Disease Briefing

Basal cell carcinoma: 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 37 studies · 1,163 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in basal cell carcinoma — PTCH1, SMO, SUFU, GLI1, GLI2, TP53, PTCH2, CDKN2A, PDCD1, MYCN, PPP6C, STK19 — 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.

4
drugs carry an FDA label naming basal cell carcinoma: Cemiplimab, Imiquimod, Sonidegib, Vismodegib. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
2
of the 12 genes above carry a drug that is approved in basal cell carcinoma itself — PDCD1, SMO. Across all of them 45 drug entries reach these genes, 42 distinct once salt forms are merged
1
registered PD-1 cell-therapy trials in basal cell carcinoma, 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
8
targets carry an Open Targets tractability signal and have no clinical programme of any kind: PTCH1, SUFU, GLI1, GLI2, TP53, PTCH2, MYCN, PPP6C. PDCD1 has cell-therapy trials, so it is undrugged rather than untouched
120
human GEO series match the disease; 37 survive on-topic filtering, and only 0 are patient cohorts of 100+ samples
15
Europe PMC full-text papers name GSE53462 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$5.2M
NIH obligations in FY2025, up 6% since 2013 — while distinct core projects went 16 to 10. Larger awards rather than more distinct core projects; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-17 · weekly

12 genes recurrently implicated in basal cell carcinoma, 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: Basal cell carcinoma does have labelled therapy — 4 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 basal cell carcinoma 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
PTCH1 across cancers → 0 No drug antibody, protein degrader, small molecule
SMO across cancers → 8 3 approvedGlasdegib, Sonidegib, VismodegibApproved in basal cell carcinoma: Sonidegib, Vismodegib.8 active of 56 basal cell carcinoma trials antibody, protein degrader, small molecule
SUFU 0 No drug protein degrader, small molecule
GLI1 0 No drug antibody, protein degrader, small molecule
GLI2 0 No drug protein degrader, small molecule
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo basal cell carcinoma trial of any of these drugs other clinical modality, protein degrader, small molecule
PTCH2 0 No drug antibody
CDKN2A across cancers → 0 No drug
PDCD1 across cancers → 26 9 approvedCemiplimab, Dostarlimab, Nivolumab, Pembrolizumab, Retifanlimab, Serplulimab, Sintilimab, Tislelizumab, ToripalimabApproved in basal cell carcinoma: Cemiplimab.13 active of 23 basal cell carcinoma trials antibody, other clinical modality, protein degrader, small molecule 1 active of 1 trial
MYCN across cancers → 0 No drug protein degrader, small molecule
PPP6C 0 No drug protein degrader
STK19 0 No drug

Dataset evidence counts studies in the 37-study ranked set whose title or abstract names the gene; the bar is scaled to PTCH1. 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

4 drugs carry an FDA label naming basal cell carcinoma: Cemiplimab, Imiquimod, Sonidegib, Vismodegib. Separately, 9 of the drugs returned for the genes in the table above are approved only for other diseases and reach basal cell carcinoma through trials, not through their labels.

4Labelled for basal cell carcinomaFDA INDICATIONS AND USAGE names the disease
9Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
1Active PD-1 cell-therapy trialsof 1 registered

Every label that names basal cell carcinoma

DrugRoleWhat the label says
CemiplimabLIBTAYOLabelled hereBasal Cell Carcinoma LIBTAYO is indicated for the treatment of adult patients with locally advanced or metastatic basal cell carcinoma (laBCC or mBCC) who have been previously treated with a hedgehog pathway inhibitor or for whom a hedgehog pathway inhibitor is not appropriate.
ImiquimodIMIQUIMOD, ImiquimodLabelled hereSuperficial Basal Cell Carcinoma Imiquimod Cream is indicated for the topical treatment of biopsy-confirmed, primary superficial basal cell carcinoma (sBCC) in immunocompetent adults, with a maximum tumor diameter of 2.0 cm, located on the trunk (excluding anogenital skin), neck, or extremities (excluding hands and feet), only when surgical methods are medically less appropriate and patient fol...
SonidegibOdomzoLabelled hereODOMZO is a hedgehog pathway inhibitor indicated for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.
VismodegibERIVEDGELabelled hereERIVEDGE is indicated for the treatment of adults with metastatic basal cell carcinoma, or with locally advanced basal cell carcinoma that has recurred following surgery or who are not candidates for surgery and who are not candidates for radiation.

19 labels match indications_and_usage:"basal cell carcinoma"; they collapse to 4 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 PD-1

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

PD-1 is the busiest cell-therapy antigen in basal cell carcinoma: 1 registered trials, 1 still active. It has no gene entry of its own and is reached through PDCD1: the receptor PD-1 encodes; cemiplimab is approved after, or for those who cannot take, a Hedgehog inhibitor.

