Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Parkinson's disease (PD)

a-Synuclein (SNCA); NEDD4 family E3 ubiquitin protein ligase (RSP5); neural precursor cell expressed developmentally downregulated 4 (NEDD4; NEDD4-1)

In vitro and ex vivo studies identified an N-arylbenzimidazole (NAB) that could help treat PD. In a yeast-based screen, the NAB reversed SNCA pathology, including reactive oxygen species (ROS) generation, vesicular trafficking disruption and SNCA foci formation. In rat and patient-derived ex vivo neuronal models of PD, the most potent NAB decreased SNCA pathology compared with vehicle by promoting RSP5- and NEDD4-mediated vesicular trafficking. In cortical neurons derived from patient induced pluripotent stem (iPS) cells expressing mutant SNCA, the NAB also corrected protein nitration and endoplasmic reticulum-associated degradation. Next steps could include testing the NAB in animal models of PD.

SciBX 6(46); doi:10.1038/scibx.2013.1327
Published online Dec. 5, 2013

Patent and licensing status unavailable for both studies

Tardiff, D.F. et al. Science; published online Oct. 24, 2013;
doi:10.1126/science.1245321
Chung, C.Y. et al. Science; published online Oct. 24, 2013;
doi:10.1126/science.1245296
Contact: Susan Lindquist, Whitehead Institute for Biomedical Research, Cambridge, Mass.
e-mail:

lindquist_admin@wi.mit.edu