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Methotrexate + targeted therapy administration
Methotrexate inhibits dihydrofolate reductase to suppress cell proliferation and immune activation, often combined with targeted therapies that inhibit specific oncogenic pathways or immune checkpoints.
Methotrexate inhibits dihydrofolate reductase to suppress cell proliferation and immune activation, often combined with targeted therapies that inhibit specific oncogenic pathways or immune checkpoints. Used for Hematologic malignancies (lymphomas, leukemias) — specific indication depends on targeted therapy partner, Solid tumors with targeted therapy combinations — indication varies by protocol.
At a glance
| Generic name | Methotrexate + targeted therapy administration |
|---|---|
| Sponsor | University Hospital, Strasbourg, France |
| Drug class | Combination chemotherapy with targeted agent |
| Target | Dihydrofolate reductase (methotrexate component); targeted agent varies by protocol |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Methotrexate is a folate antagonist that blocks DNA synthesis and cell division, making it effective as both a chemotherapy and immunosuppressive agent. When combined with targeted therapies (such as tyrosine kinase inhibitors or monoclonal antibodies), the regimen exploits synergistic mechanisms to enhance anti-tumor or anti-inflammatory effects while potentially reducing individual drug toxicity through dose optimization.
Approved indications
- Hematologic malignancies (lymphomas, leukemias)
- Solid tumors with specific molecular drivers (context-dependent on targeted agent used)
Common side effects
- Myelosuppression
- Mucositis
- Hepatotoxicity
- Nephrotoxicity
- Nausea and vomiting
Key clinical trials
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- Chemotherapy With Targeted-Immunotherapy for Newly Diagnosed Ph+ ALL (NA)
- Full-Course Immunotherapy Consolidation for Unfit or Fit B-ALL Who Decline Chemotherapy (PHASE2)
- A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256) (PHASE2)
- P-BCMA-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Multiple Myeloma (PHASE1)
- Venetoclax, SL-401, and Chemotherapy for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (PHASE2)
- Early Adalimumab Induction for Immune Checkpoint Inhibitor Associated Inflammatory Arthritis (PHASE2)
- Effects of Telitacicept vs Cyclophosphamide on Lupus Related Interstitial Lung Disease (PHASE4)
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 |
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