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Protein / target

Dihydrofolate reductase

Encoded byDHFRP00374Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
8
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

MRNA regulatory element binding translation repressor

Strongest disease association

Inherited cancer-predisposing syndrome

Via encoding gene DHFR · Genetic evidence · score 0.92

Therapeutic position

Established drug target

Small molecules

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 overview

Catalyzes the reduction of 7,8-dihydrofolate (DHF) to 5,6,7,8-tetrahydrofolate in a NADPH-dependent manner.

View complete UniProt function annotation

Catalyzes the reduction of 7,8-dihydrofolate (DHF) to 5,6,7,8-tetrahydrofolate in a NADPH-dependent manner (PubMed:12096917, PubMed:15039552, PubMed:17569517, PubMed:19196009, PubMed:19478082, PubMed:21876184, PubMed:9719595). Key enzyme in folate metabolism. Contributes to the nuclear and mitochondrial de novo thymidylate biosynthesis pathway (PubMed:21876188, PubMed:22235121). Catalyzes an essential reaction for de novo glycine and purine synthesis, and for DNA precursor synthesis. Binds its own mRNA and that of DHFR2

Subcellular location

MitochondrionCytoplasmNucleus
Domains and Gene Ontology detail (24)

Domains & features

DHFR

Gene Ontology

  • Ccytosol
  • Cmitochondrion
  • Cnucleus
  • Creplication fork
  • Fdihydrofolate reductase activity
  • Ffolic acid binding
  • FmRNA binding
  • FmRNA regulatory element binding translation repressor activity
  • FNADP binding
  • FNADPH binding
  • Fsequence-specific mRNA binding
  • P'de novo' pyrimidine nucleobase biosynthetic process

187 aa · 21 kDa · 2 isoforms

Biological roles

Reactome v97

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

Metabolic enzyme activityGO
View supporting evidence

Metabolic enzyme activity

  • ·dihydrofolate metabolic process
  • ·folic acid metabolic process
  • ·one-carbon metabolic process
  • ·tetrahydrofolate metabolic process
View underlying pathways (3)

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.

TYMSFPGSSHMT1SHMT2MTHFD1GARTATICSPRQDPRMTRDHFR

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.

Approved medicines with mapped indications

2 medicines · 13 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Arthritis1 medicine
Arthritis, Juvenile1 medicine
Arthritis, Psoriatic1 medicine
Arthritis, Rheumatoid1 medicine
Breast Neoplasms1 medicine
Immune System Diseases1 medicine
Lymphoma, Non-Hodgkin's1 medicine
Meningeal Carcinomatosis1 medicine
Mesothelioma1 medicine
Mycosis Fungoides1 medicine
Precursor Cell Lymphoblastic Leukemia-Lymphoma1 medicine
Psoriasis1 medicine
Broader indication categories (1)
Neoplasms2 medicines

Broad umbrella indications (e.g. “Neoplasms”). Shown here because every medicine also appears under a more specific disease above — kept for completeness, de-emphasised for clarity.

8 medicines meet Open Targets' target-level approved-medicine definition; the 2 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

2

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

pemetrexed
Narrow target profileApprovedInhibitor

Dihydrofolate reductase inhibitor

Indicated for Mesothelioma, Neoplasms

Direct interaction with this protein · 1 of 3 recorded protein targets — narrow recorded profile

Methotrexate
Narrow target profileApprovedInhibitor

Dihydrofolate reductase inhibitor

Indicated for Arthritis, Arthritis, Juvenile, Arthritis, Psoriatic, Arthritis, Rheumatoid

Direct interaction with this protein · Only this protein recorded as a target

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

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene DHFR

Gene-level evidence surfaced through the gene DHFRthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Inherited cancer-predisposing syndrome
0.92Well supported

Genetic evidence dominant · Open Targets 0.56

Arthritis, Rheumatoid
0.76Well supported

Clinical evidence dominant · Open Targets 0.62

Carcinoma, Non-Small-Cell Lung
0.76Well supported

Clinical evidence dominant · Open Targets 0.62

Constitutional megaloblastic anemia with severe neurologic disease
0.75Well supported

Genetic evidence dominant · Open Targets 0.79

Precursor Cell Lymphoblastic Leukemia-Lymphoma
0.75Well supported

Clinical evidence dominant · Open Targets 0.61

View evidence synthesis (5)
Inherited cancer-predisposing syndromeWell supported
0.92
agreement 0.801.00
Genetic100%

Open Targets aggregate 0.56 · 1 independent evidence family

Arthritis, RheumatoidWell supported
0.76
agreement 0.610.92
Clinical92%Literature8%

Open Targets aggregate 0.62 · 2 independent evidence families

Carcinoma, Non-Small-Cell LungWell supported
0.76
agreement 0.600.91
Clinical91%Literature9%

Open Targets aggregate 0.62 · 2 independent evidence families

Constitutional megaloblastic anemia with severe neurologic diseaseWell supported
0.75
agreement 0.610.89
Genetic99%Literature1%Genetic literaturedup

Open Targets aggregate 0.79 · 2 independent evidence families · 1 not counted as duplicate

Precursor Cell Lymphoblastic Leukemia-LymphomaWell supported
0.75
agreement 0.600.91
Clinical85%Literature15%

Open Targets aggregate 0.61 · 2 independent evidence families

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. 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 per-type scores above show the calculation.

