Preclinical and Clinical Studies on 5-Aza-2′-Deoxycytidine, a Potent Inhibitor of DNA Methylation, in Cancer Therapy

  • Richard L. Momparler
Part of the Medical Intelligence Unit book series (MIUN)


The preclinical and clinical investigations by the author on the antineoplastic activity of 5-Aza-2′-deoxycytidine (5AZA), a potent inhibitor of DNA methylation are reviewed. These include studies on the molecular, cellular and animal pharmacology of 5AZA. These preclinical studies indicated that 5AZA has enormous potential in cancer therapy. This potential is supported by reports in the literature indicate that 5AZA can reactivate many different types of genes that suppress tumorigenesis and were silenced by aberrant DNA me-thylation. However, the potential still remains to be demonstrated in clinical investigations where the author observed interesting responses in both patients with leukemia and lung can-cer. Several suggestions are made concerning the design of the optimal dose-schedule for 5AZA in cancer therapy. Preclinical studies show that 5AZA in combination with inhibitors of his-tone deacetylase (HDI) show a synergistic interaction against neoplastic cells. 5AZA plus HDI may have the potential to be a very effective chemotherapeutic regimen in patients with cancer.


Acute Myeloid Leukemia Cytosine Arabinoside Cytidine Deaminase Antineoplastic Activity L1210 Leukemia 
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Copyright information

© and Kluwer Academic/Plenum Publishers 2005

Authors and Affiliations

  • Richard L. Momparler
    • 1
  1. 1.Department de PharmacologieUniversity de Montreal, Centre de Recherche, Hosptial Sainte-JustineMontrealCanada

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