TrialPhaseStatusTitleLast update
NCT063984181RecruitingR-5780-01 In Combination With PD-1 Checkpoint Inhibitors (Checkpoint Protein on Immune Cells Called T Cells) in Patients With Solid Tumors2026-09-04

1 of 1 shown, most recently active first. 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 basal cell carcinoma literature, not a count, because the field itself grew: 2015–2018 (n=1,350) against 2021–2025 (n=1,508). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Tumor Microenvironment0.44%1.33%2.98×20 papers
Quality of Life0.59%1.53%2.57×23 papers
Sebaceous Gland Neoplasms0.37%0.93%2.51×14 papers
Eyelids1.78%3.58%2.01×54 papers
Margins of Excision2.59%4.91%1.89×74 papers
Ultrasonography0.67%1.26%1.89×19 papers
Biphenyl Compounds2.3%4.24%1.85×64 papers
Curettage0.52%0.93%1.79×14 papers
Hedgehog Proteins6.3%10.74%1.71×162 papers
Cicatrix0.81%1.26%1.55×19 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Neoplasm Staging4.67%0.8%0.17×12 papers
Neoplasm Invasiveness4.44%1.06%0.24×16 papers
Prognosis4.37%1.13%0.26×17 papers
Cell Proliferation2.67%0.86%0.32×13 papers
Skin Transplantation2.52%0.8%0.32×12 papers
Facial Neoplasms6.15%2.06%0.33×31 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Skin Neoplasms78.67%88.59%1.13×1336 papers
Mohs Surgery11.85%14.92%1.26×225 papers
Hedgehog Proteins6.3%10.74%1.71×162 papers
Antineoplastic Agents11.33%10.54%0.93×159 papers
Neoplasm Recurrence, Local8.52%10.21%1.2×154 papers
Treatment Outcome13.78%10.15%0.74×153 papers
Pyridines10.52%9.35%0.89×141 papers
Basal Cell Nevus Syndrome11.56%9.08%0.79×137 papers
Anilides9.33%7.63%0.82×115 papers
Dermoscopy7.48%5.57%0.74×84 papers

Publication mix

Type2015–182021–25
Clinical Trial0.0%0.1%
Randomized Controlled Trial2.4%1.6%
Review9.8%7.6%
Meta-Analysis0.9%0.5%
Case Reports0.0%4.2%

Query: Basal Cell Carcinoma[MeSH Major Topic] NOT ("Melanoma"[MeSH] OR "Carcinoma, Squamous Cell"[MeSH] OR "Keratosis, Actinic"[MeSH] OR "Carcinoma, Merkel Cell"[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 year5,400,000 cancers/year (lesions, not people)proxycounts basal and squamous cell skin cancer (lesions, not people), which is broader than this disease
New cases each year, estimated4,320,000 cancers/year (lesions, not people)derived proxy5,400,000 x 0.8, from basal and squamous cell skin cancer (lesions, not people)
Deaths each yearnot publishedNot published: "deaths from these cancers are very rare" and are not tracked by registries.
Five-year relative survivalnot publishedNot published; the disease is not registered, so no relative survival exists. The page says the tumours "rarely spread to other parts of the body".
Median age at diagnosisnot publishedNot published; the page says risk "goes up 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 is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming basal cell carcinoma, after removing the 5,432 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.

$5.2MNIH obligations, FY2025from $4.9M in FY2013 · +6%
10distinct projects funded16 in FY2013
$8.3Mpeak year was FY2020obligations, all institutes
50%of FY2025 awards from NCI5 of 10

NIH obligations by fiscal year

$2M$4M$6M$8M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$4.9M1616104
FY2014$5.3M212195
FY2015$6.7M242397
FY2016$5.7M2323414
FY2017$8.2M2724493
FY2018$7.5M2119546
FY2019$6.2M2117554
FY2020$8.3M2019536
FY2021$6.7M1815548
FY2022$5.0M1615554
FY2023$4.1M1010525
FY2024$2.9M77498
FY2025$5.2M1010468

Where FY2025 money went

InstitutionObligationsAwards
Division Of Basic Sciences - Nci$1.3M1
Stanford University$1.0M2
Surgivance, Inc.$1.0M1
Utah State Higher Education System--University Of Utah$0.8M2
University Of Alabama At Birmingham$0.6M1
University Of Arizona$0.5M1
Va Greater Los Angeles Healthcare System$0.0M1
Veterans Health Administration$0.0M1

Text search basal cell 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 2026-09-17.

