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Methotrexate alone
Methotrexate inhibits dihydrofolate reductase, blocking folate metabolism and reducing DNA synthesis and cell proliferation.
Methotrexate inhibits dihydrofolate reductase, blocking folate metabolism and reducing DNA synthesis and cell proliferation. Used for Rheumatoid arthritis, Acute lymphoblastic leukemia, Osteosarcoma.
At a glance
| Generic name | Methotrexate alone |
|---|---|
| Also known as | MTX: Trexan or Methotrexate |
| Sponsor | Helsinki University Central Hospital |
| Drug class | Folate antagonist / Antimetabolite |
| Target | Dihydrofolate reductase (DHFR) |
| Modality | Small molecule |
| Therapeutic area | Oncology, Immunology, Rheumatology |
| Phase | Phase 3 |
Mechanism of action
Methotrexate is a folate antagonist that inhibits the enzyme dihydrofolate reductase, preventing the conversion of dihydrofolate to tetrahydrofolate. This disrupts one-carbon transfer reactions essential for nucleotide synthesis, leading to reduced DNA replication and cell division. At lower doses used in autoimmune conditions, it also has immunosuppressive and anti-inflammatory effects through multiple mechanisms including adenosine release and inhibition of T-cell activation.
Approved indications
- Rheumatoid arthritis
- Acute lymphoblastic leukemia
- Osteosarcoma
- Breast cancer
- Psoriasis
- Inflammatory bowel disease
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Mucositis / stomatitis
- Nausea and vomiting
- Hepatotoxicity
- Nephrotoxicity
- Alopecia
- Infection
- Pneumonitis
Key clinical trials
- Testing the Use of Steroids and Tyrosine Kinase Inhibitors With Blinatumomab or Chemotherapy for Newly Diagnosed BCR-ABL-Positive Acute Lymphoblastic Leukemia in Adults (PHASE3)
- Chemotherapy With Targeted-Immunotherapy for Newly Diagnosed Ph+ ALL (NA)
- Influence of Methotrexate Discontinuation on Immunogenicity After PCV-20 Vaccine in Patients ARDs (PHASE4)
- LTA Pilot Study of Glucarpidase in Patients With Central Nervous System Lymphoma (EARLY_PHASE1)
- Effect of Anti-Psoriatic Biologics on Risk of Anogenital Warts (CONDYPSO)
- A Study to Investigate LP-118, Ponatinib, Vincristine and Dexamethasone in Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LBL) (PHASE1, PHASE2)
- Immune Modulation During Palynziq® Treatment in Adults (IMPALA) (PHASE4)
- A Study of Gefitinib, Trametinib, Disulfiram, and Sunitinib in Addition to Standard Chemotherapy in People With Osteosarcoma (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Methotrexate alone CI brief — competitive landscape report
- Methotrexate alone updates RSS · CI watch RSS
- Helsinki University Central Hospital portfolio CI