuroblastoma; the source reports an approximate 50% long-term survival among patients treated with such therapy. [7]
    cure rate 50%
    Source quote
    • “Bei Hochrisiko-Neuroblastom können dagegen nur ca. 50% der Patienten durch eine intensive multimodale Therapie geheilt werden.”
  • chemotherapy plus anti-GD2 immunotherapy: First-line (advanced disease)Pilot studies using early anti‑GD2 immunotherapy plus chemotherapy during induction/consolidation in newly diagnosed high‑risk neuroblastoma have shown promising results, but the panel does not recommend routine incorporation until larger confirmatory trials are available. [12]
  • arsenic trioxide: Arsenic trioxide was listed among the novel agents included in a minority (16.7%) of induction regimens. [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
  • difluoromethylornithine: Difluoromethylornithine (DFMO) was listed among novel agents evaluated in a minority of induction regimens. [6]
    Weighted mean end-induction response rate (anthracycline vs no anthracycline) 80.7 vs 73.9%, p = 0.036 vs no anthracycline
    Source quote
    • “Anthracycline‐containing regimens had a statistically significantly higher study‐level mean EIR (anthracycline weighted group mean 80.7%; no anthracycline weighted group mean 73.9%; p = 0.036; Figure 2B).”
PrognosisPrognosis in neuroblastoma varies widely by risk group: reported survival is higher than 95% in low‑risk disease but only around 50% in high‑risk disease, and more than half of high‑risk patients die despite intensive multimodal treatment. Clinical stage is described as the most important prognostic factor, and imaging/staging has been the subject of an INRG consensus report intended to optimize imaging and reduce interobserver variability.5 points

Key figures

Survival & outcomes
OutcomeValue95% CI
survival_rate_low-risk95%—
survival_rate_high-risk50%—
Weighted average complete response (CR) rate22.8%—
Weighted average progressive disease (PD) rate7.4%—
Source quotes
  • “Survival rate varies by risk groups, and is higher than 95% in the low-risk group whereas only around 50% in the high-risk group (5).”
  • “with a weighted average by number of participants of 22.8%.”
  • “with a weighted average by number of participants of 7.4%.”
  • More than half of high‑risk patients die from disease despite intensive multimodal treatment; in high‑risk neuroblastoma only about 50% of patients can be cured. [3][7]
  • The median complete response (CR) rate across 28 regimens was 29.6% with a weighted average CR of 22.8%, and the median progressive disease (PD) rate across 26 regimens was 4.6% with a weighted average PD of 7.4%. [6]
  • Clinical stage remains the most important prognostic factor in neuroblastoma. [5]
  • The INRG Imaging Committee prepared a consensus report intended to optimize imaging and staging and reduce interobserver variability. [5]
  • Five-year survival rates were reported as equivalent between the compared groups. [4]
What we don't know yetImportant gaps remain in neuroblastoma: the etiology is largely unknown with no clearly established modifiable risk factors, the evidence base for many treatment questions is limited, and randomized trials are needed to define the roles of specific therapies (for example ASCT omission and early anti‑GD2 strategies). Groups have issued recommendations (including on imaging) and called for larger, well‑designed trials and better data on radiotherapy utilization to address these uncertainties.6 points
  • A systematic review noted that it is not known how induction regimens’ response rates or toxicities compare. [6][4]
  • Overall the etiology of neuroblastoma remains largely unknown; to date no modifiable risk factor has been clearly established, the authors avoided strong causal claims about reported associations, and they state these associations warrant further mechanistic exploration. [3]
  • A consensus panel noted that randomized trials omitting autologous stem‑cell transplant (ASCT) are needed before ASCT can be omitted from high‑dose chemotherapy regimens. [12]
  • Pilot studies of early anti‑GD2 combined with chemotherapy are promising but require confirmation by larger studies with well‑defined control arms. [12]
  • One study found that variation in radiotherapy indications between major international treatment protocols had negligible impact in sensitivity analyses, but the authors stated that knowledge of the optimal radiotherapy utilization rate is crucial for evaluating current practices, identifying gaps in access, and planning future radiotherapy services for childhood cancer. [8]
  • SIOPEN imaging recommendations aim to improve reproducibility and diagnostic performance in order to advance the current standard of care and assist in the development of future trials. [1]
Staging & riskAccurate staging is critical for assessing risk and selecting appropriate treatment in neuroblastoma. The International Neuroblastoma Risk Group (INRG) Staging System (proposed in 2009) emphasizes imaging, uses image-defined risk factors (IDRFs) to define two localized stages, and complements classification into low‑risk, intermediate‑risk, and high‑risk groups.3 points
  • Proper staging is of paramount importance for risk assessment and selection of optimal treatment in neuroblastoma. [5]
  • The International Neuroblastoma Risk Group (INRG) Project proposed a staging system in 2009 designed for tumor staging before any treatment, including surgery; the INRG Staging System shifted the focus from surgicopathologic findings to imaging, includes two stages of localized disease that depend on whether image-defined risk factors (IDRFs) are present, IDRFs are features detected with imaging at the time of diagnosis, and recommended imaging methods include ultrasonography, magnetic resonance imaging, computed tomography, and scintigraphy. [5]
  • Neuroblastoma is heterogeneous and is classified into different risk strata such as low‑risk, intermediate‑risk, and high‑risk groups. [3]
Biology & pathwaysNeuroblastoma clinical behavior is largely determined by tumor biology, including mechanisms that can suppress the host immune system. Specific molecular factors described in the literature include RASSF7-related signaling pathways and, less commonly, secretion of vasoactive intestinal peptide (VIP) producing clinical symptoms such as diarrhoea.3 points
  • Neuroblastoma clinical behaviors are mostly influenced by biology, including unique abilities to suppress the host immune system. [3]
  • RASSF7 has been described as potentially promoting tumor development by regulating Aurora B, MKK4, MKK7, JNK, YAP, MEK, and ERK, and possibly inhibiting c-Myc. [10]
  • Diarrhoea as a presenting symptom of neuroblastoma is uncommon and usually linked to vasoactive intestinal peptide (VIP) tumour secretion. [4]
Safety & interactionsReported safety findings include study-level incidence data on common hematologic and non-hematologic toxicities from induction regimens, and an increased risk of postoperative diarrhoea after subadventitial neuroblastoma resection compared with classical resection methods.2 points
  • A systematic review collected and reported study-level incidence of common hematologic and non-hematologic toxicities from induction regimens. [6]
  • The operative technique of subadventitial neuroblastoma resection was reported to portend significant risk(s) of postoperative diarrhoea not seen with other classical methods of tumour resection. [4]

