Reprogramming adult cells into induced pluripotent stem cells (iPS cells) has been one of the most lauded discoveries in stem cell science. There is concern, however, with the potential utility of these cells since the protocol by which they are made typically uses multiple factors and a viral delivery method that may be questionable in terms of safety in a clinical setting. In order to identify small molecules that might replace these reprogramming factors, HSCI Principal Faculty member Kevin Eggan, PhD, and HSCI Director of Translational Medicine Lee Rubin, PhD, and colleagues conducted a chemical screen to identify small molecules that could replace the factor Sox 2 in the reprogramming process. Their recently published paper in the journal Cell Stem Cell reports the discovery of a small molecule they identified that takes the place of Sox2 in reprogramming by inhibiting the Tgf-beta signaling pathway and activating the factor Nanog. Such findings may help move iPS cells closer to the clinic by increasing their safety and enabling them to realize their potential therapeutic potential, such as in creating patient-specific cell lines.

Ichida, J.K., Blanchard, J., Lam, K., Son, E.Y., Chung, J.E., Egli, D., Loh, K.M., Carter, A.C., Di Giorgio, F.P., Koszka, K., Huangfu, D., Akutsu, H., Liu, D.R., Rubin, L.L., Eggan, K. (2009). A Small-Molecule Inhibitor of Tgf-beta Signaling Replaces Sox2 in Reprogramming by Inducing Nanog. Cell Stem Cell. 2009 Oct 7. [Epub ahead of print]