Drug intelligence / Profile preview

vorinostat + decitabine

Development stage
Unknown
Lead developer
Merck
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

Vorinostat + decitabine is a **combination therapy** comprised of two small molecules with epigenetic modifying activity, typically studied in hematologic malignancies, notably in acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and acute lymphoblastic leukemia (ALL). **Vorinostat** is a histone deacetylase inhibitor (HDACi) that induces hyperacetylation of histones, resulting in altered gene expression, apoptosis, and inhibition of cell proliferation. **Decitabine** is a DNA methyltransferase inhibitor (DNMTi) that causes DNA hypomethylation and reactivation of silenced genes, including tumor suppressors. The combination acts *synergistically* to reverse epigenetic gene silencing and sensitize malignant cells to chemotherapy. This drug combination has shown tolerability and preliminary efficacy in both adult and pediatric relapsed/refractory AML, MDS, and ALL clinical studies[1][2][3][4][5][7][9].

02

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)HDAC (HDAC family)

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