Common questions

What is Neuroblastoma?

Neuroblastoma is the most common extracranial solid tumor in children and the most common cancer of infants; it arises from sympathetic nervous system precursor cells and most often occurs in the adrenal gland and abdominal paravertebral ganglia. Disease behavior ranges from spontaneous regression in some patients to progressive, fatal disease in others, and clinical and molecular factors are used at diagnosis to stratify risk.

How common is Neuroblastoma?

Neuroblastoma has highly variable clinical behavior, ranging from spontaneous regression to fatal outcome despite intensive treatment. Most cases are diagnosed in early childhood (approximately 60% before age 2 and about 97% before age 10), and reported incidence in developed countries is about 11–13 per million in children <15 years (65 per million in children <1 year, and 1 per million in children aged 10–14 years).

Which biomarkers are important in Neuroblastoma?

Precise disease characterization at diagnosis is key for risk-adapted treatment. The NCCN Guidelines provide recommendations for the diagnosis, risk classification, and treatment of neuroblastoma.

What is the biology of Neuroblastoma?

Neuroblastoma clinical behavior is largely determined by tumor biology, including mechanisms that can suppress the host immune system. Specific molecular factors described in the literature include RASSF7-related signaling pathways and, less commonly, secretion of vasoactive intestinal peptide (VIP) producing clinical symptoms such as diarrhoea.

How is Neuroblastoma treated?

Management of neuroblastoma is multimodal and commonly involves systemic induction regimens, surgery, and radiotherapy; induction approaches vary internationally and many patients have an indication for radiotherapy. Consensus-based imaging recommendations exist for high‑risk disease, and a Latin American expert panel has recommended anti‑GD2 immunotherapy for newly diagnosed high‑risk patients and for early integration at relapse in salvage treatment.

What treatments are studied for Neuroblastoma?

26 distinct therapeutics and procedures across modalities — chemotherapy, radiotherapy, immunotherapy, surgical/procedural interventions and other agents — are reported in the cited sources for neuroblastoma.

Sources

Every statement above is drawn from these reviewed sources. This page reports what they describe. Sources last checked June 14, 2026.

