Last reviewed · How we verify
Methotrexate, 6-mercaptopurine
This combination of methotrexate and 6-mercaptopurine inhibits nucleotide synthesis and DNA replication to suppress cancer cell proliferation.
This combination of methotrexate and 6-mercaptopurine inhibits nucleotide synthesis and DNA replication to suppress cancer cell proliferation. Used for Acute lymphoblastic leukemia (ALL) in pediatric patients, Acute myeloid leukemia (AML) in pediatric patients.
At a glance
| Generic name | Methotrexate, 6-mercaptopurine |
|---|---|
| Also known as | maintenance therapy |
| Sponsor | Children's Cancer Group, China |
| Drug class | Antimetabolite combination |
| Target | Dihydrofolate reductase (methotrexate); hypoxanthine-guanine phosphoribosyltransferase (6-mercaptopurine) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Methotrexate is a folate antagonist that inhibits dihydrofolate reductase, blocking thymidylate synthase and purine synthesis. 6-mercaptopurine is a purine analog that is converted to active metabolites that inhibit both de novo and salvage pathway nucleotide synthesis. Together, they reduce DNA and RNA synthesis in rapidly dividing cells, particularly effective in hematologic malignancies.
Approved indications
- Acute lymphoblastic leukemia (ALL) in pediatric patients
- Acute myeloid leukemia (AML) in pediatric patients
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Mucositis
- Hepatotoxicity
- Nausea and vomiting
- Infection
- Nephrotoxicity
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)
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- Blinatumomab and Combination Chemotherapy or Dasatinib, Prednisone, and Blinatumomab in Treating Older Patients With Acute Lymphoblastic Leukemia (PHASE2)
- 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)
- Asparaginase Erwinia Chrysanthemi With Chemotherapy for the Treatment of High-Risk Adults With Newly Diagnosed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma (PHASE2)
- A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL) (PHASE3)
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:
- Methotrexate, 6-mercaptopurine CI brief — competitive landscape report
- Methotrexate, 6-mercaptopurine updates RSS · CI watch RSS
- Children's Cancer Group, China portfolio CI