Drug intelligence / Profile preview

decitabine + thrombopoietin

Development stage
Unknown
Lead developer
Astex Pharmaceuticals
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous, Subcutaneous
01

Overview

Decitabine + thrombopoietin is a combination therapy consisting of decitabine, a hypomethylating agent, and recombinant human thrombopoietin, a hematopoietic growth factor. Decitabine acts primarily as an inhibitor of DNA (cytosine-5)-methyltransferases, leading to global DNA hypomethylation and reactivation of silenced tumor suppressor genes. This results in decreased neoplastic cell proliferation and increased expression of genes involved in cell differentiation and apoptosis[1]. Thrombopoietin is the primary physiological regulator of platelet production; it stimulates megakaryocyte proliferation and maturation via activation of the thrombopoietin receptor (MPL). The combination has been studied for refractory prolonged isolated thrombocytopenia (RPIT) after allogeneic hematopoietic cell transplantation. Clinical trials have shown that this regimen improves platelet recovery rates and overall survival compared to conventional treatment or either agent alone[2][7]. Mechanistically, decitabine enhances megakaryocyte maturation and platelet release—effects that are further potentiated by co-administration with thrombopoietin[6][9].

02

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)TPOR (Thrombopoietin receptor)HDAC (HDAC family)DNMT3A (DNA methyltransferase 3 alpha)DNMT3B (DNA (cytosine-5)-methyltransferase 3B)

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