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Myelodysplastic syndrome (MDS)


Studies in cell culture and patients suggest antagonizing p53 could help treat the chromosome 5q deletion (del(5q)) subtype of MDS. Patients with MDS can develop anemia and require blood transfusions. In erythroid precursors from patients with del(5q) MDS, an antisense oligonucleotide that decreases p53 levels increased erythroid cell proliferation compared with a control oligonucleotide. In patients with Revlimid lenalidomide-resistant del(5q) MDS who are dependent on blood transfusions, Revlimid plus the p53 antagonist dexamethasone increased erythropoiesis compared with baseline and restored transfusion independence in five of eight patients. Next steps include starting a clinical trial this month in patients with lower-risk MDS.
Celgene Corp. markets Revlimid to treat MDS, multiple myeloma (MM) and anemia.

SciBX 6(39); doi:10.1038/scibx.2013.1092
Published online Oct. 10, 2013

Provisional patent application filed; available for licensing

Caceres, G. et al. Proc. Natl. Acad. Sci. USA; published online Sept. 16, 2013;
Contact: Alan F. List, H. Lee Moffitt Cancer Center & Research Institute, Tampa, Fla.