  1. GuidelineImaging of High-Risk Neuroblastoma: Recommendations From SIOPEN Radiology and Nuclear Medicine Specialty Committees · 2026
  2. GuidelineNeuroblastoma, Version 2.2024, NCCN Clinical Practice Guidelines in Oncology · 2024
  3. Meta-analysisRisk factors of neuroblastoma: a systematic review and meta-analysis · 2025
  4. Systematic reviewPre and postoperative diarrhoea associated with neuroblastoma resection - A systematic review of published studies · 2023
  5. GuidelineGuidelines for imaging and staging of neuroblastic tumors: consensus report from the International Neuroblastoma Risk Group Project · 2011
  6. Systematic reviewInduction Regimens in High-Risk Neuroblastoma: Systematic Review of Response Rates and Toxicities · 2025
  7. GuidelineGPOH Guidelines for Diagnosis and First-line Treatment of Patients with Neuroblastic Tumors, update 2025 · 2025
  8. Meta-analysisEstimation of the optimal radiotherapy utilization rate for childhood neuroblastoma · 2024
  9. Meta-analysisGender equity in pediatric surgical care in Central and South Asia: A systematic review and meta-analysis · 2026
  10. Systematic reviewA Number of the N-terminal RASSF Family: RASSF7 · 2024
  11. GuidelineGuide for paediatric radiotherapy procedures · 2022
  12. Systematic reviewLatin American Consensus on the Use of Anti-GD2 Monoclonal Antibody Therapy in Patients With High-Risk Neuroblastoma · 2025
  13. GuidelineGuide for paediatric radiotherapy procedures: 2025 update · 2025

What supports this page

The kinds of sources behind this page, strongest at the top. Faint rungs show what is not here yet.

Guideline
13
Meta-analysis
52
Systematic review
48
Randomized trial
2
Clinical trial
20
Observational
9
Case report
361
Review
1054
Preclinical
0
Other
4

Living document — last change June 14, 2026: Cancer page updated. 2 recent updates logged.

Medicines & supplements studied for Neuroblastoma

PubMedFDAClinicalTrials.gov

Every drug, supplement, and other agent the published studies cover for Neuroblastoma, ranked by how strong the evidence is — what studies report, not a recommendation. Tap any to see its full profile.

Medicines · 1

GenisteinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

No human studies yet · No numeric effect sizes reported · Based on a single study.
Other1 studyFull profile →

What recent studies report in Neuroblastoma

These are reviewed studies whose abstracts concern Neuroblastoma. Each describes only what that study reported. This is not a claim by OncoForge that any compound helps or harms Neuroblastoma. Most are early lab, animal, or small human studies, and findings often conflict.

9 studies1 human1 animal⚠ Conflicting evidenceMechanism (4)

Tracking 9 published studies of Neuroblastoma: 1 in humans, 1 in animals, 7 reviews/other.

Reported direction across studies: 4 positive, 2 mixed, 3 inconclusive.

Findings conflict — both supportive and negative/mixed results exist (see below). Human evidence is limited.

These counts summarize what the studies reported; they are not a measure of whether anything works for Neuroblastoma.

Compounds with studies mentioning Neuroblastoma

Genistein (1)
ReviewReported positivePreclinical onlyTier 1 · lab

Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

Molecular biology reports · May 2026 · narrative review

Genisteinbreast cancerovarian cancerprostate cancergliomaneuroblastomahepatocellular carcinomalung cancerbladder cancerosteosarcomarhabdomyosarcoma

This is a narrative review of the biological activities and potential therapeutic roles of soy isoflavones (including genistein and daidzein). The authors summarize proposed anticancer mechanisms (estrogen receptor modulation, apoptosis, anti-angiogenesis, epigenetic effects, etc.) and report that in vitro and in vivo studies have shown promising results across a range of tumor types. They conclude that further research—especially studies combining isoflavones with established chemotherapeutics—is needed.

Studied with: chemotherapeutic agents.

Key findings
  • Soy isoflavones (genistein, daidzein) have estrogenic and non-estrogenic activities including anti-inflammatory, antioxidant, and immunomodulatory effects.
  • Proposed anticancer mechanisms include modulation of estrogen receptors, copper ion-dependent induction of cell death, promotion of apoptosis, inhibition of angiogenesis and metastasis, regulation of epigenetic processes, and effects on platelet function.
  • Because of estrogen receptor interactions, isoflavones have been studied particularly in hormone-dependent cancers such as breast, ovarian, and prostate cancer.
  • In vitro and in vivo studies have reported promising results in multiple malignancies (gliomas, neuroblastoma, hepatocellular carcinoma, lung and bladder cancers, osteosarcoma, rhabdomyosarcoma).
  • Authors recommend further investigation, particularly combining isoflavones with established chemotherapeutics, to evaluate potential synergy.
Limitations: This article is a narrative review and does not present new clinical trial data.; The evidence summarized is primarily preclinical (in vitro and in vivo) with no clinical trial data provided in the abstract.; Mechanistic proposals are not proven clinical effects and require further experimental and clinical validation.; Safety and efficacy in patients, optimal dosing, and interactions with standard therapies are not established in this review..

