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Intrathecal Methotrexate
Intrathecal Methotrexate is a Antimetabolite; folate antagonist Small molecule drug developed by Sun Yat-sen University. It is currently FDA-approved for Leptomeningeal metastases (prophylaxis and treatment), CNS leukemia (acute lymphoblastic leukemia with CNS involvement), Lymphomatous meningitis. Also known as: Rheumatrex, Methotrexate, Rheumatrex®.
Intrathecal methotrexate is a chemotherapy agent delivered directly into the cerebrospinal fluid to inhibit dihydrofolate reductase and disrupt DNA synthesis in cancer cells, particularly those in the central nervous system.
Intrathecal methotrexate is a chemotherapy agent delivered directly into the cerebrospinal fluid to inhibit dihydrofolate reductase and disrupt DNA synthesis in cancer cells, particularly those in the central nervous system. Used for Leptomeningeal metastases (prophylaxis and treatment), CNS leukemia (acute lymphoblastic leukemia with CNS involvement), Lymphomatous meningitis.
At a glance
| Generic name | Intrathecal Methotrexate |
|---|---|
| Also known as | Rheumatrex, Methotrexate, Rheumatrex® |
| Sponsor | Sun Yat-sen University |
| 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 blocks dihydrofolate reductase, preventing the conversion of dihydrofolate to tetrahydrofolate, which is essential for one-carbon transfer reactions and DNA/RNA synthesis. When administered intrathecally (directly into the cerebrospinal fluid), it achieves high local concentrations in the CNS to treat or prevent leptomeningeal metastases and CNS leukemias. This route bypasses the blood-brain barrier and delivers cytotoxic concentrations directly to malignant cells in the meninges and brain.
Approved indications
- Leptomeningeal metastases (prophylaxis and treatment)
- CNS leukemia (acute lymphoblastic leukemia with CNS involvement)
- Lymphomatous meningitis
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Neurotoxicity (chemical arachnoiditis, transient paraplegia)
- Headache
- Fever
- Nausea and vomiting
- Leukoencephalopathy (with repeated dosing)
Key clinical trials
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- 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)
- BNHL-2015 for Children or Adolescents in China (PHASE2, PHASE3)
- CCCG-ALCL-2020 for Chinese Children and Adolescents With Newly Diagnosed High-risk ALCL (PHASE3)
- Capizzi Escalating Methotrexate Versus High Dose Methotrexate in Children With Newly Diagnosed T-cell Lymphoblastic Lymphoma (T-LBL) (PHASE3)
- LBL-2016 for Children or Adolescents in China (PHASE3)
- Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia (PHASE1)
- Identification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia/Lymphoma (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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Intrathecal Methotrexate CI brief — competitive landscape report
- Intrathecal Methotrexate updates RSS · CI watch RSS
- Sun Yat-sen University portfolio CI
Frequently asked questions about Intrathecal Methotrexate
What is Intrathecal Methotrexate?
How does Intrathecal Methotrexate work?
What is Intrathecal Methotrexate used for?
Who makes Intrathecal Methotrexate?
Is Intrathecal Methotrexate also known as anything else?
What drug class is Intrathecal Methotrexate in?
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What does Intrathecal Methotrexate target?
Related
- Drug class: All Antimetabolite; folate antagonist drugs
- Target: All drugs targeting Dihydrofolate reductase (DHFR)
- Manufacturer: Sun Yat-sen University — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Leptomeningeal metastases (prophylaxis and treatment)
- Indication: Drugs for CNS leukemia (acute lymphoblastic leukemia with CNS involvement)
- Indication: Drugs for Lymphomatous meningitis
- Also known as: Rheumatrex, Methotrexate, Rheumatrex®
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing