Disease Briefing

Anal cancer: 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 2026-09-12Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 3 studies · 180 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in anal cancer — CD274, PDCD1, PIK3CA, CDKN2A, TP53, EGFR, KMT2D, FBXW7, PTEN, SOX2, TERT, MYC — 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 anal cancer: Retifanlimab. 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 anal cancer itself — PDCD1. Across all of them 164 drug entries reach these genes, 153 distinct once salt forms are merged
6
registered HPV E6/E7 cell-therapy trials in anal cancer, 4 active. Counted from ClinicalTrials.gov across 7 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
8
targets carry an Open Targets tractability signal and have no clinical programme of any kind: PIK3CA, TP53, KMT2D, FBXW7, PTEN, SOX2, TERT, MYC. PDCD1 has cell-therapy trials, so it is undrugged rather than untouched
75
human GEO series match the disease; 3 survive on-topic filtering, and only 0 are patient cohorts of 100+ samples
2
Europe PMC full-text papers name GSE253560 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$9.0M
NIH obligations in FY2025, up 83% since 2013 — while distinct core projects went 7 to 18. More distinct projects (+157%) rather than larger ones; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-12 · weekly

12 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.

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
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 · weekly

1 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.

1Labelled for anal cancerFDA INDICATIONS AND USAGE names the disease
40Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
4Active HPV E6/E7 cell-therapy trialsof 6 registered

Every label that names anal cancer

DrugRoleWhat the label says
RetifanlimabZYNYZLabelled hereSquamous 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 · weekly

HPV E6/E7 is the busiest cell-therapy antigen in anal cancer: 6 registered trials, 4 still active.

TrialPhaseStatusTitleLast update
NCT056399721/2RecruitingE7 T-cell Receptor (TCR) -T Cell Induction Therapy for Locoregionally Advanced HPV-associated Cancers2026-06-12
NCT056862262RecruitingE7 TCR-T Cell Immunotherapy for Human Papillomavirus (HPV) Associated Cancers2026-06-12
NCT059734871ActiveA Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors2025-11-17
NCT063580531Not yet recruitingCRTE7A2-01 TCR-T Cells for HPV-16 Positive Advanced Cancers2024-04-10
NCT028583101/2CompletedE7 TCR T Cells for Human Papillomavirus-Associated Cancers2026-03-09
NCT023795201CompletedHPV-16/18 E6/E7-Specific T Lymphocytes, Relapsed HPV-Associated Cancers, HESTIA2026-04-15

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 · monthly

Every 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 term2015–182021–25ChangeShare now
Alphapapillomavirus0.72%3.26%4.5×34 papers
Proctectomy0.96%3.07%3.18×32 papers
Sexual and Gender Minorities2.65%7.47%2.82×78 papers
Laparoscopy0.6%1.15%1.91×12 papers
Margins of Excision0.6%1.15%1.91×12 papers
Rectum1.2%2.11%1.75×22 papers
Skin Neoplasms2.17%3.54%1.63×37 papers
Colorectal Neoplasms0.72%1.15%1.59×12 papers
Early Detection of Cancer7.71%12.16%1.58×127 papers
Postoperative Complications1.81%2.49%1.38×26 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Immunohistochemistry5.42%1.44%0.27×15 papers
Papillomavirus Vaccines6.63%2.01%0.3×21 papers
Combined Modality Therapy5.78%1.82%0.31×19 papers
Follow-Up Studies10.72%3.74%0.35×39 papers
Genotype3.49%1.25%0.36×13 papers
Disease-Free Survival5.78%2.3%0.4×24 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Carcinoma, Squamous Cell38.8%33.05%0.85×345 papers
Papillomavirus Infections23.86%29.5%1.24×308 papers
HIV Infections17.47%23.08%1.32×241 papers
Anal Canal16.75%22.13%1.32×231 papers
Chemoradiotherapy20.96%14.56%0.69×152 papers
Papillomaviridae13.98%13.22%0.95×138 papers
Early Detection of Cancer7.71%12.16%1.58×127 papers
Homosexuality, Male8.92%11.3%1.27×118 papers
Rectal Neoplasms8.43%10.44%1.24×109 papers
Neoplasm Recurrence, Local10.6%9.96%0.94×104 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial1.2%1.4%
Review13.4%9.0%
Meta-Analysis1.2%1.7%
Case Reports0.0%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.

MeasureValueMatchWhat it counts
New cases each year11,270 cases/yeardirect2026
Deaths each year1,700 deaths/yeardirect2026
Incidence rate2 cases per 100,000 people per yeardirect2019-2023
Death rate0.4 deaths per 100,000 people per yeardirect2020-2024
People living with it88,564 people living with the diseasedirect2023
Median age at diagnosis65.0 yearsdirect2019-2023
median age at death69 yearsdirect2020-2024
Five-year relative survival70.9%direct2016-2022

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 · quarterly

NIH 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.

$9.0MNIH obligations, FY2025from $4.9M in FY2013 · +83%
18distinct projects funded7 in FY2013
$29.1Mpeak year was FY2018obligations, all institutes
80%of FY2025 awards from NCI16 of 20

NIH obligations by fiscal year

$7M$15M$22M$29M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$4.9M7773
FY2014$6.0M8885
FY2015$16.5M8876
FY2016$24.6M9972
FY2017$28.0M121288
FY2018$29.1M131194
FY2019$22.9M131398
FY2020$6.6M161596
FY2021$6.6M171799
FY2022$6.8M1817113
FY2023$7.2M1313109
FY2024$9.7M1715110
FY2025$9.0M2018127

Where FY2025 money went

InstitutionObligationsAwards
Division Of Cancer Epidemiology And Genetics$2.6M3
Icahn School Of Medicine At Mount Sinai$1.3M2
University Of Maryland Baltimore$1.0M3
Baylor College Of Medicine$0.9M1
University Of Miami School Of Medicine$0.8M2
Emory University$0.6M3
Medical College Of Wisconsin$0.6M1
University Of California, San Francisco$0.4M1
University Of Arizona$0.4M1
University Of Tx Md Anderson Can Ctr$0.2M1

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

NCI16 · 80%
VA1 · 5%
NINR1 · 5%
NIBIB1 · 5%
NIAID1 · 5%

Projects by administering institute. The rows above are the top 5 and account for 20 of 20.

Award mechanisms

R016 · 30%
ZIA3 · 15%
U543 · 15%
U012 · 10%
R251 · 5%
I011 · 5%

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

Division Of Cancer Epidemiology And Gene$2.6M · 29.0%
Icahn School Of Medicine At Mount Sinai$1.3M · 14.2%
University Of Maryland Baltimore$1.0M · 11.3%
Baylor College Of Medicine$0.9M · 10.4%
University Of Miami School Of Medicine$0.8M · 9.0%
Emory University$0.6M · 7.0%

Share of $9.0M in FY2025. The top three hold 55%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

75 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

patient 2unspecified 1
2 patient1 unspecified2 carry clinical annotation1 carry survival0 patient cohorts ≥100 GEO samples180 GEO samples total

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE254331Inflammatory pathways confer resistance to chemoradiotherapy in anal squamous cell carcinoma2024 · sequencing9811.1
patient cohortAPT 0.758 cites
GSE253560Transcriptome and microbiome-immune changes across preinvasive and invasive anal cancer lesions2024 · sequencing7020.5
APT 0.05stagemolecularPIK3CA4 cites
GSE293618Higher expression of HPV16 derived E7_LI Transcript Observed in Men with HIV and Recurrent Anal Cancer2025 · sequencing121
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 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.

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

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 (2026-09-12), 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."
}