Target landscape
Open Targets · retrieved 2026-09-12 · weekly12 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.
| Gene | Drugs | Approval, and whether it reached this disease | Open Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it works | Cell 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 · weekly1 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.
Every label that names ewing sarcoma
| Drug | Role | What the label says |
|---|---|---|
| DactinomycinDACTINOMYCIN, Dactinomycin, dactinomycin | Labelled here | Ewing 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 · weeklyGD2 is the busiest cell-therapy antigen in ewing sarcoma: 3 registered trials, 3 still active.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT03373097 | 1/2 | Active | Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors | 2025-02-05 |
| NCT03635632 | 1 | Active | C7R-GD2.CART Cells for Patients With Relapsed or Refractory Neuroblastoma and Other GD2 Positive Cancers (GAIL-N) | 2026-07-06 |
| NCT03356782 | 1/2 | Not yet recruiting | Safety and Efficacy Evaluation of 4th Generation Safety-engineered CAR T Cells Targeting Sarcomas | 2026-08-26 |
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 · monthlyNot 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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| Incidence rate | 3 cases per 1,000,000 children and adolescents under 20 per year | direct | 2016-2020 |
| New cases each year | 4,110 cases/year | proxy | counts bone and joint cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Bone and Joint Cancer, retrieved 2026-09-12 |
| Deaths each year | 2,210 deaths/year | proxy | counts bone and joint cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Bone and Joint Cancer, retrieved 2026-09-12 |
| People living with it | 65,261 people living with the disease | proxy | counts bone and joint cancer, which is broader than this disease; 2023 SEER Cancer Stat Facts, Bone and Joint Cancer, retrieved 2026-09-12 |
| New cases each year | — | not published | SEER 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 survival | — | not published | The 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 diagnosis | — | not published | Not 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 · quarterlyNIH 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.
NIH obligations by fiscal year
| Year | Obligations | Awards | Distinct projects | Dropped as off-topic |
|---|---|---|---|---|
| FY2013 | $2.1M | 9 | 9 | 15 |
| FY2014 | $2.3M | 9 | 9 | 18 |
| FY2015 | $2.7M | 9 | 9 | 23 |
| FY2016 | $2.8M | 12 | 12 | 28 |
| FY2017 | $3.1M | 14 | 13 | 28 |
| FY2018 | $36.3M | 28 | 19 | 28 |
| FY2019 | $22.6M | 25 | 18 | 30 |
| FY2020 | $7.6M | 20 | 18 | 28 |
| FY2021 | $8.1M | 21 | 21 | 37 |
| FY2022 | $8.3M | 20 | 19 | 36 |
| FY2023 | $11.2M | 29 | 28 | 40 |
| FY2024 | $10.5M | 33 | 29 | 49 |
| FY2025 | $11.9M | 28 | 27 | 42 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| National Institute Of Neurological Disorders And Stroke | $2.9M | 0 |
| Dana-Farber Cancer Inst | $2.2M | 5 |
| University Of Nebraska Medical Center | $0.7M | 2 |
| Stanford University | $0.7M | 0 |
| University Of California, San Diego | $0.6M | 1 |
| University Of Michigan At Ann Arbor | $0.5M | 0 |
| Emory University | $0.4M | 1 |
| University Of Minnesota | $0.4M | 1 |
| Univ Of North Carolina Chapel Hill | $0.4M | 0 |
| Virginia Commonwealth University | $0.4M | 0 |
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
Projects by administering institute. The rows above are the top 4 and account for 28 of 28.
Award mechanisms
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
Share of $11.9M in FY2025. The top three hold 50%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly455 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
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE34620 | Expression profiling of Ewing sarcoma samples2012 · array | 117 | 63 | 3.6 | patient cohortAPT 0.75molecularEWSR1150 cites |
| GSE88826 | DNA methylation heterogeneity defines a disease spectrum in Ewing sarcoma2017 · methylation | 188 | 5 | 5.5 | patient cohortAPT 0.95molecularFLI1195 cites |
| GSE142162 | Expression profiling of Ewing sarcoma samples2021 · array | 79 | 7 | 3.7 | mixedAPT 0.75survivalmolecularEWSR1FLI1STAG277 cites |
| GSE63157 | Gene Expression Profiling of Ewing Sarcoma Tumors Reveals the Prognostic Importance of Tumor-Stromal Interactions: A Report from the Children's Oncology Group2014 · array | 85 | 67 | 2.1 | APT 0.5survival77 cites |
| GSE61953 | Genome-wide chromatin analysis of Ewing sarcoma2014 · chromatin | 70 | 17 | 8.7 | APT 0.75369 cites |
| GSE45544 | Ewing sarcoma compared to a normal body map2013 · array | 44 | 24 | 6.8 | cell lineAPT 0.95199 cites |
| GSE17679 | Inflammatory gene profiling of Ewing sarcoma family of tumors2011 · array | 117 | 91 | 1.9 | APT 0.2589 cites |
| GSE113604 | BET bromodomain dependency in EWS/ETS driven Ewing Sarcoma2018 · sequencing | 22 | 2 | 1.9 | patient cohortAPT 0.75survivalstagemolecularERGFLI159 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE243347 | Ewing sarcoma single-cell transcriptome analysis reveals functionally impaired antigen-presenting cells2023 · single-cell | 27 | 4 | 2.0 | patient cohortAPT 0.5stage23 cites |
| GSE229906 | Chimeric protein EWS::FLI1 drives cell proliferation in Ewing Sarcoma via aberrant expression of KCNN1/SK1 and dysregulation of calcium signaling2024 · sequencing | 45 | 2 | 1.6 | mixedAPT 0.05survivalmolecularERGFLI15 cites |
| GSE277083 | Single cell RNA sequencing of Ewing sarcoma tumors demonstrate transcriptional heterogeneity and clonal evolution2025 · single-cell | 15 | 2 | 2.4 | patient cohortAPT 0.5survival9 cites |
| GSE181554 | Transcriptional constraint of EWS/FLI by an ETS transcription factor promotes Ewing sarcoma growth.2022 · chromatin | 130 | 2 | 3.8 | APT 0.7544 cites |
| GSE311851 | LSD1 Performs Demethylase-Independent and Context-Specific Roles in Ewing Sarcoma2025 · sequencing | 114 | 1 | — | mixedAPT 0.05survivalmolecularERGFLI1KDM1A |
| GSE274115 | Polyamine Depletion Inhibits Ewing Sarcoma Metastasis by Inducing Ferroptosis2025 · sequencing | 9 | 1 | 2.4 | 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 refreshEach 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
StaticThis 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."
}