AI summary of the abstract, human-reviewed · Jul 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

Animal studyReported positivePreclinical onlyTier 2 · animal

A humanized anaplastic lymphoma kinase (ALK)-directed antibody-drug conjugate with pyrrolobenzodiazepine payload demonstrates efficacy in ALK-expressing cancers

Nature communications · Aug 2025 · xenograft antitumor assays

neuroblastomarhabdomyosarcomacolorectal carcinomamelanomaovarian carcinomabreast carcinoma

This study tested a humanized antibody-drug conjugate called CDX0239-PBD in ALK-expressing cancer models. In cell lines, it was taken up by ALK-positive neuroblastoma cells and killed them in a way that depended on surface ALK expression. In mouse xenograft models, it produced strong antitumor activity and complete responses were maintained in several ALK-expressing cancers.

Key findings
  • ALK RNA, protein, and tumor cell surface expression was elevated in multiple pediatric and adult malignancies with minimal expression in childhood normal tissues.
  • CDX0239-PBD was internalized in ALK-expressing neuroblastoma cell lines with cell surface expression-dependent cytotoxicity.
  • CDX0239-PBD exhibited potent antitumor efficacy including maintained complete responses in ALK-expressing patient and cell line-derived neuroblastoma, fusion-positive rhabdomyosarcoma, and colorectal carcinoma xenograft models.
Limitations: Preclinical study only; no human treatment data are reported in the abstract.; Efficacy was shown in cell lines and xenograft mouse models, which may not predict clinical benefit.; No quantitative effect sizes, dosing details, or toxicity results are provided in the abstract..

The abstract describes a preclinical anticancer antibody-drug conjugate targeting ALK-expressing tumors.

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

ReviewInconclusiveLimited evidenceTier 3 · early human

Atypical Pelvic Tumors in Children

Cancers · Feb 2025 · literature review and case series (selected cases presented)

sacrococcygeal teratomaovarian teratomarhabdomyosarcomaEwing sarcomacervical cancersmall cell neuroendocrine carcinoma of the ovaryEwing sarcoma/primitive neuroectodermal tumor (ES/PNET) of the ovarydiffuse large B-cell lymphoma of the ovariesovarian Sertoli-Leydig cell tumor (SLCT)neuroblastomaplexiform neurofibromaRosai-Dorfman disease

The authors review selected atypical pelvic tumors seen in children and present their own cases, focusing on imaging (MRI) characteristics. They describe a variety of reproductive-system and nervous-system tumors (including rare ovarian and testicular neoplasms, lymphomas, neuroblastoma, plexiform neurofibroma, and Rosai-Dorfman disease). The study sought radiological features that could help radiologists reach correct diagnoses but emphasizes that MRI cannot be interpreted alone and must be combined with clinical, syndromic and laboratory information.

Key findings
  • Selected atypical pelvic tumors in children are presented, many arising in the reproductive system (examples listed include cervical cancer, ovarian small cell neuroendocrine carcinoma, ES/PNET of the ovary, ovarian DLBCL, and ovarian SLCT associated with DICER1 syndrome).
  • Tumors originating from the nervous system discussed include neuroblastoma and plexiform neurofibroma (both NF1-associated and not associated with NF1).
  • Rosai-Dorfman disease involving pelvic and inguinal lymph nodes is presented as an additional differential diagnosis.
  • The authors aimed to identify radiological (MRI) features to guide radiologists toward correct diagnosis, but state that MR images must be interpreted alongside clinical picture, comorbidities/syndromes, and laboratory results.
Limitations: Study presents selected cases and a literature review rather than a systematic or comprehensive series; potential selection bias.; No sample size, quantitative diagnostic accuracy, or outcome data are reported in the abstract.; Imaging findings are descriptive and the abstract does not report validation of MRI features against definitive diagnoses or standardized criteria..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

ReviewMechanismInconclusiveLimited evidenceTier 3 · early human

Adult-Onset Cancer Predisposition Syndromes in Children and Adolescents-To Test or not to Test?

Clinical cancer research : an official journal of the American Association for Cancer Research · May 2024

childhood canceradolescent cancerhigh-grade gliomaneuroblastomarhabdomyosarcoma

This review summarizes data from germline genetic testing in children and adolescents with cancer and notes that pathogenic variants in adult-onset cancer predisposition genes have been reported in about 1–2% of this population. The authors state that causal links between these variants and pediatric cancers remain uncertain and advise against routine testing of healthy children for such variants before adulthood outside of research studies. They highlight specific examples (mismatch repair genes in glioma, BARD1 in neuroblastoma, BRCA2 in rhabdomyosarcoma) and call for more research on pediatric risks, mechanisms, treatment response, second cancer risk, and psychosocial effects.

