Disease Briefing

Uveal melanoma: 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 2026-09-17Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 110 studies · 2,293 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in uveal melanoma — GNAQ, GNA11, BAP1, SF3B1, EIF1AX, CYSLTR2, PLCB4, PMEL, MET, MITF, PRKCA, MBD4 — 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 uveal melanoma: Tebentafusp. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
1
of the 12 genes above carry a drug that is approved in uveal melanoma itself — PMEL. Across all of them 46 drug entries reach these genes, 44 distinct once salt forms are merged
2
registered liver-directed cell-therapy trials in uveal melanoma, 1 active. Counted from ClinicalTrials.gov across 5 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
9
targets carry an Open Targets tractability signal and have no clinical programme of any kind: GNAQ, GNA11, BAP1, SF3B1, EIF1AX, CYSLTR2, PLCB4, MITF, MBD4
178
human GEO series match the disease; 110 survive on-topic filtering, and only 0 are patient cohorts of 100+ samples
173
Europe PMC full-text papers name GSE22138 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$0.0M
NIH obligations in FY2025, across 0 distinct core projects — and none in FY2013: no project matched the disease that year. There is no growth rate to give from a base of zero

Target landscape

Open Targets · retrieved 2026-09-17 · weekly

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

Of 12 recurrently implicated genes, 3 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Uveal melanoma 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 uveal melanoma 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
GNAQ 0 No drug antibody, protein degrader, small molecule
GNA11 0 No drug antibody, protein degrader, small molecule
BAP1 across cancers → 0 No drug protein degrader
SF3B1 across cancers → 0 No drug protein degrader, small molecule
EIF1AX 0 No drug protein degrader, small molecule
CYSLTR2 0 No drug antibody, protein degrader, small molecule
PLCB4 0 No drug antibody, protein degrader
PMEL across cancers → 2 1 approvedTebentafuspApproved in uveal melanoma: Tebentafusp.12 active of 14 uveal melanoma trials antibody, other clinical modality
MET across cancers → 36 6 approvedAmivantamab, Cabozantinib, Cabozantinib S-Malate, Capmatinib, Crizotinib, TepotinibApproved in non-small cell lung carcinoma, thyroid cancer, thyroid tumor and 3 other indications. No uveal melanoma indication appears on these drugs’ labels.3 active of 6 uveal melanoma trials antibody, other clinical modality, protein degrader, small molecule
MITF across cancers → 0 No drug protein degrader, small molecule
PRKCA 6 1 approvedMidostaurinApproved in mast cell leukemia, acute myeloid leukemia by FAB classification, systemic mastocytosis. No uveal melanoma indication appears on these drugs’ labels.1 active of 4 uveal melanoma trials antibody, protein degrader, small molecule
MBD4 0 No drug protein degrader

Dataset evidence counts studies in the 110-study ranked set whose title or abstract names the gene; the bar is scaled to BAP1. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-17. 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 uveal melanoma: Tebentafusp. Separately, 7 of the drugs returned for the genes in the table above are approved only for other diseases and reach uveal melanoma through trials, not through their labels.

1Labelled for uveal melanomaFDA INDICATIONS AND USAGE names the disease
7Approved, but for another diseasereturned for the genes in the table above
1Backbone agents listing itbroad cytotoxics whose labels name many tumours
1Active liver-directed cell-therapy trialsof 2 registered

Every label that names uveal melanoma

DrugRoleWhat the label says
TebentafuspKIMMTRAKLabelled hereKIMMTRAK is indicated for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma.
MelphalanHepzato KitBackboneHEPZATO is an alkylating drug indicated as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.( 1 )

1 labels match indications_and_usage:"uveal melanoma" OR indications_and_usage:"metastatic uveal melanoma"; they collapse to 2 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-17. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against liver-directed

ClinicalTrials.gov · retrieved 2026-09-17 · weekly

liver-directed is the busiest cell-therapy antigen in uveal melanoma: 2 registered trials, 1 still active.