Show all associations
Constitutional megaloblastic anemia with severe neurologic disease0.79
Arthritis, Rheumatoid0.62
Carcinoma, Non-Small-Cell Lung0.62
Precursor Cell Lymphoblastic Leukemia-Lymphoma0.61
Psoriasis0.60
Breast Neoplasms0.59
Mesothelioma0.58
Arthritis, Juvenile0.58
Malignant pleural mesothelioma0.56
Inherited cancer-predisposing syndrome0.56

Drug development

9 compounds recorded · 8 approved · 1 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 2 drugs that target this protein in Forefront's canonical graph (2 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (9)
PEMETREXED MONOHYDRATEApproval
METHOTREXATE SODIUMApproval
METHOTREXATEApproval
PEMETREXED DIPOTASSIUMApproval
PRALATREXATEApproval
PEMETREXEDApproval
PEMETREXED TROMETHAMINEApproval
PEMETREXED DISODIUMApproval
PIRITREXIMPhase 2

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and Structure with Ligand support this modality.

Protein degradersEmerging

Feasibility evidence (database ubiquitination and half-life data) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (8)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

side effectsClinPGxdrug toxicityClinPGxmucositisClinPGx

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

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. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (26)

ACTIVE_NOT_RECRUITING · via Methotrexate · NCT03020030

ACTIVE_NOT_RECRUITING · via Methotrexate · NCT05192889

ClinicalTrials.gov via the drug-target graph.

What's happening now

12

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for 2 drugs that target this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Trial status changed2026-08-25

    Phase I/II Study of Neoadjuvant Inhaled Azacytidine With Platinum-Based Chemotherapy and Durvalumab (MEDI4736) - a Combined Epigenetic-Immunotherapy (AZA-AEGEAN) Regimen for Operable Early-Stage Non-Small Cell Lung Cancer (NSCLC)

    Status changed to Suspended · ClinicalTrials.gov · via pemetrexed

  2. Trial status changed2026-08-04

    JS207 (PD-1/VEGF Dual Antibody) Combined With Chemotherapy in First-line Treatment of Advanced Non-small Cell Lung Cancer

    Status changed to Active, not recruiting · ClinicalTrials.gov · via pemetrexed

  3. Withdrawn from market2024-10-09

    Market withdrawal: Pemetrexed Sandoz (EMA)

    ema · market · ema · via pemetrexed

  4. New publication2024-08-22
    Phase III KEYNOTE-789 Study of Pemetrexed and Platinum With or Without Pembrolizumab for Tyrosine Kinase Inhibitor‒Resistant, <i>EGFR</i>-Mutant, Metastatic Nonsquamous Non-Small Cell Lung Cancer.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024 · 162 citations · Europe PMC · via pemetrexed

  5. Withdrawn from market2023-10-12

    Market withdrawal: Ciambra (EMA)

    ema · market · ema · via pemetrexed

  6. Safety communication2023-08-30

    Drug Safety Update: Methotrexate: advise patients to take precautions in the sun to avoid photosensitivity reactions

    mhra · safety · mhra · via Methotrexate

  7. New publication2023-02-21
    Pembrolizumab Plus Pemetrexed and Platinum in Nonsquamous Non-Small-Cell Lung Cancer: 5-Year Outcomes From the Phase 3 KEYNOTE-189 Study.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023 · 505 citations · Europe PMC · via pemetrexed

  8. Regulatory approval2017-03-29

    Approval: Jylamvo (EMA)

    ema · regulatory · ema · via Methotrexate

  9. New publication2016-12-06
    Osimertinib or Platinum-Pemetrexed in EGFR T790M-Positive Lung Cancer.

    The New England journal of medicine · 2017 · 2,677 citations · Europe PMC · via pemetrexed

  10. Regulatory approval2016-01-18

    Approval: Armisarte (previously Pemetrexed Actavis) (EMA)

    ema · regulatory · ema · via pemetrexed

  11. New publication2014-12-01
    First-line crizotinib versus chemotherapy in ALK-positive lung cancer.

    The New England journal of medicine · 2014 · 2,470 citations · Europe PMC · via pemetrexed

  12. Regulatory approval2004-09-20

    Approval: Alimta (EMA)

    ema · regulatory · ema · via pemetrexed

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.