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Open research questions in Neuroblastoma Research and Treatments

126 gap statements mined from Neuroblastoma Research and Treatments papers in our 4.5M-paper local library, which holds 611 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • The patient's tumor showed unexpected progression after initial chemotherapy. The SDHA variant remains classified as a variant of uncertain significance. The clinical significance of the coexistence of neonatal neuroblastoma and achondroplasia with an SDHA variant is not fully understood.

    Prenatally Detected Neonatal Neuroblastoma Associated with Achondroplasia and an SDHA Variant · 2026 · DOI

  • The need for heightened caution during the interpretation of 18F-MFBG PET/CT due to its susceptibility to false-positive findings. The lack of established quantitative SUVmax thresholds to differentiate ganglia from metastatic lymph nodes.

    Uptake Patterns of Peripheral Ganglia on 18F-MFBG PET/CT in Neuroblastoma Patients · 2026 · DOI

  • The mechanism of MYCN's transcriptional activity remains incompletely understood. The cofactors that mediate MYCN's activity are not well-characterized. The genome-wide interactions of MYCN and its cofactors are not well-studied.

    MYCN drives oncogenesis by cooperating with the histone methyltransferase G9a and the WDR5 adaptor to orchestrate global gene transcription · 2024 · DOI

  • The impact of extent of resection of the primary tumor on outcomes in patients with high-risk neuroblastoma (HRNB) remains uncertain.

    Impact of Extent of Surgical Resection on Survival in Patients With High-risk Neuroblastoma · 2026 · DOI

  • However, the interplay of ECM remodeling with the traceable origin and fate of tumor cell phenotypes remains poorly understood.

    Unmasking phenotypic heterogeneity and extracellular matrix architecture in neuroblastoma through orthotopic xenograft modeling · 2026 · DOI

  • However, KSRP's functional role in neuroblastoma (NB) and the mechanism mediating IMB's antitumor effects remain unclear.

    KSRP ‐dependent immunogenic cell death induced by pyridazinone derivative IMB5036 drives antitumor immunity in neuroblastoma · 2026 · DOI

  • Discussion/Significance of Impact: These first-in-class chemical probes for BPTF PHD will enable further investigation of BPTF and high-risk neuroblastoma progression, as well as its role in other diseases.

    459 Development of small molecules targeting an epigenetic modulator for pediatric neuroblastoma · 2025 · DOI

  • Although the detailed roles of CRC in individual clinical situations remain to be elucidated, interference with the subtype-specific patterns of CRC-dependent gene expression, together with inactivation of an additional survival factor(s), offers new opportunities for mechanism-based, personalized treatment.

    Targeting transcription in neuroblastoma: focus on the core regulatory circuit · 2025 · DOI

  • Urinary homovanillic acid (HVA) and vanillylmandelic acid (VMA) are widely used diagnostic markers for neuroblastoma, but urinary markers for risk assessment prior to invasive biopsy are lacking.

    Cystathionine as a Potential Urinary Marker for Diagnosing and Assessing Pretreatment Risk in Neuroblastoma · 2025 · DOI

  • Commentary was provided for each data item to explain the rationale for selecting it as a core or non-core element, its clinical relevance and to highlight potential areas of disagreement or lack of evidence, in which case a consensus position was formulated.

    Data set for reporting of peripheral neuroblastic tumours: recommendations from the international collaboration on cancer reporting ( ICCR ) · 2025 · DOI

  • A modification genes and their variants in neuroblastoma remain to be elucidated.

    Genetic variants of m1A modification genes and the risk of neuroblastoma: novel insights from a Chinese case-control study · 2025 · DOI

  • PLK4 has been linked to NB tumorigenesis, but its role in regulating differentiation remains unclear.

    PLK4 as a Key Regulator of Neuroblastoma Differentiation and a Promising Therapeutic Target · 2025 · DOI

  • The role of disulfidptosis, a novel form of programmed cell death, remains poorly understood in the context of NB.

    Integrated machine learning-driven disulfidptosis profiling: CYFIP1 and EMILIN1 as therapeutic nodes in neuroblastoma · 2024 · DOI

  • Cyclin-dependent kinase 5 regulatory subunit-associated protein 3 (CDK5RAP3) is a newly identified super-enhancer-driven gene regulated by master TFs in NB; however, its function in NB remains unclear.

    CDK5RAP3 is a novel super-enhancer-driven gene activated by master TFs and regulates ER-Phagy in neuroblastoma · 2024 · DOI

  • The function and clinical relevance of NBPF1 in PCa remain undefined. The downstream pathways regulated by NBPF1 in PCa are unclear.