TrialPhaseStatusTitleLast update
NCT071838521Not yet recruitingLocoregional or Systemic Administration of Autologous Tumor Infiltrating Lymphocytes in Patients With Metastatic Melanoma2026-09-09
NCT048124701CompletedHepatic Arterial Infusion of Autologous Tumor Infiltrating Lymphocytes in Patients With Melanoma and Liver Metastases2025-09-22

2 of 2 shown, most recently active first. Other antigens searched: CTLA-4 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-17. 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

Every term below is a share of the uveal melanoma literature, not a count, because the field itself grew: 2015–2018 (n=833) against 2021–2025 (n=1,146). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Tumor Microenvironment0.72%6.2%8.6×71 papers
Computational Biology0.6%1.13%1.89×13 papers
Mice, Nude1.92%2.79%1.45×32 papers
Nevus, Pigmented2.04%2.97%1.45×34 papers
Nivolumab0.96%1.4%1.45×16 papers
Biomarkers, Tumor8.28%10.91%1.32×125 papers
Uveal Melanoma67.11%82.98%1.24×951 papers
Prognosis16.09%19.46%1.21×223 papers
Apoptosis2.64%3.14%1.19×36 papers
GTP-Binding Protein alpha Subunits, Gq-G113.6%4.28%1.19×49 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Time Factors6.12%1.48%0.24×17 papers
Neoplasm Staging7.2%1.92%0.27×22 papers
Diagnosis, Differential6.84%1.92%0.28×22 papers
Visual Acuity12.24%3.58%0.29×41 papers
Biopsy, Fine-Needle3.6%1.13%0.31×13 papers
Kaplan-Meier Estimate3.6%1.13%0.31×13 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Melanoma99.28%98.43%0.99×1128 papers
Uveal Melanoma67.11%82.98%1.24×951 papers
Prognosis16.09%19.46%1.21×223 papers
Choroid Neoplasms26.29%17.36%0.66×199 papers
Biomarkers, Tumor8.28%10.91%1.32×125 papers
Brachytherapy17.53%9.42%0.54×108 papers
Gene Expression Regulation, Neoplastic9.12%9.16%1.0×105 papers
Skin Neoplasms6.96%7.85%1.13×90 papers
Follow-Up Studies14.17%7.85%0.55×90 papers
Mutation6.36%7.24%1.14×83 papers

Publication mix

Type2015–182021–25
Clinical Trial0.0%0.2%
Randomized Controlled Trial1.2%0.6%
Review10.9%9.3%
Meta-Analysis0.2%1.7%
Case Reports0.0%2.4%

Query: ("Uveal Neoplasms"[Majr] AND "Melanoma"[Mesh]) NOT ("Retinoblastoma"[MeSH] OR "Conjunctival Neoplasms"[MeSH] OR "Eyelid Neoplasms"[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 2026-09-17.

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 year3,200 cases/yearproxycounts eye and orbit cancer, which is broader than this disease; 2026
Deaths each year530 deaths/yearproxycounts eye and orbit cancer, which is broader than this disease; 2026
New cases each yearnot publishedNot published for uveal melanoma alone; the eye and orbit figure is "mainly melanomas" and no share is given.
Deaths each yearnot publishedNot published for the disease alone.
Five-year relative survivalnot publishedThe American Cancer Society publishes eye-cancer survival by SEER stage, not one figure for uveal melanoma.
Median age at diagnosisnot publishedNot published; "the risk for most other types of eye cancer increases as people get older".

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 uveal melanoma, after removing the 14 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.

$0.2MNIH obligations, FY2021from $0.3M in FY2017 · -17%
1distinct projects funded1 in FY2017
$0.3Mpeak year was FY2017obligations, all institutes
100%of FY2021 awards from NEI1 of 1

NIH obligations by fiscal year

$0M$0M$0M$0M171921
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2017$0.3M110
FY2018$0.2M110
FY2019$0.2M110
FY2020$0.2M111
FY2021$0.2M111

Where FY2021 money went

InstitutionObligationsAwards
University Of California, San Francisco$0.2M1

Text search uveal melanoma OR ocular melanoma OR choroidal melanoma 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 2026-09-17.

Who funds it, FY2021

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

NIH institutes

NEI1 · 100%

Projects by administering institute. The rows above are the top 1 and account for 1 of 1.

Award mechanisms

K231 · 100%

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 1 and account for 1 of 1.

