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Updated: Jul 3, 2026

Assays for Validating Histone Acetyltransferase Inhibitors
Published on: August 6, 2020
Promising antitumor activity with MGCD0103, a novel isotype-selective histone deacetylase inhibitor
Christophe Le Tourneau1, Lillian L Siu
1University of Toronto, University Health Network, Princess Margaret Hospital, Division of Medical Oncology and Hematology, 610 University Avenue, Suite 5-718, Toronto M5G 2M9, Canada.
Background:
Histone deacetylases (HDACs), which target histones as well as non-histone proteins as substrates, have the potential to regulate aberrant gene expression and restore normal growth control in malignancies.
Objective:
This review provides an updated summary of preclinical and clinical experience with the oral isotype-selective HDAC inhibitor MGCD0103 in cancer.
Methods:
Data presented in abstract form from international conferences or journal articles found within a PubMed search of article up to May 2008 are described in this review.
Results/Conclusions:
MGCD0103 appears tolerable and exhibits favorable pharmacokinetic and pharmacodynamic profiles with evidence of target inhibition in surrogate tissues. Clinical and pharmacodynamic data support a three-times-weekly administration at a 90-mg fixed dose. MGCD0103 displays promising antitumor activity in hematological and lymphoproliferative diseases.
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