    NBPF1 acts as a tumor suppressor in prostate cancer by regulating the PI3K/AKT pathway · 2026 · DOI

  • The specific downstream targets of NBPF1 beyond PI3K/AKT modulation and the exact mechanisms linking NBPF1 loss to therapy resistance in advanced or castrate-resistant prostate cancer remain uncharacterized. Systematic identification of NBPF1 interacting proteins and transcriptional targets through co-immunoprecipitation and ChIP-seq is required.

    NBPF1 acts as a tumor suppressor in prostate cancer by regulating the PI3K/AKT pathway · 2026 · DOI

  • Substantial unmet needs remain, as many pediatric cancers still lack effective molecularly guided therapies. Looking forward, the future trajectory of pediatric oncology drug development will depend heavily on deeper, biology‑driven research.

    Advances in targeted therapies for pediatric tumors · 2026 · DOI

  • Further studies are needed to confirm the findings and identify other predictors of improved OS and EFS. Studies should be conducted in multiple centers to increase generalizability. Research should focus on improving supportive care and reducing infection risks in resource-limited settings.

    Autologous stem cell transplantation for pediatric solid tumors in a resource-limited setting: a single-center experience of 15 years · 2026 · DOI

  • Distinguishing residual disease from postoperative changes remains challenging. Residual disease frequently presents with heterogeneous signal characteristics.

    Inter-reader agreement and diagnostic accuracy of early postoperative magnetic resonance imaging for detecting macroscopic residual disease in neuroblastic tumors · 2026 · DOI

  • MYCN non-amplified (MYCN-NA) neuroblastoma (NB) demonstrates considerable heterogeneity in both biological and clinical aspects, and its molecular and biological characteristics remain inadequately defined.

    Comprehensive transcriptomics and proteome analysis to identify prognostic risk factors for MYCN non-amplified high-risk neuroblastoma · 2026 · DOI

  • Segmental gain of chromosome 17q is the most common genetic aberration in high-risk neuroblastoma, but its role in disease progression is poorly understood.

    Gain of chromosome 17 is an early genetic abnormality in neuroblastoma with PPM1D emerging as a strong candidate oncogene driving tumor progression · 2025 · DOI

  • MALAT1 is a dual oncogenic lncRNA with some therapeutic potential but has rarely been studied in neuroblastoma.

    Association of MALAT1 gene polymorphisms with neuroblastoma susceptibility in children from Jiangsu Province · 2025 · DOI

  • Further studies are needed to validate the use of 18F-MFBG PET/CT in neuroblastoma diagnosis and treatment, - Research on the use of 18F-MFBG PET/CT in other types of cancer is warranted, - Studies on the long-term effects of 18F-MFBG PET/CT on patients…

    Uptake Patterns of Peripheral Ganglia on 18F-MFBG PET/CT in Neuroblastoma Patients · 2026 · DOI

  • Small sample size - Limited knowledge regarding the long-term side effects of anti-GD2 therapy - No reports of the specific late side effects and health-related quality of life after…

    Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study · 2025 · DOI

  • Further research is needed to assess the long-term side effects of anti-GD2 therapy - Studies should investigate the health-related quality of life after this therapy - Research should focus on reducing the risk of secondary malignant…

    Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study · 2025 · DOI

Most-cited papers in Neuroblastoma Research and Treatments

Most recent work

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Open questions raised by papers listed on this page include: (1) The clinical significance of the coexistence of neonatal neuroblastoma and achondroplasia with an SDHA variant is not fully understood (“Prenatally Detected Neonatal Neuroblastoma Associated with Achondroplasia and…”, 2026); (2) The lack of established quantitative SUVmax thresholds to differentiate ganglia from metastatic lymph nodes (“Uptake Patterns of Peripheral Ganglia on 18F-MFBG PET/CT in Neuroblastoma…”, 2026); (3) The interplay of ECM remodeling with the traceable origin and fate of tumor cell phenotypes remains poorly understood (“Unmasking phenotypic heterogeneity and extracellular matrix architecture in…”, 2026); (4) KSRP's functional role in neuroblastoma (NB) and the mechanism mediating IMB's antitumor effects remain unclear (“KSRP ‐dependent immunogenic cell death induced by pyridazinone derivative…”, 2026); (5) The downstream pathways regulated by NBPF1 in PCa are unclear (“NBPF1 acts as a tumor suppressor in prostate cancer by regulating the PI3K/AKT…”, 2026). 126 gap statements have been mined from Neuroblastoma Research and Treatments papers in our 4.5M-paper local library, which holds 611 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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