Where it lands

University Of California, San Francisco$0.2M · 100.0%

Share of $0.2M in FY2021. The top three hold 100%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

178 human GEO series match uveal melanoma. Keyword relevance cannot tell a 63-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 110-study ranked set

unspecified 44cell line 36patient 21mixed 8xenograft 1
44 unspecified36 cell line21 patient8 mixed1 xenograft69 carry clinical annotation28 carry survival0 patient cohorts ≥100 GEO samples2,293 GEO samples totalin 5 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE22138Expression Data from Uveal Melanoma primary tumors.2010 · array631733.3
patient cohortAPT 0.5survivalmolecular141 cites
GSE139829Single-cell analysis reveals new evolutionary complexity in uveal melanoma2020 · single-cell1110613.7
APT 0.75molecularCTLA4LAG3369 cites
GSE84976Upregulation of HLA expression in primary Uveal Melanoma by infiltrating leukocytes2016 · array28752.3
patient cohortAPT 0.5survivalmolecular69 cites
GSE27831Syntenin-1 is expressed in uveal melanoma and correlates with metastatic progression2012 · array29322.0
patient cohortAPT 0.5survivalmolecular72 cites
GSE73652Gene expression analysis of uveal melanoma Class 1 tumors with and without metastasis2015 · array1398.5
APT 0.95molecularBAP1236 cites
GSE137675Analysis of gene expression, motif and pattern of m6A modified region and m6A enrichment level in Ocular melanoma and Melanocyte cell lines. Gene expressin analysis of METTL3 knock down in PIG1, ALKBH5 knock down and HINT2 over expression in OCM1.2019 · sequencing4277.0
cell lineAPT 0.25survivalmolecular103 cites
GSE138665Single-cell RNA-seq reveals intratumoral heterogeneity in primary uveal melanomas2021 · chromatin18174.2
APT 0.7579 cites
GSE162739BAP1 activity regulates Polycomb occupancy and global chromatin condensation counteracting diffuse PCGF3/5-dependent H2AK119ub1 deposition2021 · chromatin19224.1
APT 0.25molecularBAP188 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE259383Tebentafusp elicits on-target cutaneous immune responses driven by cytotoxic T cells in uveal melanoma patients2025 · single-cell612.9
patient cohortAPT 0.75survivalmolecularGP100LAG3TEBENTAFUSP11 cites
GSE212219Cerivastatin synergizes with trametinib and enhances its efficacy in the therapy of Uveal Melanoma2022 · array1221.4
mixedAPT 0.25survivalmolecularBAP1EIF1AXGNA1112 cites
GSE197656In uveal melanoma Gα-protein GNA11 mutations convey a shorter disease-specific survival and are more strongly associated with loss of BAP1 and chromosomal alterations than Gα-protein GNAQ mutations2023 · array812.1
patient cohortAPT 0.5survivalstagemolecularBAP1GNA11GNAQ26 cites
GSE202687Pilot Trial of Arginine Deprivation plus Nivolumab and Ipilimumab in Patients with Metastatic Uveal Melanoma2022 · sequencing1521.3
patient cohortAPT 0.5stagemolecularIPILIMUMABNIVOLUMAB19 cites
GSE248838Divergent local and systemic antitumor response in primary uveal melanomas2024 · single-cell1511.3
APT 0.75survivalstagemolecularPD-111 cites
GSE287090Exploration of tumor associated macrophages in patients with metastatic uveal melanoma by single cell RNAseq2025 · sequencing1213.9
patient cohortAPT 0.75molecular15 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-17. SubSeries are collapsed to one row per study by linked PMID. Of 178 series retrieved, 10 were dropped by the profile’s exclusion rules and 27 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.

GNAQ

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

GNA11

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

BAP1

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

SF3B1

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

EIF1AX

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

CYSLTR2

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

PLCB4

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

MITF

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

MBD4

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. 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 (2026-09-17), ClinicalTrials.gov (2026-09-17), openFDA (2026-09-17), NCBI GEO (2026-09-17), PubMed (2026-09-17), NIH RePORTER (2026-09-17).

Negatives stated explicitly

Where nothing exists for uveal melanoma — 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

5 exclusion patterns are applied to free text before anything is ranked, because 92.1 (unlistable) is used as a model system in "92.1" is a number. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Uveal melanoma",
  "mesh": "Uveal Neoplasms",
  "facts": "https://usebiotransfer.org/disease/uveal-melanoma.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."
}