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This combination therapy consists of azacitidine (5-aza-cytidine) and thalidomide, currently being investigated by the Tel-Aviv Sourasky Medical Center for the treatment of higher-risk myelodysplastic syndromes (MDS). Azacitidine is a pyrimidine nucleoside analog that functions as a DNA methyltransferase inhibitor, promoting DNA hypomethylation and the restoration of normal hematopoiesis by reactivating silenced tumor suppressor genes. Thalidomide is an immunomodulatory drug (IMiD) that exerts anti-angiogenic, anti-inflammatory, and pro-apoptotic effects, partially through its interaction with the cereblon E3 ubiquitin ligase complex. The dual-action approach of this regimen is intended to enhance therapeutic efficacy in patients with intermediate-2 or high-risk MDS who often have poor prognoses with monotherapy.
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