Disease Briefing

Osteosarcoma: 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 2026-09-30Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 248 studies · 5,783 GEO samples
Page Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in osteosarcoma — TP53, RB1, MYC, CDK4, MDM2, VEGFA, KDR, ERBB2, IGF1R, CD276, PTHLH, CDK6 — 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.

0
of the 12 genes above carries a drug approved in osteosarcoma — 201 drug entries reach them, 181 distinct once salt forms are merged, and 29 of those are approved for other indications. A statement about these gene targets, not about the disease
6
registered GD2 cell-therapy trials in osteosarcoma, 4 active. Counted from ClinicalTrials.gov across 3 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
5
targets carry an Open Targets tractability signal and no trial in osteosarcoma that the searches found (no trial naming a drug Open Targets lists for the gene, none naming it as a cell-therapy target): TP53, RB1, MYC, MDM2, PTHLH. ERBB2, CD276 have cell-therapy trials, so they are undrugged rather than untouched
1,013
human GEO series match the disease; 248 survive on-topic filtering, and only 4 are patient cohorts of 100+ samples
467
Europe PMC full-text papers name GSE21257 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$21.1M
NIH obligations in FY2025, up 287% since 2013 — while distinct core projects went 16 to 32. Both more projects (+100%) and larger awards, the latter carrying more of the growth; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-30 · weekly

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

Of 12 recurrently implicated genes, 10 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: No drug carries an FDA label naming osteosarcoma either, which the next section states in full.

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 osteosarcoma 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
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo osteosarcoma trial of any of these drugs other clinical modality, protein degrader, small molecule —
RB1 across cancers → 0 No drug— protein degrader, small molecule —
MYC across cancers → 0 No drug— protein degrader, small molecule —
CDK4 across cancers → 15 2 approvedRibociclib, TrilaciclibApproved in breast cancer, breast carcinoma, small cell lung carcinoma. No osteosarcoma indication appears on these drugs’ labels.3 active of 9 osteosarcoma trials protein degrader, small molecule —
MDM2 across cancers → 4 Phase 3Alrizomadlin, Idasanutlin, Navtemadlin, SiremadlinNo osteosarcoma trial of any of these drugs antibody, protein degrader, small molecule —
VEGFA across cancers → 11 1 approvedAbicipar PegolApproved in ocular vascular disorder. No osteosarcoma indication appears on these drugs’ labels.1 active of 3 osteosarcoma trials antibody, other clinical modality, protein degrader, small molecule —
KDR across cancers → 67 12 approvedAxitinib, Cabozantinib, Cediranib, Lenvatinib, Midostaurin, Nintedanib, Pazopanib, Ramucirumab, Rivoceranib, Sorafenib, Surufatinib, TivozanibApproved in renal cell carcinoma, thyroid gland carcinoma, thyroid cancer and 12 other indications. No osteosarcoma indication appears on these drugs’ labels.19 active of 47 osteosarcoma trials antibody, other clinical modality, protein degrader, small molecule —
ERBB2 across cancers → 43 10 approvedAfatinib, Dacomitinib, Lapatinib, Margetuximab, Masoprocol, Neratinib, Trastuzumab Duocarmazine, Tucatinib, Zanidatamab, ZenocutuzumabApproved in non-small cell lung carcinoma, breast cancer, HER2 positive breast carcinoma and 6 other indications. No osteosarcoma indication appears on these drugs’ labels.2 active of 4 osteosarcoma trials antibody, other clinical modality, protein degrader, small molecule 1 active of 1 trial
IGF1R across cancers → 20 2 approvedMasoprocol, MecaserminApproved in prostate cancer, Growth delay, hypothyroidism and 1 other indications. No osteosarcoma indication appears on these drugs’ labels.6 trials, none active antibody, other clinical modality, protein degrader, small molecule —
CD276 across cancers → 2 Phase 2/38H9 131I, Enoblituzumab2 trials, none active antibody, protein degrader 4 active of 5 trials
PTHLH 1 Phase 1/2CalNo osteosarcoma trial of any of these drugs antibody, small molecule —
CDK6 across cancers → 10 2 approvedRibociclib, TrilaciclibApproved in breast cancer, breast carcinoma, small cell lung carcinoma. No osteosarcoma indication appears on these drugs’ labels.3 active of 9 osteosarcoma trials protein degrader, small molecule —

Dataset evidence counts studies in the 248-study ranked set whose title or abstract names the gene; the bar is scaled to MYC. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-30. 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

No drug carries an FDA label naming osteosarcoma. The 26 approved drugs returned for the genes above are approved for other diseases and reach this one only through trials.

