Identifying Therapeutic Targets to Treat MDS

News Brief

Identifying Therapeutic Targets to Treat MDS

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In a group of blood cancers known as myelodysplastic syndrome (MDS), blood stem cells do not mature into blood cells. This results in anemia, frequent infections, and difficult-to-control bleeding.

Kira Gritsman, M.D., Ph.D., has received a four-year, $2.1 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases to study the role of the PI3 kinase (PI3K) signaling pathway in MDS development. Dr. Gritsman and colleagues have previously found that the PI3K pathway is inactive in some MDS patients. And in studies of a mouse model with deletions in multiple PI3K proteins, they discovered that these mice develop a blood disorder resembling MDS. Dr. Gritsman’s team will investigate how loss of PI3K activity leads to impaired maturation of stem cells and will also study other signaling pathways that regulate blood stem-cell maturation. In addition, the researchers will test therapies that might be able to treat MDS by correcting the PI3K pathway or the proteins that it regulates.

Dr. Gritsman is an associate professor of oncology, of medicine, and of cell biology at Einstein and an attending physician at Montefiore Health System. (1R01DK130895-01A1)