Target landscape
Open Targets · retrieved 2026-09-12 · weekly12 genes recurrently implicated in anal cancer, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.
Of 12 recurrently implicated genes, 6 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Anal cancer 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 anal cancer 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 |
|---|---|---|---|---|
| CD274 across cancers → | 13 | 5 approvedAtezolizumab, Avelumab, Cosibelimab, Durvalumab, SugemalimabApproved in urothelial carcinoma, non-small cell lung carcinoma, breast cancer and 6 other indications. No anal cancer indication appears on these drugs’ labels.6 active of 16 anal cancer trials | antibody, other clinical modality, protein degrader, small molecule | — |
| PDCD1 across cancers → | 26 | 9 approvedCemiplimab, Dostarlimab, Nivolumab, Pembrolizumab, Retifanlimab, Serplulimab, Sintilimab, Tislelizumab, ToripalimabApproved in anal cancer: Retifanlimab.25 active of 43 anal cancer trials | antibody, other clinical modality, protein degrader, small molecule | 0 active of 2 trials |
| PIK3CA across cancers → | 31 | 3 approvedAlpelisib, Copanlisib, InavolisibApproved in breast cancer, breast neoplasm, breast carcinoma and 3 other indications. No anal cancer indication appears on these drugs’ labels.No anal cancer trial of any of these drugs | antibody, protein degrader, small molecule | — |
| CDKN2A across cancers → | 0 | No drug— | — | — |
| TP53 across cancers → | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo anal cancer trial of any of these drugs | other clinical modality, protein degrader, small molecule | — |
| EGFR across cancers → | 74 | 23 approvedAfatinib, Afatinib Dimaleate, Amivantamab, Aumolertinib, Brigatinib, Cetuximab, Cetuximab Sarotalocan, Dacomitinib, Erlotinib, Gefitinib, Icotinib, Lapatinib, Lapatinib Ditosylate, Lazertinib, Mobocertinib, Necitumumab, Neratinib, Nimotuzumab, Olmutinib, Osimertinib, Panitumumab, Rociletinib, VandetanibApproved in non-small cell lung carcinoma, anaplastic large cell lymphoma, malignant tumor of neck and 14 other indications. No anal cancer indication appears on these drugs’ labels.12 trials, none active | antibody, other clinical modality, protein degrader, small molecule | — |
| KMT2D across cancers → | 0 | No drug— | antibody, protein degrader, small molecule | — |
| FBXW7 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| PTEN across cancers → | 0 | No drug— | antibody, protein degrader, small molecule | — |
| SOX2 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| TERT across cancers → | 1 | 1 approvedImetelstatApproved in anemia, myelodysplastic syndrome. No anal cancer indication appears on these drugs’ labels.No anal cancer trial of any of these drugs | antibody, other clinical modality, protein degrader, small molecule | — |
| MYC across cancers → | 0 | No drug— | protein degrader, small molecule | — |
Dataset evidence counts studies in the 3-study ranked set whose title or abstract names the gene; the bar is scaled to PIK3CA. 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 anal cancer: Retifanlimab. Separately, 40 of the drugs returned for the genes in the table above are approved only for other diseases and reach anal cancer through trials, not through their labels.
Every label that names anal cancer
| Drug | Role | What the label says |
|---|---|---|
| RetifanlimabZYNYZ | Labelled here | Squamous Cell Carcinoma of the Anal Canal ZYNYZ, in combination with carboplatin and paclitaxel, is indicated for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). |
0 labels match indications_and_usage:"anal cancer" OR indications_and_usage:"anal canal cancer" OR indications_and_usage:"squamous cell carcinoma of the anal canal" OR indications_and_usage:"anal squamous" OR indications_and_usage:"carcinoma of the anus"; 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 HPV E6/E7
ClinicalTrials.gov · retrieved 2026-09-12 · weeklyHPV E6/E7 is the busiest cell-therapy antigen in anal cancer: 6 registered trials, 4 still active.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT05639972 | 1/2 | Recruiting | E7 T-cell Receptor (TCR) -T Cell Induction Therapy for Locoregionally Advanced HPV-associated Cancers | 2026-06-12 |
| NCT05686226 | 2 | Recruiting | E7 TCR-T Cell Immunotherapy for Human Papillomavirus (HPV) Associated Cancers | 2026-06-12 |
| NCT05973487 | 1 | Active | A Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors | 2025-11-17 |
| NCT06358053 | 1 | Not yet recruiting | CRTE7A2-01 TCR-T Cells for HPV-16 Positive Advanced Cancers | 2024-04-10 |
| NCT02858310 | 1/2 | Completed | E7 TCR T Cells for Human Papillomavirus-Associated Cancers | 2026-03-09 |
| NCT02379520 | 1 | Completed | HPV-16/18 E6/E7-Specific T Lymphocytes, Relapsed HPV-Associated Cancers, HESTIA | 2026-04-15 |
6 of 6 shown, most recently active first. Other antigens searched: PD-1 (2). 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 · monthlyEvery term below is a share of the anal cancer literature, not a count, because the field itself grew: 2015–2018 (n=830) against 2021–2025 (n=1,044). A topic whose papers doubled while the field doubled has not risen.
Rising, with a real baseline
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Alphapapillomavirus | 0.72% | 3.26% | 4.5× | 34 papers |
| Proctectomy | 0.96% | 3.07% | 3.18× | 32 papers |
| Sexual and Gender Minorities | 2.65% | 7.47% | 2.82× | 78 papers |
| Laparoscopy | 0.6% | 1.15% | 1.91× | 12 papers |
| Margins of Excision | 0.6% | 1.15% | 1.91× | 12 papers |
| Rectum | 1.2% | 2.11% | 1.75× | 22 papers |
| Skin Neoplasms | 2.17% | 3.54% | 1.63× | 37 papers |
| Colorectal Neoplasms | 0.72% | 1.15% | 1.59× | 12 papers |
| Early Detection of Cancer | 7.71% | 12.16% | 1.58× | 127 papers |
| Postoperative Complications | 1.81% | 2.49% | 1.38× | 26 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Immunohistochemistry | 5.42% | 1.44% | 0.27× | 15 papers |
| Papillomavirus Vaccines | 6.63% | 2.01% | 0.3× | 21 papers |
| Combined Modality Therapy | 5.78% | 1.82% | 0.31× | 19 papers |
| Follow-Up Studies | 10.72% | 3.74% | 0.35× | 39 papers |
| Genotype | 3.49% | 1.25% | 0.36× | 13 papers |
| Disease-Free Survival | 5.78% | 2.3% | 0.4× | 24 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Carcinoma, Squamous Cell | 38.8% | 33.05% | 0.85× | 345 papers |
| Papillomavirus Infections | 23.86% | 29.5% | 1.24× | 308 papers |
| HIV Infections | 17.47% | 23.08% | 1.32× | 241 papers |
| Anal Canal | 16.75% | 22.13% | 1.32× | 231 papers |
| Chemoradiotherapy | 20.96% | 14.56% | 0.69× | 152 papers |
| Papillomaviridae | 13.98% | 13.22% | 0.95× | 138 papers |
| Early Detection of Cancer | 7.71% | 12.16% | 1.58× | 127 papers |
| Homosexuality, Male | 8.92% | 11.3% | 1.27× | 118 papers |
| Rectal Neoplasms | 8.43% | 10.44% | 1.24× | 109 papers |
| Neoplasm Recurrence, Local | 10.6% | 9.96% | 0.94× | 104 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Randomized Controlled Trial | 1.2% | 1.4% |
| Review | 13.4% | 9.0% |
| Meta-Analysis | 1.2% | 1.7% |
| Case Reports | 0.0% | 3.1% |
Query: Anus Neoplasms[MeSH Major Topic] NOT ("Anal Gland Neoplasms"[MeSH] OR "Hemorrhoids"[MeSH] OR "Fissure in Ano"[MeSH] OR "Rectal Fistula"[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 2026-09-12.
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 |
|---|---|---|---|
| New cases each year | 11,270 cases/year | direct | 2026 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| Deaths each year | 1,700 deaths/year | direct | 2026 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| Incidence rate | 2 cases per 100,000 people per year | direct | 2019-2023 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| Death rate | 0.4 deaths per 100,000 people per year | direct | 2020-2024 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| People living with it | 88,564 people living with the disease | direct | 2023 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| Median age at diagnosis | 65.0 years | direct | 2019-2023 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| median age at death | 69 years | direct | 2020-2024 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
| Five-year relative survival | 70.9% | direct | 2016-2022 SEER Cancer Stat Facts, Anal Cancer, retrieved 2026-09-12 |
Years of life lost
3.9 years per case, 43,946 a year, from survival and age at diagnosis rather than from a death count. derived proxy What this costs to fund is in Funding.
