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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
PubMed

Insights

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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