Key findings
  • Pathogenic variants (PVs) in adult-onset cancer predisposition genes (aoCPG) are reported in 1% to 2% of children and adolescents with cancer.
  • Causal relationship between PVs in aoCPGs and childhood cancer is still under investigation.
  • Examples cited: heterozygous PVs in mismatch repair genes in mismatch repair deficient high-grade glioma; heterozygous PVs in BARD1 in childhood neuroblastoma; heterozygous PVs in BRCA2 in children with rhabdomyosarcoma.
  • Low penetrance for pediatric cancers is likely due to low baseline childhood cancer risk plus modest relative risk in childhood.
  • Recommendation: do not empirically test healthy children for PVs in aoCPGs before adulthood outside of a research study.
  • Authors call for further research on precise pediatric cancer risks/spectra, underlying cellular mechanisms and somatic mutational signatures, treatment response, second neoplasm risks, and psycho-oncological aspects.
Limitations: This is a review/perspective and does not present new primary cohort data.; Precise pediatric risk estimates and spectra for aoCPG PVs are not established in the abstract.; Causal relationships between aoCPG PVs and childhood cancers remain uncertain.; Recommendations are conservative due to limited evidence and low penetrance in childhood; more research needed..

Addresses prevalence and implications of germline adult-onset cancer predisposition gene variants in pediatric oncology.

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

ReviewMechanismInconclusiveLimited evidenceTier 3 · early human

Rare embryonal and sarcomatous central nervous system tumours: State-of-the art and future directions

European journal of medical genetics · Jan 2023

central nervous system tumoursembryonal tumour with multilayered rosettes (ETMR)CNS neuroblastoma, FOXR2-activated (CNS NB-FOXR2)CNS tumour with BCOR-ITD (CNS BCOR-ITD)primary intracranial sarcoma, DICER1-mutant (CNS DICER1)CIC-rearranged sarcoma (CNS CIC)

This review summarizes how molecular diagnostic methods have led to delineation of novel rare embryonal and sarcomatous central nervous system tumour types now recognized in the WHO 5th edition. Because clinical data are limited and primarily from small retrospective cohorts, the authors provide diagnostic and clinical management guidance and call for comprehensive molecular diagnostics and international collaborative data collection.

Key findings
  • Introduction of molecular methods into CNS tumour diagnostics has revealed significant molecular heterogeneity and led to definition of novel rare tumour types included in the WHO 5th edition.
  • Rare embryonal and sarcomatous CNS tumours have distinct histopathological and molecular features and characteristic clinical properties that require different therapeutic approaches.
  • Current therapeutic recommendations must often be based on data from small, predominantly retrospective patient cohorts due to limited clinical data availability.
  • The article provides guidance for diagnostic work-up and clinical management of specific rare entities: ETMR, CNS NB-FOXR2, CNS BCOR-ITD, CNS DICER1, and CNS CIC.
  • The authors emphasize broad implementation of comprehensive molecular diagnostics and the need for joint international efforts to collect and study these rare tumour types.
Limitations: Narrative review with no new primary patient-level data reported in the abstract.; Clinical recommendations are based on limited clinical data and small, predominantly retrospective cohorts.; Rarity and novelty of tumour types limit available evidence and create diagnostic and therapeutic uncertainty.; No prospective or randomized clinical trial data cited in the abstract to support management recommendations..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Human · observationalMechanismMixed resultsLimited evidenceTier 3 · early humann = 5800

PRAME Expression in Cancer. A Systematic Immunohistochemical Study of >5800 Epithelial and Nonepithelial Tumors

The American journal of surgical pathology · Nov 2022 · systematic immunohistochemical survey of normal tissues and human tumors using EP461 monoclonal antibody

endometrial carcinomauterine serous carcinomauterine carcinosarcomaovarian clear cell carcinomaovarian serous carcinomaadenoid cystic carcinomaseminomathymic carcinomabasal cell carcinomasynovial sarcomamyxoid liposarcomaneuroblastomametastatic melanomapoorly differentiated carcinomassarcomasmelanoma (marker-negative subset)

This study used immunohistochemistry with the EP461 monoclonal antibody to evaluate PRAME protein expression in normal tissues and over 5,800 human tumors. PRAME was detected in normal testis and proliferative endometrium and showed variable expression across many tumor types, with high positivity rates in several epithelial and mesenchymal malignancies. The authors conclude PRAME is relatively nonspecific as an immunohistochemical marker, which limits its diagnostic utility, but that IHC can detect PRAME-positive tumors for potential immunotherapy approaches.

