Protein / target

DNA topoisomerase 2-alpha

TOP2AP11388Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
17
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

ATP-dependent activity, acting on DNA

Strongest disease association

neoplasm

Clinical evidence · score 0.62

Therapeutic maturity

Clinically validated target

17 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

17 approved · 7 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity)

Subcellular location

CytoplasmNucleus, nucleoplasmNucleusNucleus, nucleolus
Domains and Gene Ontology detail (35)

Domains & features

ToprimTopo IIA-type catalytic

Gene Ontology

  • Cchromosome, centromeric region
  • Ccondensed chromosome
  • Ccytoplasm
  • CDNA topoisomerase type II (double strand cut, ATP-hydrolyzing) complex
  • Cnucleolus
  • Cnucleoplasm
  • Cnucleus
  • Cprotein-containing complex
  • Cribonucleoprotein complex
  • FATP binding
  • FATP-dependent activity, acting on DNA
  • Fchromatin binding

1531 aa · 174 kDa · 4 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Transcriptional regulationUniProt · ReactomeKinase signallingGOApoptosis & cell deathGO
View supporting evidence

Transcriptional regulation

  • ·Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA m…
  • ·Transcription of E2F targets under negative control by DREAM complex

Kinase signalling

  • ·protein kinase C binding

Apoptosis & cell death

  • ·positive regulation of apoptotic process
View underlying pathways (2)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

NUSAP1CDK1ASPMTOP1MKI67CCNB1BUB1CCNA2BUB1BCDC20TOP2A

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

Etoposide
ApprovedInhibitor

DNA topoisomerase II inhibitor

Appears in clinical studies involving neoplasm, small cell lung carcinoma, lung cancer, small cell carcinoma

Acts on a complex — shared with TOP2B · 1 of 2 recorded protein targets — narrow recorded profile

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

acute myeloid leukemia0.97

Clinical · overall 0.61

plasma cell myeloma0.97

Clinical · overall 0.59

breast cancer0.97

Clinical · overall 0.62

neoplasm0.97

Clinical · overall 0.62

Kaposi's sarcoma0.95

Clinical · overall 0.58

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

small cell lung carcinoma0.58

Clinical

Hodgkins lymphoma0.57

Clinical

ovarian cancer0.55

Clinical

ovarian carcinoma0.55

Clinical

acute myeloid leukemia by FAB classification0.54

Clinical

Show all associations
neoplasm0.62
breast cancer0.62
acute myeloid leukemia0.61
plasma cell myeloma0.59
small cell lung carcinoma0.58
Kaposi's sarcoma0.58
Hodgkins lymphoma0.57
ovarian cancer0.55
ovarian carcinoma0.55
acute myeloid leukemia by FAB classification0.54

Open Targets ranks 730 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 24 total

DEXRAZOXANEApproval

breast cancer · Hodgkins lymphoma · acute lymphoblastic leukemia

PAZUFLOXACINApproval

streptococcal pneumonia · Sepsis · legionellosis

DOXORUBICINApproval

Kaposi's sarcoma · breast neoplasm · plasma cell myeloma

DOXORUBICIN HYDROCHLORIDEApproval

bone sarcoma · urinary bladder carcinoma · breast cancer

TENIPOSIDEApproval

neoplasm · acute lymphoblastic leukemia · hemophagocytic syndrome

MITOXANTRONE HYDROCHLORIDEApproval

multiple sclerosis · prostate cancer · acute myeloid leukemia

BECATECARINPhase 3

neuroblastoma · colorectal cancer · breast cancer

ETOPOSIDE PHOSPHATEPhase 3

embryonal carcinoma · germinoma · nongerminomatous germ cell tumor

VALRUBICINApproval

urinary bladder carcinoma · carcinoma · neoplasm

VOSAROXINApproval

neoplasm · acute myeloid leukemia by FAB classification · acute myeloid leukemia

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · Advanced ClinicalPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Safety liabilities

neutropenia

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ACTIVE_NOT_RECRUITING · via Etoposide · NCT03020030

COMPLETED · via Etoposide · NCT00426101

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

neoplasmWell supported
0.77
agreement 0.610.92
Clinical83%Literature17%

Open Targets aggregate 0.62 · 2 independent evidence families

breast cancerWell supported
0.76
agreement 0.610.92
Clinical84%Literature16%

Open Targets aggregate 0.62 · 2 independent evidence families

acute myeloid leukemiaModerately supported
0.75
agreement 0.590.90
Clinical91%Literature9%

Open Targets aggregate 0.61 · 2 independent evidence families

plasma cell myelomaModerately supported
0.73
agreement 0.580.89
Clinical98%Literature2%

Open Targets aggregate 0.59 · 2 independent evidence families

small cell lung carcinomaModerately supported
0.72
agreement 0.570.88
Clinical88%Literature12%

Open Targets aggregate 0.58 · 2 independent evidence families

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

5

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  1. New publication2023-06-17
    Clinical insights into small cell lung cancer: Tumor heterogeneity, diagnosis, therapy, and future directions.

    CA: a cancer journal for clinicians · 2023 · 299 citations · Europe PMC · via Etoposide

  2. New publication2021-01-13
    Updated Overall Survival and PD-L1 Subgroup Analysis of Patients With Extensive-Stage Small-Cell Lung Cancer Treated With Atezolizumab, Carboplatin, and Etoposide (IMpower133).

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2021 · 649 citations · Europe PMC · via Etoposide

  3. New publication2018-09-25
    First-Line Atezolizumab plus Chemotherapy in Extensive-Stage Small-Cell Lung Cancer.

    The New England journal of medicine · 2018 · 2,697 citations · Europe PMC · via Etoposide

  4. New publication2018-07-01
    As-Needed Vs Immediate Etoposide Chemotherapy in Combination With Antiretroviral Therapy for Mild-to-Moderate AIDS-Associated Kaposi Sarcoma in Resource-Limited Settings: A5264/AMC-067 Randomized Clinical Trial.

    Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2018 · 27 citations · Europe PMC · via Etoposide

  5. New publication2002-01-01
    Irinotecan plus cisplatin compared with etoposide plus cisplatin for extensive small-cell lung cancer.

    The New England journal of medicine · 2002 · 852 citations · Europe PMC · via Etoposide

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.