0Labelled for osteosarcomaFDA INDICATIONS AND USAGE names the disease
26Approved, but for another diseasereturned for the genes in the table above
1Backbone agents listing itbroad cytotoxics whose labels name many tumours
4Active cell-therapy trials naming GD2of 6 registered

Every label that names osteosarcoma

DrugRoleWhat the label says
MethotrexateMETHOTREXATE, MethotrexateBackboneOsteosarcoma Methotrexate Injection is indicated for the treatment of adults and pediatric patients with osteosarcoma as part of a combination chemotherapy regimen.
Leucovorin CalciumLEUCOVORIN CALCIUM, Leucovorin Calcium, Leucovorin calciumSupportiveLeucovorin rescue is indicated after high dose methotrexate therapy in osteosarcoma.
LevoleucovorinKHAPZORY, LEVOLEUCOVORIN, LEVOLEUCOVORIN CALCIUMSupportiveLevoleucovorin injection rescue is indicated after high-dose methotrexate therapy in osteosarcoma.
Levoleucovorin CalciumLevoleucovorinSupportiveLevoleucovorin Injection is a folate analog indicated for: Rescue after high-dose methotrexate therapy in adult and pediatric patients with osteosarcoma.

37 labels carry a positive indication naming the disease under indications_and_usage:"osteosarcoma" OR indications_and_usage:"osteogenic sarcoma"; they collapse to 4 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Supportive agents (Leucovorin Calcium, Levoleucovorin, Levoleucovorin Calcium) are given in the disease but do not act on it. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-18. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against GD2

ClinicalTrials.gov · retrieved 2026-09-18 · weekly

GD2 is the busiest cell-therapy target among those this briefing searched in osteosarcoma: 6 registered trials naming it, 4 still active.

TrialPhaseStatusTitleLast update
NCT054006031RecruitingAllogeneic Expanded Gamma Delta T Cells With GD2 Chemoimmunotherapy in Relapsed /Refractory Neuroblastoma or Refractory/ Relapsed Osteosarcoma2025-12-26
NCT037210681RecruitingStudy of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma2026-03-27
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
NCT021079631CompletedA Phase I Trial of T Cells Expressing an Anti-GD2 Chimeric Antigen Receptor in Children and Young Adults With GD2+ Solid Tumors2024-05-16
NCT045393661SuspendedTesting a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, GD2-CAR PERSIST Trial2026-08-05

6 of 6 shown, most recently active first. Other targets searched: B7-H3 (5), HER2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-18. A trial counts here when its registered conditions name osteosarcoma or a tumour-agnostic basket, its title names an administered cell product, and the title names this target: a HER2 CAR-T trial that adds a PD-1 antibody is a HER2 trial. A trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the osteosarcoma literature, not a count, because the field itself grew: 2015–2018 (n=2,726) against 2021–2025 (n=3,665). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Tumor Microenvironment1.21%10.18%8.41×373 papers
Molecular Docking Simulation0.18%1.45%7.88×53 papers
RNA, Circular0.51%3.41%6.64×125 papers
Printing, Three-Dimensional0.18%0.98%5.36×36 papers
Polymers0.18%0.85%4.61×31 papers
SEER Program0.22%0.95%4.34×35 papers
Hydrogels0.22%0.93%4.21×34 papers
Tissue Scaffolds0.29%1.2%4.09×44 papers
Sarcoma1.1%3.68%3.35×135 papers
Immunotherapy0.88%2.92%3.32×107 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Transfection4.0%0.35%0.09×13 papers
3' Untranslated Regions3.52%0.44%0.12×16 papers
Genetic Predisposition to Disease2.27%0.33%0.14×12 papers
Neoplasm Transplantation2.27%0.33%0.14×12 papers
Neoplasm Staging5.32%0.82%0.15×30 papers
Down-Regulation7.45%1.15%0.15×42 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Bone Neoplasms70.43%86.63%1.23×3175 papers
Cell Proliferation34.34%26.28%0.77×963 papers
Gene Expression Regulation, Neoplastic27.33%19.21%0.7×704 papers
Prognosis14.82%15.55%1.05×570 papers
Apoptosis23.04%14.76%0.64×541 papers
Cell Movement18.71%13.32%0.71×488 papers
MicroRNAs19.88%12.33%0.62×452 papers
Tumor Microenvironment1.21%10.18%8.41×373 papers
Signal Transduction14.75%9.41%0.64×345 papers
Antineoplastic Agents10.71%8.84%0.83×324 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial0.4%0.4%
Review6.2%8.7%
Meta-Analysis1.9%0.8%
Case Reports0.0%1.8%

