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MTX therapy
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 Acute lymphoblastic leukemia (ALL), Osteosarcoma, Breast cancer.
At a glance
| Generic name | MTX therapy |
|---|---|
| Sponsor | NYU Langone Health |
| Drug class | Folate antagonist / Antimetabolite |
| Target | Dihydrofolate reductase (DHFR) |
| Modality | Small molecule |
| Therapeutic area | Oncology, Immunology, Rheumatology |
| Phase | FDA-approved |
Mechanism of action
MTX is a folate antagonist that inhibits dihydrofolate reductase, an enzyme critical for converting dihydrofolate to tetrahydrofolate, which is essential for one-carbon transfer reactions in nucleotide synthesis. This leads to decreased DNA and RNA synthesis, making it effective as both a chemotherapy agent and an immunosuppressant. At lower doses, it also has immunomodulatory effects through adenosine release and inhibition of inflammatory cytokine production.
Approved indications
- Acute lymphoblastic leukemia (ALL)
- Osteosarcoma
- Breast cancer
- Rheumatoid arthritis
- Psoriasis
- Inflammatory bowel disease
Common side effects
- Myelosuppression (anemia, leukopenia, thrombocytopenia)
- Mucositis / stomatitis
- Nausea and vomiting
- Hepatotoxicity
- Nephrotoxicity
- Neurotoxicity (high-dose)
- Alopecia
- Infection
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)
- 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)
- Testing the Addition of Lenalidomide and Nivolumab to the Usual Treatment for Primary CNS Lymphoma (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |