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Pluripotent cell-specific inhibitors (PluriSIns) to reduce the tumorigenic risk of stem cell-based therapies

PluriSIns that are selectively toxic to human pluripotent stem cells could reduce the tumorigenic risk of stem cell-based therapies. Residual undifferentiated stem cells in stem cell-derived cell therapies can lead to teratoma formation. A screen of about 50,000 small molecules identified 15 PluriSIns that were cytotoxic to human embryonic stem cells (ESCs) and induced pluripotent stem (iPS) cells but not to differentiated cells derived from these stem cells. In a mixture of differentiated and undifferentiated stem cells injected into mice, none of the cell mixtures pretreated with the lead PluriSIn developed teratomas, whereas all vehicle-treated cell mixtures did. Next steps include examining the ability of PluriSIns to reduce the risk of teratoma formation in vivo.

SciBX 6(5); doi:10.1038/scibx.2013.126
Published online Feb. 7, 2013

Two patent applications filed; available for licensing through Yissum, the technology transfer company for The Hebrew University of Jerusalem, and Roche

Ben-David, U. et al. Cell Stem Cell; published online Jan. 9, 2013;
Contact: Nissim Benvenisty, The Hebrew University of Jerusalem, Jerusalem, Israel