Neuroblastoma: research and therapeutic landscape

Nine genes recurrently implicated in neuroblastoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of nine recurrently implicated genes, only three carry any drug at all, and none of those drugs is approved in neuroblastoma. That is a statement about these nine gene targets, not about the disease: neuroblastoma does have approved therapies, listed below. The whitespace is narrower than the zeros suggest — GPC2, L1CAM and GD2 are already in cell-therapy trials, and MYCN is the one target here with no clinical programme at all.

Dataset evidence counts studies in the 235-study ranked set whose title or abstract names the gene; the bar is scaled to MYCN. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-05. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

Read this before the table above. Every "approved" drug listed against a neuroblastoma gene is approved for a different disease. Zero of the sixteen drugs returned for these nine genes carries a neuroblastoma indication. ALK inhibitors reach neuroblastoma only through trials, not through their labels.

GD2 is a glycolipid with no gene of its own, so conventional target databases miss it entirely. Reached through its synthase B4GALNT1, it is the single busiest antigen in the disease: 28 registered trials, 21 still active.

7 of 28 shown. Source: ClinicalTrials.gov (US NIH/NLM, public domain), retrieved 2026-09-05. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code may be missed.

What the field is actually publishing on, measured as share of the neuroblastoma literature rather than raw counts — so a topic only registers as rising if it is growing faster than the field itself. Two windows of MeSH-indexed papers: 2015–2018 (n=1,931) against 2021–2025 (n=3,122).

Read the middle band first. Terms with a real prior baseline are the trustworthy signal; the top band is inflated by MeSH vocabulary that did not exist in the earlier window.

Two shifts matter commercially: chimeric antigen receptors have grown 5.9× as a share of the literature, and glypicans — the GPC2 target that still carries no drug — have grown 3.5×. Academic attention is moving toward targets the clinic has not yet reached.

Rising with a real baseline

Present in both windows, so the growth is a genuine shift in attention rather than a new MeSH descriptor.

New vocabulary

Absent from the earlier window entirely. Some are genuinely new science, some are MeSH descriptors introduced after 2018 — the fold change is meaningless either way, so counts are shown instead.

Cooling

Losing share fastest. Almost every one is a method, not a biological question — the field is not abandoning these topics, it is changing the instruments it studies them with.

Where the mass still sits

Largest topics by share of the 2021–2025 window, with their trend. The field's centre of gravity has barely moved.

Source: PubMed E-utilities (esearch / efetch), retrieved 2026-09-05. Query restricted to neuroblastoma[MeSH Major Topic] with explicit exclusions. Two corrections were required and both changed the answer. An unrestricted query returned Alzheimer Disease at 5.4% and Parkinson Disease at 4.3% of "neuroblastoma" papers — these are neurodegeneration studies using SH-SY5Y neuroblastoma cells as a neuron model, and they dominated the rising list. Olfactory esthesioneuroblastoma, an unrelated sinonasal tumour of adults, contributed a further 4.6%. Known limitation: MeSH indexing lags publication by roughly a year, so counts for 2025–2026 are incomplete and no year past 2024 should be read as a trend.

NIH awards whose title, abstract or terms name neuroblastoma, by fiscal year. Award dollars are the obligations recorded in that year, not the lifetime value of a grant.

The money nearly doubled while the number of funded projects did not move. Larger awards, not more labs — and the median award grew 72%.

US public funding for this disease went from $89M to $186M in twelve years, but is concentrated: ten institutions hold two-thirds of it, and NIH intramural divisions alone hold 24%.

NIH obligations by fiscal year

Dollars up, labs flat

Six of thirteen years shown; all thirteen were retrieved. Project counts are distinct core project numbers, so supplements and multi-year records do not inflate them.

Where it goes — FY2024

Source: NIH RePORTER API v2, retrieved 2026-09-06; 13 fiscal years, 3,461 award records before filtering. The same contamination pattern as the literature section applies here and was filtered the same way — 25 to 50 records per year (roughly 13%) were olfactory neuroblastoma, or neurodegeneration grants using SH-SY5Y cells; these are excluded from every figure above. Known limitations: this is NIH only — it excludes EU, UK, and the foundation funding (St. Baldrick's, Alex's Lemonade Stand, CRUK) that is disproportionately large in pediatric oncology, so it is a floor rather than a total. FY2025 records may still be incomplete. Publication counts compared here are worldwide while funding is US-only, so the two series are directional, not a ratio.

1,625 human GEO series match neuroblastoma. Keyword relevance cannot tell a 498-patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Sorted by this briefing’s heuristic reusability score — the weights below are editorial choices, published so they can be argued with — rather than relevance. A patient cohort carrying survival annotation outranks a larger cell-line panel, and the reuse count tells you whether anyone has successfully worked with the deposited files.

Read the reuse column first: it separates data that has been independently validated from data that was deposited once and never touched again. Only 11 of 235 studies clear ten reuses.

Composition of the 235-study ranked set

Reading GSE26494: very high citation impact (RCR 21.7) but only two reuses — the paper is influential, the deposited data is not travelling. That divergence is why both columns are shown instead of being blended into one number.

How the ranking is computed

Published in full so a reader — or a model — can check it rather than trust it. No learned weights, no hidden features.

Sources: NCBI GEO (esearch / esummary), Europe PMC REST, NIH iCite — all retrieved 2026-09-05. SubSeries are collapsed to one row per study by linked PMID. Known limitation: the on-topic filter is text-based and still admits studies that merely mention the disease — GSE16716 (MAQC-II) ranked second and was removed by hand. The 235-study set is a relevance-ranked sample of the 1,625 matches, not a census, so the composition figures describe the sample only.

Nothing in this section is written by hand. Each card is a rule applied to the tables above: a target that is tractable but undrugged, or drugged but not in this disease.

The reason this page is shaped the way it is. Every design choice above serves a crawler as much as a reader.

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