3 assumptions behind this estimate
- Five-year relative survival stands in for cure; a death after five years is not counted.
- The median age at diagnosis stands in for the whole age distribution.
- Life expectancy at birth (78.4 years) is the reference, rather than remaining expectancy at the age of death from a life table.
- Each assumption moves the result by more than the difference between the two diseases currently on this site, so this figure separates a disease from one ten times its size, not from a close neighbour.
Funding
NIH RePORTER · quarterlyNIH obligations naming anal cancer, after removing the 1,240 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 | $4.9M | 7 | 7 | 73 |
| FY2014 | $6.0M | 8 | 8 | 85 |
| FY2015 | $16.5M | 8 | 8 | 76 |
| FY2016 | $24.6M | 9 | 9 | 72 |
| FY2017 | $28.0M | 12 | 12 | 88 |
| FY2018 | $29.1M | 13 | 11 | 94 |
| FY2019 | $22.9M | 13 | 13 | 98 |
| FY2020 | $6.6M | 16 | 15 | 96 |
| FY2021 | $6.6M | 17 | 17 | 99 |
| FY2022 | $6.8M | 18 | 17 | 113 |
| FY2023 | $7.2M | 13 | 13 | 109 |
| FY2024 | $9.7M | 17 | 15 | 110 |
| FY2025 | $9.0M | 20 | 18 | 127 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Division Of Cancer Epidemiology And Genetics | $2.6M | 3 |
| Icahn School Of Medicine At Mount Sinai | $1.3M | 2 |
| University Of Maryland Baltimore | $1.0M | 3 |
| Baylor College Of Medicine | $0.9M | 1 |
| University Of Miami School Of Medicine | $0.8M | 2 |
| Emory University | $0.6M | 3 |
| Medical College Of Wisconsin | $0.6M | 1 |
| University Of California, San Francisco | $0.4M | 1 |
| University Of Arizona | $0.4M | 1 |
| University Of Tx Md Anderson Can Ctr | $0.2M | 1 |
Text search anal cancer 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.
What that buys
Against 43,946 years of life lost a year, FY2025 obligations are $206 per life-year — $803 per case. The estimate and its assumptions are in Disease burden.
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 5 and account for 20 of 20.
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 10 and account for 20 of 20.
Where it lands
Share of $9.0M in FY2025. The top three hold 55%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly75 human GEO series match anal cancer. Keyword relevance cannot tell a 98-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 3-study ranked set
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE254331 | Inflammatory pathways confer resistance to chemoradiotherapy in anal squamous cell carcinoma2024 · sequencing | 98 | 1 | 1.1 | patient cohortAPT 0.758 cites |
| GSE253560 | Transcriptome and microbiome-immune changes across preinvasive and invasive anal cancer lesions2024 · sequencing | 70 | 2 | 0.5 | APT 0.05stagemolecularPIK3CA4 cites |
| GSE293618 | Higher expression of HPV16 derived E7_LI Transcript Observed in Men with HIV and Recurrent Anal Cancer2025 · sequencing | 12 | 1 | — | patient cohortAPT 0.25survivalstage1 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. SubSeries are collapsed to one row per study by linked PMID. Of 75 series retrieved, 11 were dropped by the profile’s exclusion rules and 61 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.
PIK3CA
3 approved drugs (Alpelisib, Copanlisib, Inavolisib) and no registered trial in anal cancer. The molecules exist; nobody has tested them here.
CDKN2A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
KMT2D
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
FBXW7
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
PTEN
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
SOX2
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
TERT
1 approved drug (Imetelstat) and no registered trial in anal cancer. The molecules exist; nobody has tested them here.
MYC
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. 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 (2026-09-12), ClinicalTrials.gov (2026-09-12), openFDA (2026-09-12), NCBI GEO (2026-09-12), PubMed (2026-09-12), NIH RePORTER (2026-09-12).
Negatives stated explicitly
Where nothing exists for anal cancer — 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 none is used as a model system in none: no anal cancer cell line is in general use. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Anal cancer",
"mesh": "Anus Neoplasms",
"facts": "https://usebiotransfer.org/disease/anal-cancer.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."
}