The MDS Beacon™

Strategies for the optimization of epigenetic therapies for myelodysplastic syndromes were covered at the 10th International Symposium on Myelodysplastic Syndromes (ISMDS). Several speakers presented their work, including Valeria Santini from the University of Florence, who discussed the activity of Vidaza (azacitidine) and Dacogen (decitabine) in the treatment of myelodysplastic syndromes (MDS).

Epigenetic therapy is the use of drugs to correct mistakes in the production of protein from DNA. These mistakes are commonly the result of environmental factors, and result in the production of defective cells. Vidaza and its analog Dacogen are demethylating agents used in epigenetic therapy. Demethylating agents turn on genes that were previously silenced which helps the body develop healthy cells.

Currently Vidaza is approved for high-risk MDS patients in Europe. The classification of low-risk and high-risk is determined by the International Prognostic Scoring System (IPSS). Survival medians range from 5.7 years for patients with low-risk, to 0.4 years for patients with high-risk MDS.

Santini has pioneered a new use for Vidaza in low-risk MDS patients. In her study, low-risk patients achieved the same percentage response to Vidaza and Dacogen as high-risk patients. Initially, patients were transfusion dependent and had received prior treatments such as growth factors, erythropoietin (EPO), and chemotherapy, but were unresponsive to these treatments.

“We had quite a high response rate,” states Santini. The rate of response for patients who received more than four cycles of treatment with Vidaza was greater than 50 percent. Patients either lowered their transfusion dependence or became transfusion independent.

Since this study was conducted, Vidaza has been used to treat low-risk, resistant MDS patients in Italy.

“We still have a long way to go,” said Santini. A better understanding of why demethylating agents are effective in patients with low-risk MDS is required before the best treatment schedule and dose can be determined for these patients.

For more information, please see the MDS Foundation Web site.