Trials with 'epigenetic' drugs: an update
Angela Nebbioso1, Vincenzo Carafa, Rosaria Benedetti
1Dipartimento di Patologia Generale, Seconda Università degli Studi di Napoli, Vico L. de Crecchio 7, Napoli 80138, Italy.
Molecular Oncology
|October 30, 2012
Summary
Epigenetic gene inactivation drives human diseases like cancer but is reversible. Re-expressing these genes with novel epi-drugs offers potential therapeutic strategies, with some already FDA-approved for cancer treatment.
Area of Science:
- Molecular Biology
- Genetics
- Oncology
Background:
- Epigenetic alterations, including DNA methylation and histone modifications, are key drivers of cellular dysfunction and disease, particularly cancer.
- These epigenetic changes impact gene expression and are linked to disease progression, unlike genetic mutations.
- Epigenetic modifications are potentially reversible, offering therapeutic opportunities.
Purpose of the Study:
- To explore the biological characteristics of epigenetic molecules.
- To analyze the mechanisms of action for epigenetic drugs.
- To review the current clinical applications of epigenetic therapies in disease treatment.
Main Methods:
- Literature review focusing on epigenetic mechanisms (DNA methylation, histone modifications, non-coding RNAs).
- Analysis of small molecules targeting epigenetic modifications (epi-drugs).
- Examination of clinical trial data and regulatory approvals (FDA, EMA) for epi-drugs in cancer therapy.
Main Results:
- Epigenetic inactivation of critical genes is a common feature in human pathologies, especially cancer.
- Re-expression of epigenetically silenced genes can reverse disease states or enhance treatment efficacy.
- Several epi-drugs are in clinical trials, with some approved by regulatory agencies for cancer treatment.
Conclusions:
- Epigenetic mechanisms play a crucial role in disease development and progression.
- Targeting epigenetic modifications with epi-drugs represents a promising therapeutic avenue.
- The clinical application of epi-drugs is expanding, offering new hope for patients with various diseases, particularly cancer.
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