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Updated: Jun 5, 2026

Assays for Validating Histone Acetyltransferase Inhibitors
Published on: August 6, 2020
Histone deacetylase (HDAC) inhibitors in recent clinical trials for cancer therapy
Abstract:
Heritable changes in gene expression that are not based upon alterations in the DNA sequence are defined as epigenetics. The most common mechanisms of epigenetic regulation are the methylation of CpG islands within the DNA and the modification of amino acids in the N-terminal histone tails. In the last years, it became evident that the onset of cancer and its progression may not occur only due to genetic mutations but also because of changes in the patterns of epigenetic modifications. In contrast to genetic mutations, which are almost impossible to reverse, epigenetic changes are potentially reversible. This implies that they are amenable to pharmacological interventions. Therefore, a lot of work in recent years has focussed on the development of small molecule enzyme inhibitors like DNA-methyltransferase inhibitors or inhibitors of histone-modifying enzymes. These may reverse misregulated epigenetic states and be implemented in the treatment of cancer or other diseases, e.g., neurological disorders. Today, several epigenetic drugs are already approved by the FDA and the EMEA for cancer treatment and around ten histone deacetylase (HDAC) inhibitors are in clinical development. This review will give an update on recent clinical trials of the HDAC inhibitors used systemically that were reported in 2009 and 2010 and will present an overview of different biomarkers to monitor the biological effects.
Insights
Epigenetic modifications, unlike genetic mutations, are reversible and offer potential cancer treatments. This review highlights recent clinical trials of histone deacetylase (HDAC) inhibitors and biomarkers for epigenetic drug development.
Area of Science:
- Epigenetics and Molecular Oncology
- Pharmacological Interventions in Disease
Background:
- Epigenetics involves heritable gene expression changes without altering DNA sequence.
- Key mechanisms include DNA methylation and histone modifications.
- Epigenetic alterations are increasingly recognized as drivers of cancer progression.
Purpose of the Study:
- To review recent clinical trials (2009-2010) of systemically administered histone deacetylase (HDAC) inhibitors.
- To provide an overview of biomarkers for monitoring epigenetic drug effects.
- To discuss the potential of epigenetic modifications as therapeutic targets.
Main Methods:
- Review of systemic histone deacetylase (HDAC) inhibitor clinical trials reported in 2009 and 2010.
- Analysis of biomarkers used to assess biological effects of epigenetic interventions.
Main Results:
- Several epigenetic drugs, including HDAC inhibitors, are approved for cancer treatment.
- Numerous HDAC inhibitors are in clinical development, indicating significant therapeutic interest.
- Biomarkers are crucial for monitoring the efficacy and biological impact of these agents.
Conclusions:
- Epigenetic modifications represent a promising, potentially reversible therapeutic avenue for cancer and other diseases.
- Histone deacetylase (HDAC) inhibitors are a key class of epigenetic drugs with ongoing clinical investigation.
- Further research into biomarkers will enhance the development and application of epigenetic therapies.
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