Target landscape
Open Targets · retrieved 2026-09-08 · weekly11 genes recurrently implicated in hepatocellular carcinoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.
Of 11 recurrently implicated genes, 8 carry any drug at all. That is a statement about these 11 gene targets, not about the disease: Hepatocellular carcinoma does have labelled therapy — 12 drugs, listed in the next section.
Two different questions
The table below asks what can be aimed at 11 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what hepatocellular carcinoma 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 |
|---|---|---|---|---|
| TERT | 1 | 1 approvedImetelstatApproved in anemia, myelodysplastic syndrome. No hepatocellular carcinoma indication appears on these drugs’ labels.No hepatocellular carcinoma trial of any of these drugs | antibody, other clinical modality, protein degrader, small molecule | — |
| TP53 | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, Teprasiran2 trials, none active | other clinical modality, protein degrader, small molecule | — |
| CTNNB1 | 1 | Phase 2Pri-724No hepatocellular carcinoma trial of any of these drugs | antibody, other clinical modality, protein degrader, small molecule | — |
| AXIN1 | 0 | No drug— | antibody, protein degrader, small molecule | — |
| ARID1A | 0 | No drug— | protein degrader | — |
| MET | 36 | 6 approvedAmivantamab, Cabozantinib, Cabozantinib S-Malate, Capmatinib, Crizotinib, TepotinibApproved in hepatocellular carcinoma: Cabozantinib.23 active of 60 hepatocellular carcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| FGF19 | 0 | No drug— | antibody | — |
| FGFR4 | 16 | 5 approvedErdafitinib, Futibatinib, Infigratinib, Nintedanib, Nintedanib EsylateApproved in urothelial carcinoma, urinary bladder carcinoma, biliary tract cancer and 7 other indications. No hepatocellular carcinoma indication appears on these drugs’ labels.2 active of 32 hepatocellular carcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| KDR | 70 | 19 approvedAxitinib, Cabozantinib, Cabozantinib S-Malate, Cediranib, Fruquintinib, Lenvatinib, Midostaurin, Nintedanib, Nintedanib Esylate, Pazopanib, Ramucirumab, Regorafenib, Rivoceranib, Sorafenib, Sunitinib, Sunitinib Malate, Surufatinib, Tivozanib, VandetanibApproved in hepatocellular carcinoma: Cabozantinib, Lenvatinib, Ramucirumab, Regorafenib, Sorafenib.158 active of 566 hepatocellular carcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| VEGFA | 13 | 8 approvedAbicipar Pegol, Aflibercept, Bevacizumab, Bevacizumab Gamma, Brolucizumab, Faricimab, Pegaptanib, RanibizumabApproved in hepatocellular carcinoma: Bevacizumab.105 active of 178 hepatocellular carcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| GPC3 | 1 | Phase 2Codrituzumab3 trials, none active | antibody, protein degrader | 20 active of 39 trials |
Dataset evidence counts studies in the 307-study ranked set whose title or abstract names the gene; the bar is scaled to CTNNB1. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-08. 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 · weekly12 drugs carry an FDA label naming hepatocellular carcinoma: Atezolizumab, Bevacizumab, Cabozantinib, Durvalumab, Ipilimumab, Lenvatinib, Nivolumab, Pembrolizumab, Ramucirumab, Regorafenib, Sorafenib, Tremelimumab. Separately, 29 of the drugs returned for the genes in the table above are approved only for other diseases and reach hepatocellular carcinoma through trials, not through their labels.
Every label that names hepatocellular carcinoma
| Drug | Role | What the label says |
|---|---|---|
| AtezolizumabTECENTRIQ, Tecentriq Hybreza | Labelled here | Hepatocellular Carcinoma (HCC) in combination with bevacizumab for the treatment of adult patients with unresectable or metastatic HCC who have not received prior systemic therapy. |
| BevacizumabAvastin | Labelled here | Hepatocellular Carcinoma (HCC) in combination with atezolizumab for the treatment of patients with unresectable or metastatic HCC who have not received prior systemic therapy |
| CabozantinibCABOMETYX | Labelled here | Hepatocellular Carcinoma CABOMETYX is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. |
| DurvalumabIMFINZI | Labelled here | Hepatocellular Carcinoma IMFINZI, in combination with tremelimumab-actl, is indicated for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC). |
| IpilimumabYERVOY | Labelled here | Hepatocellular Carcinoma • adult patients with unresectable or metastatic hepatocellular carcinoma (HCC) as first-line treatment in combination with nivolumab. |
| LenvatinibLenvima | Labelled here | Hepatocellular Carcinoma (HCC) For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC). |
| NivolumabOPDIVO, OPDIVO QVANTIG | Labelled here | Hepatocellular Carcinoma (HCC) • adult patients with unresectable or metastatic hepatocellular carcinoma (HCC), as a first-line treatment in combination with ipilimumab. |
| PembrolizumabKEYTRUDA, KEYTRUDA QLEX | Labelled here | Hepatocellular Carcinoma KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) secondary to hepatitis B who have received prior systemic therapy other than a PD-1/PD-L1-containing regimen. |
| RamucirumabCYRAMZA | Labelled here | Hepatocellular Carcinoma CYRAMZA, as a single agent, is indicated for the treatment of adults with hepatocellular carcinoma (HCC) who have an alpha fetoprotein (AFP) of ≥400 ng/mL and have been treated with sorafenib. |
| RegorafenibStivarga | Labelled here | Hepatocellular Carcinoma STIVARGA is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. |
| SorafenibNexavar, Sorafenib, Sorafenib Tosylate | Labelled here | Hepatocellular Carcinoma NEXAVAR ® is indicated for the treatment of patients with unresectable hepatocellular carcinoma (HCC). |
| TremelimumabIMJUDO | Labelled here | Hepatocellular Carcinoma IMJUDO, in combination with durvalumab, is indicated for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC). |
| Ethiodized OilLIPIODOL | Not a therapy | Lipiodol is an oil-based radio-opaque contrast agent indicated for: hysterosalpingography in adults lymphography in adult and pediatric patients selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma (HCC) Lipiodol is an oil-based radiopaque contrast agent indicated for: hysterosalpingography in adults lymphography in adult and pediatric patients s... |
24 labels match indications_and_usage:"hepatocellular carcinoma"; they collapse to 13 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Diagnostic and contrast agents (Ethiodized Oil) are listed but are not treatments. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-08. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy against GPC3
ClinicalTrials.gov · retrieved 2026-09-08 · weeklyGPC3 is the busiest cell-therapy antigen in hepatocellular carcinoma: 39 registered trials, 20 still active, 2 withdrawn before enrolling anyone.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT05620706 | no phase | Recruiting | Study of GPC-3 CAR-T Cells in Treating With Hepatocellular Carcinoma | 2022-11-22 |
| NCT05926726 | no phase | Recruiting | GPC3-directed CAR-T in the Treatment Amongst Subjects With Advanced Hepatocellular Carcinoma | 2023-07-03 |
| NCT05652920 | 1/2 | Recruiting | Ori-C101Chimeric Antigen Receptor (CAR) Modified T Cells for the Treatment of HCC | 2024-04-03 |
| NCT03198546 | 1 | Recruiting | GPC3-CAR-T Cells for Immunotherapy of Cancer With GPC3 Expression | 2024-06-25 |
| NCT06560827 | 1 | Recruiting | CT011 Autologous CAR-T Cells in Patients With Hepatocellular Carcinoma at Risk of Recurrence After Surgical Resection | 2024-08-19 |
| NCT06461624 | 1 | Recruiting | Clinical Trial of Autologous GPC3 CAR-T Cells (CBG166) Therapy for Advanced Hepatocellular Carcinoma | 2024-11-06 |
| NCT06902389 | 1/2 | Recruiting | A Clinical Study of Multi-target Hi-TCR-T Cells in the Treatment of Advanced Hepatocellular Carcinoma | 2025-09-03 |
| NCT06198296 | 1 | Recruiting | Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells | 2025-11-03 |
| NCT05155189 | 1 | Recruiting | A Study to Evaluate Safety and Efficacy of Armored CAR-T Cell Injection C-CAR031 in Advanced Hepatocellular Carcinoma | 2026-01-21 |
| NCT07148050 | 1 | Recruiting | Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells | 2026-02-17 |
10 of 39 shown, most recently active first. Other antigens searched: AFP (6), CD147 (3), MUC1 (3), EpCAM (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-08. 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 · monthlyEvery term below is a share of the hepatocellular carcinoma literature, not a count, because the field itself grew: 2015–2018 (n=6,000) against 2021–2025 (n=6,000). A topic whose papers doubled while the field doubled has not risen.
These are sample shares. The two windows hold 15,942 and 26,842 papers; MeSH terms were read from an evenly spaced sample of 6,000 and 6,000 of them, taken across the whole window rather than from its most recent papers. Percentages carry sampling error of roughly a percentage point and small differences between two terms are not meaningful.
