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MTX
MTX (methotrexate) inhibits dihydrofolate reductase, blocking the synthesis of purines and pyrimidines required for DNA replication and cell division.
MTX (methotrexate) inhibits dihydrofolate reductase, blocking the synthesis of purines and pyrimidines required for DNA replication and cell division. Used for Acute lymphoblastic leukemia (ALL) in children, Acute myeloid leukemia (AML), Lymphomas.
At a glance
| Generic name | MTX |
|---|---|
| Also known as | methotrexate, Methotrexate, Rheumatrex, Trexall, Commercially available 2.5 mg MTX oral tablets |
| Sponsor | South China Children's Leukemia Group |
| Drug class | Antimetabolite; folate antagonist |
| Target | Dihydrofolate reductase (DHFR) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Methotrexate is a folate antagonist that disrupts one-carbon transfer reactions essential for nucleotide synthesis. By inhibiting dihydrofolate reductase, it depletes tetrahydrofolate cofactors, thereby suppressing DNA and RNA synthesis. This mechanism makes it effective as both a cytotoxic chemotherapy agent and an immunosuppressant, particularly useful in rapidly dividing cells such as leukemic blasts and lymphocytes.
Approved indications
- Acute lymphoblastic leukemia (ALL) in children
- Acute myeloid leukemia (AML)
- Lymphomas
- Osteosarcoma
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Mucositis
- Hepatotoxicity
- Nephrotoxicity
- Nausea and vomiting
- Alopecia
- Neurotoxicity (with intrathecal administration)
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)
- A Study to Test the Addition of the Drug Cabozantinib to Chemotherapy in Patients With Newly Diagnosed Osteosarcoma (PHASE2, PHASE3)
- Testing the Addition of MEDI4736 (Durvalumab) to Chemotherapy Before Surgery for Patients With High-Grade Upper Urinary Tract Cancer (PHASE2, PHASE3)
- Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia (PHASE2)
- 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)
- Blinatumomab and Combination Chemotherapy or Dasatinib, Prednisone, and Blinatumomab in Treating Older Patients With Acute Lymphoblastic Leukemia (PHASE2)
- A Study to Find the Highest Dose of SNDX-5613 (Revumenib) as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Mixed Phenotype Acute Leukemia (PHASE1)
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:
- MTX CI brief — competitive landscape report
- MTX updates RSS · CI watch RSS
- South China Children's Leukemia Group portfolio CI