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5-Azacytidine/Azacitidine

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Small Molecules in Oncology

Part of the book series: Recent Results in Cancer Research ((RECENTCANCER,volume 184))

Abstract

5-Azacytidine is a pyrimidine nucleoside analog that has been discovered more than 40 years ago. Despite remarkable responses in the treatment of acute myeloid leukemias in the 1970s no earlier than 2004 has this agent been approved by the US FDA for the treatment of all subtypes of myelodysplatic syndromes (MDS). For the first time a drug was proven to alter the natural course of MDS, as demonstrated in three clinical trials conducted by the CALG B. Complete remission rates ranged between 10–17%, and more recently, a significant survival benefit for MDS patients treated with 5-Azacytidine could be established. The antineoplastic activity is due to incorporation into RNA with disruption of RNA metabolism, and inhibition of DNA methylation.

Strategies of combining epigenetic manipulation with other ‘new’ drugs aim at increasing the efficacy of the hypomethylating agents. Particularly histone deacetylase inhibitors have been deemed useful therapeutic partners, and preliminary results are promising.

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  1. Division of Blood and Marrow Transplantation, Stanford University, School of Medicine, 269, West Campus Drive CCSR, Stanford, CA, 94305, USA

    Antonia Müller

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  1. Antonia Müller
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  2. Mareike Florek
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Correspondence to Antonia Müller .

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  1. Medizinische Klinik III, SLK-Kliniken Heilbronn GmbH, Am Gesundbrunen 20-26, Heilbronn, 74078, Germany

    Uwe M. Martens

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Müller, A., Florek, M. (2010). 5-Azacytidine/Azacitidine. In: Martens, U. (eds) Small Molecules in Oncology. Recent Results in Cancer Research, vol 184. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-01222-8_11

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