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5-Azacytidin
5-Azacytidine is a DNA methyltransferase inhibitor that reduces DNA methylation and promotes re-expression of silenced tumor suppressor genes.
5-Azacytidine is a DNA methyltransferase inhibitor that reduces DNA methylation and promotes re-expression of silenced tumor suppressor genes. Used for Myelodysplastic syndromes, Acute myeloid leukemia.
At a glance
| Generic name | 5-Azacytidin |
|---|---|
| Sponsor | Technische Universität Dresden |
| Drug class | DNA methyltransferase inhibitor |
| Target | DNA methyltransferase (DNMT) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
5-Azacytidine is a cytidine analog that incorporates into DNA during replication and inhibits DNA methyltransferase (DNMT) activity, leading to hypomethylation of CpG islands. This reactivates epigenetically silenced genes, including tumor suppressors, and can induce differentiation and apoptosis in myelodysplastic and other cancer cells. It also has immunomodulatory effects that enhance anti-tumor immunity.
Approved indications
- Myelodysplastic syndromes
- Acute myeloid leukemia
Common side effects
- Myelosuppression (anemia, thrombocytopenia, neutropenia)
- Nausea and vomiting
- Fatigue
- Injection site reactions
- Fever
Key clinical trials
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 |