Reported effects: PRAME positivity in endometrial carcinomas 82% · PRAME positivity in uterine serous carcinomas 82% · +12 more

Key findings
  • In normal tissues, PRAME was expressed in the testis and proliferative endometrium.
  • Among epithelial tumors, high PRAME positivity rates included endometrial carcinomas (82%), uterine serous carcinomas (82%), uterine carcinosarcomas (60%), ovarian clear cell carcinomas (90%), ovarian serous carcinomas (63%), and adenoid cystic carcinomas (81%), as well as seminomas (78%), thymic carcinomas (75%), and basal cell carcinomas (62%).
  • In mesenchymal and neuroectodermal malignancies, PRAME was frequently expressed in synovial sarcoma (71%), myxoid liposarcoma (76%), neuroblastoma (61%) and metastatic melanoma (87%).
  • PRAME was consistently expressed in 4 melanomas that lacked all melanoma markers including S100 protein and SOX10 but harbored typical for melanoma BRAF or NRAS driver mutations.
  • Strong and diffuse PRAME immunoreactivity was seen in many types of nonmelanocytic poorly differentiated carcinomas and sarcomas.
  • The authors state PRAME is a relatively unspecific immunohistochemical marker, limiting its diagnostic use, but that immunohistochemistry is a reliable and inexpensive method useful to detect PRAME-positive malignancies for potential immunotherapy.
Limitations: Observational immunohistochemical survey without clinical outcome or functional correlation reported in the abstract.; Use of a single monoclonal antibody (EP461) — potential antibody-specific effects or cross-reactivity not explored in the abstract.; Abstract reports percentages but does not provide denominators or detailed methods in the abstract.; PRAME shown to be relatively unspecific, limiting diagnostic specificity..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

ReviewReported positiveModerate evidenceTier 4 · clinical

Retinoblastoma and Neuroblastoma Predisposition and Surveillance

Clinical cancer research : an official journal of the American Association for Cancer Research · Jul 2017 · consensus recommendations from the AACR Childhood Cancer Predisposition Workshop

Supportive careretinoblastomaneuroblastomapineoblastoma

This AACR workshop review summarizes recommendations for genetic predisposition and surveillance in hereditary retinoblastoma and neuroblastoma. It recommends intensive ocular screening and neuroimaging for pineoblastoma surveillance in children with germline RB1 mutations, notes a substantial lifetime risk of second cancers after hereditary RB (higher with prior radiotherapy), and states that no established surveillance protocols currently exist for neuroblastoma despite known familial predisposition genes (ALK, PHOX2B).

Reported effects: percent_hereditary_retinoblastoma 40% · risk_second_primary_cancers_hereditary_RB 20% · +2 more

Key findings
  • Approximately 40% of retinoblastomas are hereditary due to germline RB1 mutations.
  • Recommend intensive ocular screening for patients with germline RB1 mutations and neuroimaging for pineoblastoma surveillance.
  • There is an approximately 20% risk of developing second primary cancers among individuals with hereditary RB, higher among those who received radiotherapy for their primary RB tumors.
  • Neuroblastoma accounts for 15% of pediatric cancer deaths; about 2% of NB patients have an underlying genetic predisposition.
  • Germline mutations in ALK and PHOX2B account for most familial neuroblastoma cases, but no established surveillance protocols for NB currently exist.
Limitations: Review/consensus document rather than new primary data or prospective study.; Recommendations based on expert consensus and prior studies; optimal screening protocols (especially for second primaries and for NB) are not clearly established.; No details on specific screening intervals, ages, or standardized protocols for many recommendations provided in the abstract..

Provides consensus guidance on surveillance and genetic predisposition for hereditary retinoblastoma and neuroblastoma.

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

ReviewMechanismReported positiveLimited evidenceTier 4 · clinical

The role of CT10 regulation of kinase-like in cancer

Future oncology (London, England) · Dec 2014 · Review

gastric cancerglioblastoma multiformehepatocellular carcinomabladder cancerlung cancercolon cancerovarian cancerleukemiabreast cancerhead and neck cancerrhabdomyosarcomaneuroblastoma

This is a narrative review summarizing published reports about the adaptor protein CRKL in cancer. The authors report that CRKL is overexpressed in many tumor types and appears to promote aggressive or malignant behaviors, and they suggest CRKL has potential as a diagnostic/prognostic biomarker.

