Decitabine has been the focus of numerous trials in a range of indications and was approved by the EMA for patients over 65 years with acute myeloid leukemia in 2012 . Genistein is derived from soy products  and has orphan drug designation for Sanfilippo syndrome. It is not yet indicated by the EMA or US FDA for medicinal use. The combination (DEC-GEN) has been previously investigated in non-small-cell lung cancer  in adults.
In a study analyzing DEC-GEN in solid tumors and recurrent/refractory leukemia, principal investigators Noël Raynal and Henrique Bittencourt (Université de Montréal, QC, Canada) will be assessing the clinical benefit of DEC-GEN in children who do not respond to standard treatments – the first pediatric study of DEC-GEN for this indication . They will also investigate the effect of the treatment on quality of life.
Cancer treatments are notorious for their side effects, but investigators are optimistic that DEC-GEN will yield reduced toxicity due to the harmonious actions of the two agents. Decitabine acts as a DNA methyltransferase inhibitor, allowing it to interrupt malignant epigenetic changes . Although the mechanism of action of genistein is less well defined, studies have shown increased DNA methylation  and an effect on cell-cycle regulation  and apoptosis . As an oral agent, genistein may prove less distressing, especially in a pediatric population.
According to Clinical Trials.gov (NCT02499861), the study aims to assess 12 patients in the first year before expanding to a Phase II design, which will look to recruit 27 patients in total once the optimal dose is confirmed. The study is being supported by Gateway for Cancer Research, Pharmascience and DSM Nutritional Products.
There is hope that the growing understanding of cancer epigenomics may provide a rationale for further studies to investigate previously researched compounds in new indications. With new treatment options urgently needed for difficult to treat patients, these results (and those from similar studies) will be eagerly awaited.
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