Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Endocrine/metabolic disease

Diabetes

Pancreatic and duodenal homeobox 1 (PDX1; IPF1)

In vitro studies identified a small molecule that increases PDX1 expression and could help treat diabetes. In a gene expression assay using human pancreatic ductal adenocarcinoma cells, high throughput screening identified a glucose-based synthetic compound that increased PDX1 expression compared with vehicle and induced insulin production. DNA methylation and gene profiling studies showed that the compound induced histone modifications at the PDX1 gene and expression of the forkhead box A2 (FOXA2) transcription factor, both resulting in elevated PDX1 expression. Next steps include testing whether the resulting insulin-producing cells could replace b cells in an animal model of diabetes.

SciBX 7(2); doi:10.1038/scibx.2014.55
Published online Jan. 16, 2014

Unpatented; unavailable for licensing

Yuan, Y. et al. Chem. Biol.; published online Nov. 27, 2013;
doi:10.1016/j.chembiol.2013.10.013
Contact: Bridget K. Wagner, Broad Institute of MIT and Harvard, Cambridge, Mass.
e-mail:
bwagner@broadinstitute.org