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Methotrexate and folic acid
Methotrexate inhibits dihydrofolate reductase to suppress cell proliferation and immune activation, while folic acid supplementation mitigates methotrexate-induced folate deficiency and toxicity.
Methotrexate inhibits dihydrofolate reductase to suppress cell proliferation and immune activation, while folic acid supplementation mitigates methotrexate-induced folate deficiency and toxicity. Used for Rheumatoid arthritis, Various malignancies (as part of combination chemotherapy regimens), Autoimmune and inflammatory conditions.
At a glance
| Generic name | Methotrexate and folic acid |
|---|---|
| Sponsor | University Health Network, Toronto |
| Drug class | Antimetabolite / Folate antagonist |
| Target | Dihydrofolate reductase (DHFR) |
| Modality | Small molecule |
| Therapeutic area | Oncology / Immunology / Rheumatology |
| Phase | Phase 3 |
Mechanism of action
Methotrexate is a folate antagonist that blocks dihydrofolate reductase, reducing tetrahydrofolate cofactors needed for nucleotide synthesis and cell division. This mechanism makes it effective as both a chemotherapy and immunosuppressive agent. Folic acid (folinic acid or leucovorin) supplementation rescues normal cells from methotrexate toxicity by providing an alternative source of one-carbon units, thereby reducing adverse effects while preserving therapeutic benefit.
Approved indications
- Rheumatoid arthritis
- Various malignancies (as part of combination chemotherapy regimens)
- Autoimmune and inflammatory conditions
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Mucositis / stomatitis
- Nausea and vomiting
- Hepatotoxicity
- Nephrotoxicity
- Infection (due to immunosuppression)
- Alopecia
Key clinical trials
- Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia (PHASE2)
- A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia (PHASE3)
- Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia (PHASE2)
- A Study of the Efficacy and Safety of Lisocabtagene Maraleucel (Liso-cel) as First-Line Therapy in Adults With Transplant-Ineligible Primary Central Nervous System Lymphoma (PHASE2)
- LTA Pilot Study of Glucarpidase in Patients With Central Nervous System Lymphoma (EARLY_PHASE1)
- A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL) (PHASE3)
- Venetoclax Basket Trial for High Risk Hematologic Malignancies (PHASE1)
- A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia (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 and folic acid CI brief — competitive landscape report
- Methotrexate and folic acid updates RSS · CI watch RSS
- University Health Network, Toronto portfolio CI