Disease Briefing

Ewing sarcoma: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-12Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 242 studies · 9,063 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in ewing sarcoma — EWSR1, FLI1, CD99, IGF1R, PARP1, STAG2, TP53, CDKN2A, KDM1A, CDK4, ERG, NKX2-2 — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

1
drugs carry an FDA label naming ewing sarcoma: Dactinomycin. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
0
of the 12 genes above carries a drug approved in ewing sarcoma — 60 drug entries reach them, 56 distinct once salt forms are merged, and 15 of those are approved for other indications. A statement about these gene targets, not about the disease
3
registered GD2 cell-therapy trials in ewing sarcoma, 3 active. Counted from ClinicalTrials.gov across 2 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
8
targets carry an Open Targets tractability signal and have no clinical programme of any kind: EWSR1, FLI1, CD99, STAG2, TP53, KDM1A, ERG, NKX2-2
455
human GEO series match the disease; 242 survive on-topic filtering, and only 5 are patient cohorts of 100+ samples
91
Europe PMC full-text papers name GSE17679 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$11.9M
NIH obligations in FY2025, up 470% since 2013 — while distinct core projects went 9 to 27. Both more projects (+200%) and larger awards, the latter carrying more of the growth; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-12 · weekly

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

Of 12 recurrently implicated genes, 5 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Ewing sarcoma does have labelled therapy — 1 drugs, listed in the next section.

Two different questions

The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what ewing sarcoma is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell therapy here
EWSR1 0 No drug antibody, protein degrader, small molecule
FLI1 0 No drug protein degrader, small molecule
CD99 0 No drug antibody, protein degrader
IGF1R 20 3 approvedMasoprocol, Mecasermin, TeprotumumabApproved in prostate cancer, Growth delay, hypothyroidism and 3 other indications. No ewing sarcoma indication appears on these drugs’ labels.1 active of 13 ewing sarcoma trials antibody, other clinical modality, protein degrader, small molecule
PARP1 12 8 approvedNiraparib, Niraparib Tosylate, Olaparib, Pamiparib, Rucaparib, Rucaparib Camsylate, Talazoparib, VeliparibApproved in fallopian tube neoplasm, peritoneal neoplasm, ovarian neoplasm and 12 other indications. No ewing sarcoma indication appears on these drugs’ labels.2 active of 8 ewing sarcoma trials protein degrader, small molecule
STAG2 0 No drug protein degrader
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo ewing sarcoma trial of any of these drugs other clinical modality, protein degrader, small molecule
CDKN2A across cancers → 0 No drug
KDM1A 1 Phase 3BomedemstatNo ewing sarcoma trial of any of these drugs protein degrader, small molecule
CDK4 across cancers → 15 4 approvedAbemaciclib, Palbociclib, Ribociclib, TrilaciclibApproved in breast cancer, breast neoplasm, breast carcinoma and 2 other indications. No ewing sarcoma indication appears on these drugs’ labels.4 active of 9 ewing sarcoma trials protein degrader, small molecule
ERG 0 No drug protein degrader, small molecule
NKX2-2 0 No drug protein degrader

Dataset evidence counts studies in the 242-study ranked set whose title or abstract names the gene; the bar is scaled to FLI1. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-12. 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.

What is actually approved here

openFDA drug labels · weekly

1 drug carries an FDA label naming ewing sarcoma: Dactinomycin. Separately, 15 of the drugs returned for the genes in the table above are approved only for other diseases and reach ewing sarcoma through trials, not through their labels.

1Labelled for ewing sarcomaFDA INDICATIONS AND USAGE names the disease
15Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
3Active GD2 cell-therapy trialsof 3 registered

Every label that names ewing sarcoma

DrugRoleWhat the label says
DactinomycinDACTINOMYCIN, Dactinomycin, dactinomycinLabelled hereEwing Sarcoma Dactinomycin for Injection is indicated for the treatment of adult and pediatric patients with Ewing sarcoma, as part of a multi-phase, combination chemotherapy regimen.

