Research using cancer cell lines demonstrated that supplementing standard epigenetic therapy with vitamin C enhanced the drug’s antineoplastic action, according to a report in the Proceedings of the National Academy of Sciences.1

In the cell lines, the addition of vitamin C to decitabine treatment increased cancer cell growth inhibition, triggering cellular self-destruction.

In an ongoing pilot clinical trial in Copenhagen, Denmark, investigators are combining vitamin C with azacitidine, the standard-of-care therapy for myeloid dysplastic syndrome (MDS) and acute myeloid leukemia (AML). The approach would also correct a vitamin C deficiency, which occurs in many patients with cancer.

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If the pilot trial is successful, the investigators plan a larger trial to explore the potential of this strategy to improve treatments for AML and MDS.

A blinded clinical trial that measures azacitidine combined with vitamin C vs placebo would be needed to validate any benefits of adding vitamin C to a treatment. Furthermore, there is no evidence that these benefits would work for other cancers or other chemotherapies.

Preliminary results are expected by spring or summer 2017.


1. Liu M, Ohtani H, Zhou W, et al. Vitamin C increases viral mimicry induced by 5-aza-2′-deoxycytidine. Proc Natl Acad Sci U S A. 2016. doi: 10.1073/pnas.1612262113. [Epub ahead of print]