Rising, with a real baseline
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Machine Learning | 0.08% | 2.35% | 28.17× | 141 papers |
| Bevacizumab | 0.1% | 2.82% | 28.14× | 169 papers |
| Molecular Docking Simulation | 0.12% | 1.98% | 16.99× | 119 papers |
| Antibodies, Monoclonal, Humanized | 0.28% | 4.22% | 14.88× | 253 papers |
| Quinolines | 0.28% | 4.0% | 14.11× | 240 papers |
| Tumor Microenvironment | 1.45% | 11.5% | 7.93× | 690 papers |
| Gastrointestinal Microbiome | 0.15% | 0.98% | 6.55× | 59 papers |
| Immunotherapy | 0.97% | 5.8% | 6.0× | 348 papers |
| Nomograms | 0.45% | 2.63% | 5.85× | 158 papers |
| Progression-Free Survival | 0.33% | 1.7% | 5.1× | 102 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Niacinamide | 3.38% | 0.25% | 0.07× | 15 papers |
| Survival Analysis | 5.28% | 0.42% | 0.08× | 25 papers |
| Immunohistochemistry | 3.02% | 0.33% | 0.11× | 20 papers |
| Gene Knockdown Techniques | 1.93% | 0.23% | 0.12× | 14 papers |
| Neoplasm Grading | 1.63% | 0.25% | 0.15× | 15 papers |
| Patient Selection | 1.52% | 0.25% | 0.16× | 15 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Liver Neoplasms | 97.23% | 99.77% | 1.03× | 5986 papers |
| Prognosis | 16.85% | 20.43% | 1.21× | 1226 papers |
| Gene Expression Regulation, Neoplastic | 18.02% | 19.85% | 1.1× | 1191 papers |
| Cell Proliferation | 17.45% | 16.3% | 0.93× | 978 papers |
| Biomarkers, Tumor | 11.73% | 12.92% | 1.1× | 775 papers |
| Treatment Outcome | 15.05% | 11.75% | 0.78× | 705 papers |
| Tumor Microenvironment | 1.45% | 11.5% | 7.93× | 690 papers |
| Antineoplastic Agents | 8.98% | 8.97% | 1.0× | 538 papers |
| Mice, Nude | 7.13% | 8.82% | 1.24× | 529 papers |
| Signal Transduction | 10.92% | 8.58% | 0.79× | 515 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Clinical Trial | 0.0% | 0.1% |
| Randomized Controlled Trial | 1.0% | 0.8% |
| Review | 8.1% | 9.9% |
| Meta-Analysis | 2.4% | 2.1% |
| Case Reports | 0.0% | 1.5% |
Query: Carcinoma, Hepatocellular[MeSH Major Topic] NOT ("Cholangiocarcinoma"[MeSH] OR "Bile Duct Neoplasms"[MeSH] OR "Hepatoblastoma"[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. Windows are read whole where they fit and sampled where they do not; the totals and the sample sizes are both in the JSON beside this page. Source: PubMed MeSH, retrieved 2026-09-08.
Funding
NIH RePORTER · quarterlyNIH obligations naming hepatocellular carcinoma, after removing the 241 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 | $154.4M | 430 | 392 | 17 |
| FY2014 | $150.7M | 415 | 381 | 19 |
| FY2015 | $156.7M | 418 | 371 | 13 |
| FY2016 | $158.1M | 423 | 388 | 18 |
| FY2017 | $178.7M | 451 | 401 | 15 |
| FY2018 | $224.1M | 559 | 471 | 10 |
| FY2019 | $240.2M | 600 | 517 | 18 |
| FY2020 | $232.6M | 577 | 514 | 19 |
| FY2021 | $244.5M | 598 | 530 | 18 |
| FY2022 | $265.4M | 637 | 564 | 21 |
| FY2023 | $281.7M | 640 | 573 | 19 |
| FY2024 | $268.8M | 614 | 539 | 23 |
| FY2025 | $263.2M | 532 | 463 | 31 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Ut Southwestern Medical Center | $15.0M | 23 |
| Division Of Basic Sciences - Nci | $11.5M | 11 |
| Sanford Burnham Prebys Medical Discovery Institute | $11.0M | 17 |
| Johns Hopkins University | $9.8M | 22 |
| Baylor College Of Medicine | $7.6M | 16 |
| North Carolina State University Raleigh | $7.4M | 0 |
| Virginia Commonwealth University | $7.0M | 14 |
| University Of Pennsylvania | $6.6M | 14 |
| Northwestern University | $6.2M | 0 |
| University Of California Los Angeles | $6.0M | 11 |
Text search hepatocellular carcinoma 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 2026-09-08.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly4,594 human GEO series match hepatocellular carcinoma. Keyword relevance cannot tell a 2,606-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 307-study ranked set
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE76427 | Microarray expression data for tumor and adjacent non-tumor tissues from hepatocellular carcinoma patients2017 · array | 167 | 825 | 6.0 | patient cohortAPT 0.75survivalstagemolecular187 cites |