9 labels match indications_and_usage:"Ewing sarcoma"; they collapse to 1 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-12. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against GD2

ClinicalTrials.gov · retrieved 2026-09-12 · weekly

GD2 is the busiest cell-therapy antigen in ewing sarcoma: 3 registered trials, 3 still active.

TrialPhaseStatusTitleLast update
NCT033730971/2ActiveAnti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors2025-02-05
NCT036356321ActiveC7R-GD2.CART Cells for Patients With Relapsed or Refractory Neuroblastoma and Other GD2 Positive Cancers (GAIL-N)2026-07-06
NCT033567821/2Not yet recruitingSafety and Efficacy Evaluation of 4th Generation Safety-engineered CAR T Cells Targeting Sarcomas2026-08-26

3 of 3 shown, most recently active first. Other antigens searched: B7-H3 (3). Source: ClinicalTrials.gov API v2, retrieved 2026-09-12. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Not builtNo literature momentum facts for this disease yet. Run pipeline/momentum.py and rebuild.

Disease burden

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

MeasureValueMatchWhat it counts
Incidence rate3 cases per 1,000,000 children and adolescents under 20 per yeardirect2016-2020
New cases each year4,110 cases/yearproxycounts bone and joint cancer, which is broader than this disease; 2026
Deaths each year2,210 deaths/yearproxycounts bone and joint cancer, which is broader than this disease; 2026
People living with it65,261 people living with the diseaseproxycounts bone and joint cancer, which is broader than this disease; 2023
New cases each yearnot publishedSEER does not publish Ewing sarcoma; the PDQ gives a rate per million under 20 and no count, and multiplying it by a population would be a derivation the source does not make.
Five-year relative survivalnot publishedThe PDQ gives five-year survival as "80% to 85% for children younger than 15 years" and "69% for adolescents aged 15 to 19"; a single figure would be an average the source does not make.
Median age at diagnosisnot publishedNot published as a median; incidence peaks in adolescence.

Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis, incident cases is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming ewing sarcoma, after removing the 402 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

$11.9MNIH obligations, FY2025from $2.1M in FY2013 · +470%
27distinct projects funded9 in FY2013
$36.3Mpeak year was FY2018obligations, all institutes
89%of FY2025 awards from NCI25 of 28

NIH obligations by fiscal year

$9M$18M$27M$36M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$2.1M9915
FY2014$2.3M9918
FY2015$2.7M9923
FY2016$2.8M121228
FY2017$3.1M141328
FY2018$36.3M281928
FY2019$22.6M251830
FY2020$7.6M201828
FY2021$8.1M212137
FY2022$8.3M201936
FY2023$11.2M292840
FY2024$10.5M332949
FY2025$11.9M282742

Where FY2025 money went

InstitutionObligationsAwards
National Institute Of Neurological Disorders And Stroke$2.9M0
Dana-Farber Cancer Inst$2.2M5
University Of Nebraska Medical Center$0.7M2
Stanford University$0.7M0
University Of California, San Diego$0.6M1
University Of Michigan At Ann Arbor$0.5M0
Emory University$0.4M1
University Of Minnesota$0.4M1
Univ Of North Carolina Chapel Hill$0.4M0
Virginia Commonwealth University$0.4M0

Text search Ewing sarcoma over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-12.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

NIH institutes

NCI25 · 89%
NIGMS1 · 4%
NCATS1 · 4%
NINDS1 · 4%

Projects by administering institute. The rows above are the top 4 and account for 28 of 28.

Award mechanisms

R0112 · 43%
K083 · 11%
F312 · 7%
U012 · 7%
R212 · 7%
ZIA2 · 7%

R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 11 and account for 28 of 28.

Where it lands

National Institute Of Neurological Disor$2.9M · 24.4%
Dana-Farber Cancer Inst$2.2M · 18.8%
University Of Nebraska Medical Center$0.7M · 6.3%
Stanford University$0.7M · 5.9%
University Of California, San Diego$0.6M · 5.3%
University Of Michigan At Ann Arbor$0.5M · 3.9%

Share of $11.9M in FY2025. The top three hold 50%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

455 human GEO series match ewing sarcoma. Keyword relevance cannot tell a 1,000-sample 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.