Key findings
  • CRKL is a member of the CRK family and functions as an adaptor protein in intracellular signal transduction.
  • CRKL has been reported overexpressed in a variety of cancers.
  • CRKL appears to play a tumor-promotion role in multiple cancers, including those listed in the abstract.
  • The review summarizes associations between CRKL and malignant tumor behaviors and potential mechanisms of action.
  • The authors state CRKL has potential to be used as a biomarker for diagnosis, treatment and prognosis of certain tumors.
Limitations: This is a review article and does not present new primary experimental data.; Abstract provides no information on search strategy, inclusion criteria, or quality assessment of included studies.; Heterogeneity across many cancer types and study designs likely limits generalizability of conclusions.; The abstract does not report quantitative synthesis or effect sizes..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

ReviewMixed resultsLimited evidenceTier 4 · clinical

The systemic administration of intravenous melphalan

Journal of clinical oncology : official journal of the American Society of Clinical Oncology · Nov 1988 · narrative review

breast cancerovarian cancermultiple myelomarhabdomyosarcomamelanomacolon carcinomanon-Hodgkin's lymphomaHodgkin's diseaseneuroblastomanonhematologic tumor types

This article reviews clinical experience with intravenous (IV) melphalan. It reports that IV administration yields higher and more predictable blood levels than oral dosing, that lower IV doses (30–70 mg/m2) have been evaluated only in a limited number of diseases but showed notable activity in untreated rhabdomyosarcoma, and that high-dose IV melphalan (>140 mg/m2) with bone marrow reinfusion produces high response rates in several tumor types though survival benefit was seen only in certain poor-prognosis hematologic malignancies; toxicity is considerable. The authors state additional clinical trials are needed to define activity and subgroups most likely to benefit.

Studied with: bone marrow reinfusion, combination chemotherapy, high-dose regimens.

Key findings
  • IV melphalan provides higher and more predictable blood levels compared with oral administration.
  • An extensive phase I evaluation of IV melphalan has not been undertaken.
  • Lower doses (eg, 30 to 70 mg/m2) have been evaluated in a limited number of diseases, with striking activity observed in previously untreated rhabdomyosarcoma.
  • High-dose melphalan (>140 mg/m2) with bone marrow reinfusion yields high response rates in a variety of nonhematologic tumor types but did not improve survival in those settings.
  • In poor-prognosis patients with non-Hodgkin's lymphoma, Hodgkin's disease, multiple myeloma, or neuroblastoma, high-dose melphalan-containing regimens produced both high response rates and improved survival despite considerable toxicity.
  • Additional clinical trials are necessary to define the spectrum of activity at lower doses and to identify patient subgroups most likely to benefit from high-dose melphalan.
Limitations: Not a primary trial report; narrative summary without presented sample sizes or primary data in the abstract.; An extensive phase I evaluation of IV melphalan has not been undertaken, limiting dose-optimization data.; Lower-dose IV melphalan has been evaluated in only a limited number of diseases.; High-dose regimens are associated with considerable toxicity.; Abstract does not provide quantitative response or survival figures, follow-up durations, or trial design details..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Browse all studies mentioning Neuroblastoma →

Study mix

9 published studies by what they were done in. Lab and animal findings often do not carry over to people.

1 Human1 Animal7 Review/other
Reported directionReported positive4Mixed results2Inconclusive3

Compounds with reported-positive results in Neuroblastoma

Where at least one study reported a positive result, shown with the full picture, not just the wins. Positive results are more likely to be published, and most of these are early lab or animal studies that may not translate to people. This reports what studies found, not what works.

Preclinical only: lab / animal (1)
Genistein1 positive
Limitations: This article is a narrative review and does not present new clinical trial data.; The evidence summarized is primarily preclinical (in vitro and in vivo) with no clinical trial data provided in the abstract.; Mechanistic proposals are not proven clinical effects and require further experimental and clinical validation.; Safety and efficacy in patients, optimal dosing, and interactions with standard therapies are not established in this review..
Cited positive studies (1)

Evidence at a glance: compounds studied in Neuroblastoma

A deterministic grade of what published studies report for each: strength of evidence, the reported direction, and the largest credible effect, strongest-evidence first. This summarizes findings; it is not a claim that anything works.

GenisteinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

No human studies yet · No numeric effect sizes reported · Based on a single study.

What the research shows for Neuroblastoma

A plain-language summary of the reviewed studies OncoForge tracks for Neuroblastoma. It reports what those studies described, not a claim that any compound or therapy helps or harms Neuroblastoma. Most of this evidence is early, and findings often conflict.