Query: Osteosarcoma[MeSH Major Topic] NOT ("Sarcoma, Ewing"[MeSH] OR "Chondrosarcoma"[MeSH] OR "Giant Cell Tumor of Bone"[MeSH] OR "Dogs"[MeSH] OR "Dog Diseases"[MeSH] OR "Osteoblasts"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-16.

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
New cases each year1,000 cases/yeardirect
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
Incidence rate1.1 cases per 100,000 per yearproxycounts bone and joint cancer, which is broader than this disease; 2019-2023
Death rate0.5 deaths per 100,000 per yearproxycounts bone and joint cancer, which is broader than this disease; 2020-2024
People living with it65,261 people living with the diseaseproxycounts bone and joint cancer, which is broader than this disease; 2023
Deaths each year—not publishedNot published on the page; SEER reports deaths for bone and joint cancer as a whole.
Five-year relative survival—not publishedThe American Cancer Society publishes five-year relative survival by SEER stage, not one figure; an average would be a derivation the source does not make.
Median age at diagnosis—not publishedNot published as a median: "most osteosarcomas occur in children, teens, and young adults between the ages of 10 and 30", and "about 1 in 5 osteosarcomas occur in people older than 60".

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 is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming osteosarcoma, after removing the 2,869 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.

$21.1MNIH obligations, FY2025from $5.5M in FY2013 · +287%
32distinct projects funded16 in FY2013
$21.1Mpeak year was FY2025obligations, all institutes
94%of FY2025 awards from NCI30 of 32

NIH obligations by fiscal year

$5M$11M$16M$21M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$5.5M1816232
FY2014$4.7M1514225
FY2015$5.5M1716214
FY2016$5.9M1919210
FY2017$7.9M2724217
FY2018$7.3M2521237
FY2019$7.2M1918204
FY2020$7.1M1919206
FY2021$13.6M2826213
FY2022$13.5M2625217
FY2023$18.8M2827248
FY2024$17.6M3030235
FY2025$21.1M3232211

Where FY2025 money went

InstitutionObligationsAwards
Division Of Basic Sciences - Nci$6.1M4
Division Of Cancer Epidemiology And Genetics$2.4M0
University Of California Los Angeles$2.1M0
Stanford University$1.3M2
University Of Florida$1.2M0
Virginia Polytechnic Inst And St Univ$1.1M3
Dana-Farber Cancer Inst$1.1M2
University Of Tx Md Anderson Can Ctr$0.9M3
Johns Hopkins University$0.7M1
Albert Einstein College Of Medicine$0.7M1

Text search osteosarcoma 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 2026-09-19.

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

NCI30 · 94%
OD2 · 6%

Projects by administering institute. The rows above are the top 2 and account for 32 of 32.

Award mechanisms

R0111 · 34%
ZIA5 · 16%
K012 · 6%
R372 · 6%
F322 · 6%
R212 · 6%

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 13 and account for 32 of 32.

