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What treatments for cancer does research on epigenetics offer?

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Through epigenetic therapy, researchers are finding ways to repair epigenetic changes by specifically targeting abnormal cells while minimizing damage to normal cells. For example, standard chemotherapy drugs for cancer treatment typically use highly toxic doses that affect healthy cells along with abnormal cells. By contrast, an epigenetic-acting drug for myelodysplastic syndrome, a cancer typically found in older patients, has minimal toxicity because a smaller dose can be effective.1

Research shows that the four epigenetic drugs approved by the U.S. Food and Drug Administration (FDA) for cancer treatment have the greatest efficacy in hematopoietic (pronounced hee-muh-toh-poi-ET-ik) malignancies, or those that relate directly to the formation of blood cells.2

Types of Epigenetic Therapies for Cancer

The Food and Drug Administration has approved the following epigenetic therapies for cancer treatment:

Azacitidine (Vidaza)

A type of chemotherapy that when given at very low doses does not kill cancer cells, but instead appears to coax genes to switch on to fight cancerous cells. Recent research shows promise of azacitidine’s effectiveness when treating non-small cell lung cancer patients.3

Decitabine (Dacogen)

A drug used to treat myelodysplasia (mi-uh-loh-dis-PLEY-zhuh), a condition affecting the bone marrow’s ability to produce red and white blood cells4, and leukemia. Researchers have shown that decitabine can produce remissions or clinical improvements in greater than 30% of treated patients.2

Vorinostat (Zolinza)

Approved for the treatment of patients with a form of immune-system cancer (cutaneous T-cell lymphoma) who continue to have progressive, persistent, or recurrent disease despite being treated with at least two different therapies. Vorinostat is the first in a new class of anticancer agents that inhibit enzymes called histone deacetylases (HDAC), which have a key role in the epigenetic regulation of gene expression.5

Romidepsin (Istodax)

A type of epigenetic therapy used as an injection. Like vorinostat, romidepsin is a member of the HDAC class of cancer drugs and is approved to treat peripheral T-cell lymphoma in patients who have received at least one prior therapy.6


  1. NOVA. (2012). Epigenetic therapy. Retrieved June 4, 2012, from http://www.pbs.org/wgbh/nova/body/epigenetic-therapy.html External Web Site Policy [top]
  2. Boumber, Y., & Issa, J. P. (2011). Epigenetics in cancer: What’s the future? Oncology (Williston Park), 25, 220-226, 228. Retrieved July 24, 2012, from http://www.ncbi.nlm.nih.gov/pubmed/21548464 [top]
  3. Juergens, R. A., Wrangle, J., Vendetti, F. P., Murphy, S. C., Zhao, M., Coleman, B., et al. (2011). Combination epigenetic therapy has efficacy in patients with refractory advanced non-small cell lung cancer. Cancer Discovery, 1, 598-607. Retrieved July 24, 2012, from http://www.ncbi.nlm.nih.gov/pubmed/22586682 [top]
  4. National Library of Medicine. (2012). Myelodysplastic syndromes. Retrieved July 9, 2012, from http://www.nlm.nih.gov/medlineplus/myelodysplasticsyndromes.html [top]
  5. Grant, S., Easley, C., & Kirkpatrick, P. (2007). Vorinostat. Nature Reviews Drug Discovery, 6, 21-22. [top]
  6. Cutaneous Lymphoma Foundation. (2011). FDA grants accelerated approval of ISTODAX [press release]. Retrieved June 5, 2012, from http://www.clfoundation.org/newsroom/fda-grants-accelerated-approval-of-istodax External Web Site Policy [top]

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Last Updated Date: 11/30/2012
Last Reviewed Date: 10/28/2013
Vision National Institutes of Health Home BOND National Institues of Health Home Home Storz Lab: Section on Environmental Gene Regulation Home Machner Lab: Unit on Microbial Pathogenesis Home Division of Intramural Population Health Research Home Bonifacino Lab: Section on Intracellular Protein Trafficking Home Lilly Lab: Section on Gamete Development Home Lippincott-Schwartz Lab: Section on Organelle Biology