  • A preclinical study of the antibody–drug conjugate CDX0239-PBD tested ALK-targeted delivery in ALK-expressing cancer models and reported that the conjugate was taken up by ALK-positive neuroblastoma cell lines and produced ALK-dependent cell killing in vitro.
  • The same preclinical study reported antitumor activity of CDX0239-PBD in mouse xenograft models of ALK-expressing cancers that included neuroblastoma models (results reported as positive in animal experiments).
  • A separate narrative review summarized published data on the adaptor protein CRKL across several tumor types, reporting CRKL overexpression and a possible role in aggressive behavior in the cancers reviewed, but that review did not present direct data on neuroblastoma.
  • Across the available reports, evidence for neuroblastoma is limited to laboratory and animal-model work focused on ALK-targeted antibody–drug conjugates; findings depend on target (ALK) expression and are preclinical in nature.

Supportive & alternative options discussed

  • Mind–body (MBSR / CBT): Also discussed as a supportive option for stress reduction, coping, and family-centered psychosocial care in neuroblastoma, but these specific studies do not provide evidence for its effects.
  • Exercise / prehabilitation: Also discussed as a supportive strategy to maintain function and quality of life during and after therapy in pediatric cancers including neuroblastoma; the studies provided here do not address exercise.
  • Acupuncture: Also discussed as a possible option for symptom control (for example, pain or nausea) in pediatric oncology; the studies above do not evaluate acupuncture.
  • Hyperthermia (heat): Also discussed experimentally as an adjunctive modality in some cancer settings; the studies summarized here do not test hyperthermia in neuroblastoma.
  • Ketogenic / metabolic therapy: Also discussed by some as a supportive dietary approach in oncology, but the studies provided do not examine ketogenic diets in neuroblastoma.
  • Mistletoe (VAE): Also discussed in some complementary-care contexts for cancer symptom support, but none of the studies summarized here provide evidence on mistletoe in neuroblastoma.

What we don’t know yet

  • Whether CDX0239-PBD is safe, tolerable, or active in humans with neuroblastoma — no clinical trial results in patients with neuroblastoma are reported in these studies.
  • How common and how heterogeneous ALK expression is across neuroblastoma patients and whether ALK expression reliably predicts response in patients.
  • Long-term effects, optimal dosing, and potential toxicities of ALK-targeted antibody–drug conjugates in children are unknown from the presented data.
  • Whether CRKL has a clinically relevant role in neuroblastoma (prevalence, prognostic value, or as a therapeutic target) is not established by the review, which focused on other tumor types.
  • How ALK-targeted ADCs would interact with current standard therapies for neuroblastoma, and whether resistance mechanisms would limit their usefulness, remain unaddressed.
These findings are preliminary and come mainly from laboratory and animal studies; clinical safety and efficacy in people with neuroblastoma have not been established.

Clinical trials in Neuroblastoma

11 ongoing · 29 completed · tracked from ClinicalTrials.gov. Recruiting is not the same as proven, and completed is not the same as positive — read the results. Not a recommendation.

Completed
5 stopped (terminated / withdrawn / suspended)

Search all trials on ClinicalTrials.gov →

Getting care & support

Nonprofit / Gov

Practical, vetted help for Neuroblastoma — advocacy, paying for treatment, second opinions, and caregivers.

If you’re struggling emotionally, you don’t have to wait.

Advocacy & community

No dedicated organization for this specific cancer is curated yet — these general organizations can help in the meantime.

Financial help

  • PAN Foundation ↗ — Copay assistance funds by diagnosis (funds open and close as money allows). · status changes often — check the fund’s site
  • HealthWell Foundation ↗ — Copay and premium assistance funds by disease. · status changes often — check the fund’s site
  • CancerCare — financial assistance ↗ — Limited grants plus free financial counseling. · status changes often — check the fund’s site
  • Family Reach ↗ — Help with everyday living costs (rent, transport, food) during treatment. · status changes often — check the fund’s site
  • NeedyMeds ↗ — Searchable directory of drug patient-assistance and discount programs. · status changes often — check the fund’s site
What you’ll typically need to apply
  • Your diagnosis and, if you have it, the specific drug/treatment name (from your care team).
  • Insurance details — your member ID card, or a note that you're uninsured (some funds require active insurance, some don't).
  • Proof of income and household size (recent pay stubs, a tax return, or a benefits letter) — most funds are income-based.
  • Your prescriber's contact information; some programs need the clinic to submit part of the application.
  • Apply early and re-check: funds open and close as money is available, so a closed fund may reopen.

General guidance — each program sets its own eligibility. Confirm requirements on the program’s site.

Help paying for the medicines on this page

Second opinions

Caregiver support

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