Where it lands

Division Of Basic Sciences - Nci$6.1M · 29.0%
Division Of Cancer Epidemiology And Gene$2.4M · 11.2%
University Of California Los Angeles$2.1M · 9.9%
Stanford University$1.3M · 6.1%
University Of Florida$1.2M · 5.7%
Virginia Polytechnic Inst And St Univ$1.1M · 5.2%

Share of $21.1M in FY2025. The top three hold 50%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

1,013 human GEO series match osteosarcoma. Keyword relevance cannot tell a 144-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 248-study ranked set

cell line 98patient 54unspecified 52mixed 41xenograft 3
98 cell line54 patient52 unspecified41 mixed3 xenograft89 carry clinical annotation74 carry survival4 patient cohorts ≥100 GEO samples5,783 GEO samples totalin 14 single-cell series the GEO sample count is the submitter’s unit (cells, libraries or pools), not patients

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE21257Genome-wide gene expression profiling on pre-chemotherapy biopsies of osteosarcoma patients who developed metastases within 5yrs (n=34) and patients who did not develop metastases within 5yrs (n=19)2011 · array5346710.2
patient cohortAPT 0.75survivalstage419 cites
GSE152048Single cell analysis of osteosarcoma tissues2020 · single-cell7616121.1
APT 0.75525 cites
GSE16091Gene expression profiles of human osteosarcoma, set22009 · array341085.7
patient cohortAPT 0.75survival229 cites
GSE162454Single-cell RNA sequencing of human osteosarcoma2021 · single-cell61858.9
APT 0.75157 cites
GSE99671Whole transcriptome analysis identifies differentially regulated networks between osteosarcoma and normal bone samples2017 · sequencing51923.7
mixedAPT 0.7599 cites
GSE33383Identification of osteosarcoma driver genes by integrative analysis of copy number and gene expression data2012 · array131752.8
mixedAPT 0.5survivalstage116 cites
GSE65071Plasma profiling of miRNAs in human specimens from osteosarcoma patients.2015 · other35632.3
patient cohortAPT 0.5survival67 cites
GSE39058microRNA- and mRNA-based studies in paraffin-archived human osteosarcoma specimens reveal profiles with reproducible and independent prognostic value2013 · array1641222.7
APT 0.5survival90 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE218035Long-read transcriptome sequencing reveals expression characteristics of osteosarcoma2023 · sequencing36144.0
patient cohortAPT 0.2517 cites
GSE237070Single-cell RNA sequencing of human various osteosarcoma tissues2024 · single-cell966.8
patient cohortAPT 0.554 cites
GSE237033MAGEA antigens as immunotherapeutic targets for advanced osteosarcoma2024 · sequencing2362.4
mixedAPT 0.5survival17 cites
GSE253548The whole transcriptome analysis using FFPE and fresh tissue samples identifies the molecular fingerprint of osteosarcoma2024 · sequencing9043.7
mixedAPT 0.523 cites
GSE246405Systematic analyses of RNA-binding proteins uncover therapeutically promising vulnerabilities in osteosarcoma2024 · sequencing1432.2
patient cohortAPT 0.25survivalmolecularMYC15 cites
GSE270231Aberrant activation of wound healing programs within the metastatic niche facilitates lung colonization by osteosarcoma cells2024 · single-cell743.4
patient cohortAPT 0.7514 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-19. SubSeries are collapsed to one row per study by linked PMID. Of 1,013 series retrieved, 191 were dropped by the profile’s exclusion rules and 526 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.

RB1

Open Targets lists no drug against it and no trial in osteosarcoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

MYC

Open Targets lists no drug against it and no trial in osteosarcoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

CD276

5 cell-therapy trials against B7-H3, 4 active, and no approved product. The clinical activity is real and none of it has reached a label.

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. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

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 (2026-09-30), ClinicalTrials.gov (2026-09-18), openFDA (2026-09-18), NCBI GEO (2026-09-19), PubMed (2026-09-16), NIH RePORTER (2026-09-19).

Negatives stated explicitly

Where the searches find nothing for osteosarcoma — no drug, no trial, no approval — the page says so in those words rather than omitting the row, and names the source searched. An absent row is unquotable; a bounded 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

5 exclusion patterns are applied to free text before anything is ranked, because U2OS, MG-63, Saos-2 and HOS (general-purpose lines) is used as a model system in DNA-damage and cell-cycle biology (U2OS), osteoblast differentiation and biomaterials (MG-63, Saos-2), virology (HOS). What was removed and why is stated in each section rather than silently applied.

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