Composition of the 242-study ranked set

cell line 97unspecified 76mixed 32patient 30xenograft 7
97 cell line76 unspecified32 mixed30 patient7 xenograft150 carry clinical annotation48 carry survival5 patient cohorts ≥100 GEO samples9,063 GEO samples totalin 5 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE34620Expression profiling of Ewing sarcoma samples2012 · array117633.6
patient cohortAPT 0.75molecularEWSR1150 cites
GSE88826DNA methylation heterogeneity defines a disease spectrum in Ewing sarcoma2017 · methylation18855.5
patient cohortAPT 0.95molecularFLI1195 cites
GSE142162Expression profiling of Ewing sarcoma samples2021 · array7973.7
mixedAPT 0.75survivalmolecularEWSR1FLI1STAG277 cites
GSE63157Gene Expression Profiling of Ewing Sarcoma Tumors Reveals the Prognostic Importance of Tumor-Stromal Interactions: A Report from the Children's Oncology Group2014 · array85672.1
APT 0.5survival77 cites
GSE61953Genome-wide chromatin analysis of Ewing sarcoma2014 · chromatin70178.7
APT 0.75369 cites
GSE45544Ewing sarcoma compared to a normal body map2013 · array44246.8
cell lineAPT 0.95199 cites
GSE17679Inflammatory gene profiling of Ewing sarcoma family of tumors2011 · array117911.9
APT 0.2589 cites
GSE113604BET bromodomain dependency in EWS/ETS driven Ewing Sarcoma2018 · sequencing2221.9
patient cohortAPT 0.75survivalstagemolecularERGFLI159 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE243347Ewing sarcoma single-cell transcriptome analysis reveals functionally impaired antigen-presenting cells2023 · single-cell2742.0
patient cohortAPT 0.5stage23 cites
GSE229906Chimeric protein EWS::FLI1 drives cell proliferation in Ewing Sarcoma via aberrant expression of KCNN1/SK1 and dysregulation of calcium signaling2024 · sequencing4521.6
mixedAPT 0.05survivalmolecularERGFLI15 cites
GSE277083Single cell RNA sequencing of Ewing sarcoma tumors demonstrate transcriptional heterogeneity and clonal evolution2025 · single-cell1522.4
patient cohortAPT 0.5survival9 cites
GSE181554Transcriptional constraint of EWS/FLI by an ETS transcription factor promotes Ewing sarcoma growth.2022 · chromatin13023.8
APT 0.7544 cites
GSE311851LSD1 Performs Demethylase-Independent and Context-Specific Roles in Ewing Sarcoma2025 · sequencing1141
mixedAPT 0.05survivalmolecularERGFLI1KDM1A
GSE274115Polyamine Depletion Inhibits Ewing Sarcoma Metastasis by Inducing Ferroptosis2025 · sequencing912.4
mixedAPT 0.25survivalstage9 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. SubSeries are collapsed to one row per study by linked PMID. Of 455 series retrieved, 17 were dropped by the profile’s exclusion rules and 88 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Where the gaps are

Derived from the sections above · recomputed on every refresh

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

EWSR1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

FLI1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

CD99

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

STAG2

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

CDKN2A

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

ERG

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

NKX2-2

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

How a machine reads this page

Static

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite.

Fully server-rendered

Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.

Every number carries a source

Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-12), ClinicalTrials.gov (2026-09-12), openFDA (2026-09-12), NCBI GEO (2026-09-12), NIH RePORTER (2026-09-12).

Negatives stated explicitly

Where nothing exists for ewing sarcoma — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated negative is the finding.

Denominators, not just percentages

Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.

The filters are published

3 exclusion patterns are applied to free text before anything is ranked, because A673 and SK-N-MC is used as a model system in generic sarcoma and (for SK-N-MC, historically) neuroblastoma work. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Ewing sarcoma",
  "mesh": "Sarcoma, Ewing",
  "facts": "https://usebiotransfer.org/disease/ewing-sarcoma.json",
  "methods": "https://usebiotransfer.org/methods/",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}