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Methotrexate (MTX)
Methotrexate inhibits dihydrofolate reductase, blocking the synthesis of purines and pyrimidines required for DNA replication and cell division.
Methotrexate inhibits dihydrofolate reductase, blocking the synthesis of purines and pyrimidines required for DNA replication and cell division. Used for Acute lymphoblastic leukemia (ALL), Osteosarcoma, Breast cancer.
At a glance
| Generic name | Methotrexate (MTX) |
|---|---|
| Also known as | MTX, Low dose Methotrexate, Rheumatrex, Methotrexate sodium,, MTX, |
| Sponsor | Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh |
| Drug class | Antimetabolite; folate antagonist |
| Target | Dihydrofolate reductase (DHFR) |
| Modality | Small molecule |
| Therapeutic area | Oncology; Immunology; Rheumatology |
| Phase | FDA-approved |
Mechanism of action
MTX is a folate antagonist that competitively inhibits dihydrofolate reductase (DHFR), an enzyme essential for converting dihydrofolate to tetrahydrofolate. This disrupts one-carbon transfer reactions needed for nucleotide synthesis, leading to cell cycle arrest and apoptosis in rapidly dividing cells. At lower doses, it also has immunosuppressive effects through adenosine release and T-cell inhibition.
Approved indications
- Acute lymphoblastic leukemia (ALL)
- Osteosarcoma
- Breast cancer
- Rheumatoid arthritis
- Psoriasis
- Inflammatory bowel disease
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Mucositis and gastrointestinal toxicity
- Hepatotoxicity
- Nephrotoxicity
- Nausea and vomiting
- Alopecia
- Neurotoxicity (at high doses)
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
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |