An estimated 13,000 people in the United States are diagnosed with myelodysplastic syndrome (MDS) annually and about 20,000 are diagnosed with acute myeloid leukemia (AML). Currently, only about half of patients with MDS and AML respond to the epigenetic therapy alone.
A simple adjustment to patients' therapeutic regimen may improve the effectiveness of the standard epigenetic treatment for myeloid dysplastic syndrome (MDS) and acute myeloid leukemia (AML). Combining vitamin C with a demethylating agent in patients with MDS and AML may improve the therapy's ability to combat cancers.
At this point, this effect has only been shown in cell lines, and the efficacy in patients can only be confirmed by conducting a rigorous and controlled clinical trial. A pilot clinical trial is underway to investigate the safety and effectiveness of this therapy. If successful, investigators plan to pursue further clinical studies. The investigators urge patience and caution patients to wait for the results of the clinical trial.
"If the pilot trial is successful, we plan to pursue a larger trial to explore this strategy's potential as a straightforward and cost-effective way to improve the existing therapy for AML and MDS," said Peter Jones, co-senior author of the PNAS study, chief scientific officer at Van Andel Research Institute (VARI) and co-leader of the Van Andel Research Institute-Stand Up To Cancer (VARI-SU2C) Epigenetics Dream Team.
"At the same time, we must urge patience and caution. Only a clinical trial that combines azacitidine with the blinded addition of either vitamin C or a placebo will give the true answer as to whether or not vitamin C increases the efficacy of azacitidine in patients. We must emphasize that our trial is limited to a certain subset of patients with MDS or AML on a specific therapeutic regimen. We do not have evidence that this approach is appropriate for other cancers or chemotherapies."
The proposed strategy reflects a continuing move toward combination therapies, particularly when it comes to epigenetic approaches, which target the mechanisms that control whether genes are switched "on" or "off." In cancer, these switches inappropriately activate or silence important genes, such as those that regulate cell growth and life cycle, ultimately leading to tumors. Epigenetic therapies are thought to work in two ways to fix these errors in cancer cells--by correcting the "position" of the gene switches and by making the cell appear as though it's infected by a virus, triggering the immune system.