| GSE109211 | Molecular predictors of prevention of recurrence in hepatocellular carcinoma with sorafenib as adjuvant treatment in the phase 3 STORM trial2018 · array | 140 | 216 | 9.5 | patient cohortAPT 0.75survivalstagemolecularVEGFA268 cites |
| GSE63898 | DNA methylation-based prognosis and epidrivers in hepatocellular carcinoma2015 · array | 396 | 147 | 11.8 | patient cohortAPT 0.95survivalmolecular300 cites |
| GSE112790 | Comprehensive molecular characterization of liver cancer and inheritance of the phenotypic traits during tumor recurrence [tissue]2019 · array | 198 | 156 | 7.2 | patient cohortAPT 0.75survivalmolecularCTNNB1197 cites |
| GSE164760 | Molecular characterization of hepatocellular carcinoma in patients with non-alcoholic steatohepatitis2021 · array | 170 | 117 | 10.6 | patient cohortAPT 0.75molecularCTNNB1TERTTP53199 cites |
| GSE112679 | Genome-wide Mapping of 5-Hydroxymethylcytosines in Circulating Cell-free DNA as a Non-invasive Approach for Early Detection of Hepatocellular Carcinoma2019 · methylation | 2,606 | 7 | 8.5 | patient cohortAPT 0.95survivalstagemolecularAFP226 cites |
| GSE64041 | Gene expression profiling in paired human hepatocellular carcinoma and liver parenchyma biopsies and normal liver biopsies.2016 · array | 125 | 226 | 2.0 | patient cohortAPT 0.5survivalstage65 cites |
| GSE121248 | Gene expression profiling of chronic hepatitis B induced HCC and adjacent-normal tissues2018 · array | 107 | 302 | 3.1 | APT 0.75molecular152 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE193066 | Transcriptome profile of liver biopsy tissues from patients with non-alcoholic fatty liver disease (tissue validation set 1).2022 · sequencing | 164 | 16 | 6.4 | patient cohortAPT 0.95survivalmolecular98 cites |
| GSE174570 | Cabozantinib enhances anti-PD1 efficacy and elicits a neutrophil-based immune response in murine models: implications for human HCC2022 · array | 114 | 18 | 5.0 | patient cohortAPT 0.75survivalmolecular75 cites |
| GSE190967 | Multi-omics identifies the link between intratumor steatosis and exhausted tumor immune microenvironment in hepatocellular carcinoma2022 · sequencing | 113 | 5 | 11.9 | patient cohortAPT 0.75survival141 cites |
| GSE214846 | A novel risk score based on immune-related genes for hepatocellular carcinoma as a reliable prognostic biomarker and correlated with immune infiltration2022 · sequencing | 130 | 29 | 1.4 | patient cohortAPT 0.25survival22 cites |
| GSE242154 | Spatial immuno-oncology protein abundance profiles in hepatocellular carcinoma tissues from patients treated with bavituximab and pembrolizumab2024 · spatial | 251 | 1 | 4.2 | patient cohortAPT 0.75survivalstage36 cites |
| GSE243245 | Crosstalk between myeloid and B cells shapes the distinct microenvironments of primary and secondary liver cancer2023 · single-cell | 123 | 2 | 3.8 | APT 0.75survivalmolecular52 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-08. SubSeries are collapsed to one row per study by linked PMID. Of 723 series retrieved, 12 were dropped by the profile’s exclusion rules and 356 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.
TERT
1 approved drug (Imetelstat) and no registered trial in hepatocellular carcinoma. The molecules exist; nobody has tested them here.
AXIN1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
ARID1A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
FGF19
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
GPC3
39 cell-therapy trials against GPC3, 20 active, and no approved product. The clinical activity is real and none of it has reached a label.
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 (2026-09-08), ClinicalTrials.gov (2026-09-08), openFDA (2026-09-08), NCBI GEO (2026-09-08), PubMed (2026-09-08), NIH RePORTER (2026-09-08).
Negatives stated explicitly
Where nothing exists for hepatocellular carcinoma — 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 HepG2 and Huh7 is used as a model system in hepatotoxicity screening, drug metabolism, and HCV virology. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Hepatocellular carcinoma",
"mesh": "Carcinoma, Hepatocellular",
"facts": "https://usebiotransfer.org/disease/hepatocellular-carcinoma.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."
}