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

NCI5 · 50%
NIAMS3 · 30%
VA2 · 20%

Projects by administering institute. The rows above are the top 3 and account for 10 of 10.

Award mechanisms

R013 · 30%
R211 · 10%
I011 · 10%
ZIA1 · 10%
IK21 · 10%
R371 · 10%

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 8 and account for 10 of 10.

Where it lands

Division Of Basic Sciences - Nci$1.3M · 24.2%
Stanford University$1.0M · 19.0%
Surgivance, Inc.$1.0M · 19.0%
Utah State Higher Education System--Univ$0.8M · 16.4%
University Of Alabama At Birmingham$0.6M · 12.4%
University Of Arizona$0.5M · 9.0%

Share of $5.2M in FY2025. The top three hold 62%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

120 human GEO series match basal cell carcinoma. Keyword relevance cannot tell a 72-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 37-study ranked set

unspecified 21patient 14cell line 2
21 unspecified14 patient2 cell line14 carry clinical annotation5 carry survival0 patient cohorts ≥100 GEO samples1,163 GEO samples totalin 5 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE58377SMO variants explain the majority of drug resistance in basal cell carcinoma2015 · sequencing1071110.3
APT 0.95molecularSMO347 cites
GSE42109Identification of anaplastic lymphoma kinase as a candidate of new therapeutic target for BCC2013 · array21110.6
patient cohortAPT 0.25molecularGLI1HEDGEHOGSMOOTHENED20 cites
GSE6520Microarray analysis shows multiple signaling pathways are involved in basal cell carcinoma growth2006 · array3132.4
patient cohortAPT 0.5survivalmolecularHEDGEHOG99 cites
GSE125285Characterizing gene expression patterns in Basal Cell Carcinomas and Squamous Cell Carcinomas2020 · sequencing70131.0
APT 0.526 cites
GSE34535Microarray analysis of microRNA expression in basal cell carcinoma2012 · array1434.7
patient cohortAPT 0.75164 cites
GSE5121Imiquimod treated Basal Cell Carcinoma2006 · array7211.5
patient cohortAPT 0.75molecularIMIQUIMOD80 cites
GSE53462Molecular classification of non-melanoma skin cancer by gene expression profiling2014 · array26150.8
APT 0.524 cites
GSE156855AP-1 and TGFb cooperativity drives non-canonical Hedgehog signaling in resistant basal cell carcinoma2020 · chromatin38152.3
APT 0.75molecularHEDGEHOG58 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE268728A neoadjuvant, phase II trial demonstrates efficacy and tolerability of Talimogene laherparepvec in cutaneous basal cell carcinoma (NeoBCC trial)2024 · sequencing1212.0
patient cohortAPT 0.05stagemolecularONCOLYTICT-VECTALIMOGENE9 cites
GSE210648Integrated multi-omics reveals cellular and molecular interactions governing the invasive niche of basal cell carcinoma (Digital Spatial Profiling)2022 · single-cell9583.0
APT 0.552 cites
GSE269601COL10A1 expression distinguishes a subset of cancer-associated fibroblasts present in the stroma of high-risk basal cell carcinoma2024 · single-cell2111.4
patient cohortAPT 0.05survivalstage10 cites
GSE196292Immune cell gene expression in healthy, basal cell carcinoma and melanoma skin2022 · sequencing4021.8
APT 0.2522 cites
GSE180706RNA-seq analysis of sporadic and Basal Cell Nevus Syndrome-associated odontogenic keratocysts (OKCs)2022 · sequencing1810.8
patient cohortAPT 0.257 cites
GSE233744The Hedgehog gene expression program regulates lipid metabolism in basal cell carcinoma and medulloblastoma2023 · sequencing990
molecularGLI1HEDGEHOGPTCH1

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-17. SubSeries are collapsed to one row per study by linked PMID. Of 120 series retrieved, 26 were dropped by the profile’s exclusion rules and 46 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.

PTCH1

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

SUFU

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

GLI1

No drug in any database targets GLI1, while 29 trials target Hedgehog — the antigen it produces. A gene-centric search finds nothing here and concludes wrongly.

GLI2

No drug in any database targets GLI2, while 29 trials target Hedgehog — the antigen it produces. A gene-centric search finds nothing here and concludes wrongly.

PTCH2

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

CDKN2A

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

MYCN

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

PPP6C

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

STK19

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 basal cell 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

5 exclusion patterns are applied to free text before anything is ranked, because none: no line is in regular use is used as a model system in basal cell carcinoma. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Basal cell carcinoma",
  "mesh": "Basal Cell Carcinoma",
  "facts": "https://usebiotransfer.org/disease/basal-cell-carcinoma.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."
}