Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.



Azacitidine (Vidaza; Pharmion), an inhibitor of DNA methylation, was approved by the US FDA for the treatment of myelodysplastic syndromes in May 2004. It is the first drug to be approved by the FDA for treating this rare family of bone-marrow disorders, and has been given orphan-drug status. It is also a pioneering example of an agent that targets 'epigenetic' gene silencing, a mechanism that is exploited by cancer cells to inhibit the expression of genes that counteract the malignant phenotype.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Buy article

Get time limited or full article access on ReadCube.


All prices are NET prices.

Figure 1: Azacitidine and DNA methylation.


  1. List, A. F., Vardiman, J., Issa, J. -P. & DeWitte, T. M. Myelodysplastic syndromes. Hematology (Am. Soc. Hematol. Educ. Program) 297–317 (2004).

  2. Hofmann, W. -K. et al. Myelodysplastic syndromes. Hematol. J. 5, 1–8 (2004).

    Article  Google Scholar 

  3. Herman, J. G. & Baylin, S. B. Gene silencing in cancer in association with promoter hypermethylation. N. Engl. J. Med. 349, 2042–2054 (2003).

    Article  CAS  Google Scholar 

  4. Egger, G., Liang, G., Aparicio, A. & Jones, P. A. Epigenetics in human disease and prospects for epigenetic therapy. Nature 429, 457–463 (2004).

    Article  CAS  Google Scholar 

  5. FDA labelling information [online], <> (2004).

  6. Greenberg, P. et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 89, 2079–2088 (1997).

    CAS  PubMed  Google Scholar 

  7. Silverman, L. R. et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J. Clin. Oncol. 20, 2429–2440 (2002).

    Article  CAS  Google Scholar 

  8. Faderl, S. & Kantarjian, H. M. Novel therapies for myelodysplastic syndromes. Cancer 101, 226–241 (2004).

    Article  CAS  Google Scholar 

  9. Anzai, H., Frost, P., & Abbruzzese, J. L. Synergistic cytotoxicity with 2′-deoxy-5-azacytidine and topotecan in vitro and in vivo. Cancer Res. 52, 2180–2185 (1992).

    CAS  PubMed  Google Scholar 

  10. Kantarjian, H. et al. Phase 2 clinical and pharmacologic study of clofarabine in patients with refractory or relapsed acute leukemia. Blood 102, 2379–2386 (2003).

    Article  CAS  Google Scholar 

  11. List, A. et al. Efficacy of lenalidomide in myelodysplastic syndromes. N. Engl. J. Med. 352, 549–557 (2005).

    Article  CAS  Google Scholar 

  12. Bird, A. DNA methylation patterns and epigenetic memory. Genes Dev. 16, 6–21 (2002).

    Article  CAS  Google Scholar 

  13. Kuendgen, A. et al. Treatment of myelodysplastic syndromes with valproic acid alone or in combination with all-trans retinoic acid. Blood 104, 1266–1269 (2004).

    Article  CAS  Google Scholar 

  14. Wijermans, P. et al. Low-dose 5-aza-2′-deoxycytidine, a DNA hypomethylating agent, for the treatment of high-risk myelodysplastic syndrome: a multicenter phase II study in elderly patients. J. Clin. Oncol. 18, 956–962 (2000).

    Article  CAS  Google Scholar 

  15. Claus, R. & Lubbert, M. Epigenetic targets in hematopoietic malignancies. Oncogene 22, 6489–6496 (2003).

    Article  CAS  Google Scholar 

  16. Saiki, J. H. et al. 5-azacytidine in acute leukemia. Cancer 42, 2111–2114 (1978).

    Article  CAS  Google Scholar 

  17. Issa, J. P. et al. Phase I study of low-dose prolonged exposure schedules of the hypomethylating agent 5-aza-2′-deoxycytidine (Decitabine) in hematopoietic malignancies. Blood 103, 1635–1640 (2004).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations


Corresponding authors

Correspondence to Jean-Pierre J. Issa or Peter Kirkpatrick.

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Issa, JP., Kantarjian, H. & Kirkpatrick, P. Azacitidine. Nat Rev Drug Discov 4, 275–276 (2005).

Download citation

  • Issue Date:

  • DOI:

This article is cited by


Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing