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Mercaptopurine (Purinethol)
Mercaptopurine is a purine analog that inhibits de novo purine synthesis and is converted to active metabolites that interfere with DNA and RNA synthesis, leading to cell death.
Mercaptopurine is a purine analog that inhibits de novo purine synthesis and is converted to active metabolites that interfere with DNA and RNA synthesis, leading to cell death. Used for Acute lymphoblastic leukemia (ALL), Chronic myeloid leukemia (CML), Inflammatory bowel disease (Crohn's disease, ulcerative colitis).
At a glance
| Generic name | Mercaptopurine (Purinethol) |
|---|---|
| Also known as | Purinethol |
| Sponsor | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) |
| Drug class | Purine analog antimetabolite |
| Target | HGPRT (hypoxanthine-guanine phosphoribosyltransferase); inosinate dehydrogenase |
| Modality | Small molecule |
| Therapeutic area | Oncology; Immunology |
| Phase | Phase 3 |
Mechanism of action
Mercaptopurine is metabolized to thioinosinate monophosphate, which inhibits inosinate dehydrogenase and feedback-inhibits PRPP amidotransferase, blocking purine synthesis. The active metabolites also incorporate into DNA and RNA, disrupting nucleic acid synthesis and function in rapidly dividing cells, particularly lymphocytes and leukemic cells.
Approved indications
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Inflammatory bowel disease (Crohn's disease, ulcerative colitis)
Common side effects
- Myelosuppression (leukopenia, thrombocytopenia, anemia)
- Hepatotoxicity
- Nausea and vomiting
- Immunosuppression and increased infection risk
- Pancreatitis
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)
- Effectiveness of Ozanimod in Patients With Steroid-Dependent Ulcerative Colitis
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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