Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Autism

Eukaryotic translation initiation factor 4E (eIF4E); eIF4E binding protein 2 (eIF4EBP2)

Mouse studies suggest inhibition of eIF4E signaling could help treat autism spectrum disorder (ASD). In mice, knocking out eif4ebp2 led to electrophysiological abnormalities and autistic behaviors. In those autistic mice, a small molecule inhibitor of eIF4E signaling reversed the electrophysiological abnormalities and decreased autistic behaviors compared with vehicle. Next steps include developing mouse models with brain region-specific or conditional knockout of eif4ebp2.
Isis Pharmaceuticals Inc.'s eIF-4E ASO, a second-generation antisense compound targeting eIF4E, is in Phase II testing to treat prostate cancer and non-small cell lung cancer (NSCLC).
Clavis Pharma ASA and Translational Therapeutics Inc. have TRX-201, a Lipid Vector Technology (LVT) derivative of ribavirin that inhibits eIF4E, in preclinical development to treat thyroid cancer.

SciBX 5(47); doi:10.1038/scibx.2012.1239
Published online Dec. 6, 2012

Eif4ebp2 knockout mouse model unpatented; available for licensing

Gkogkas, C.G. et al. Nature; published online Nov. 21, 2012;
doi:10.1038/nature11628
Contact: Nahum Sonenberg, McGill University, Montreal, Quebec, Canada
e-mail:

nahum.sonenberg@mcgill.ca