This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Down syndrome

Smoothened (SMO)

Mouse studies suggest agonizing SMO could be useful for treating neurodevelopmental deficits in patients with Down syndrome. In a mouse model of Down syndrome, cerebellar and hippocampal development were compromised. Perinatal treatment with a Smo-agonizing compound improved cerebellar and hippocampal development and performance in motor and learning tasks. Next steps include mouse studies to identify the brain tissues most directly affected by SMO agonists and dose-ranging and teratogenicity studies (see Hedgehog battles Down syndrome, page 6).

SciBX 6(37); doi:10.1038/scibx.2013.1036
Published online Sept. 26, 2013

Unpatented; licensing status not applicable

Das, I. et al. Sci. Transl. Med.; published online Sept. 4, 2013;
doi:10.1126/scitranslmed.3005983
Contact: Roger H. Reeves,
The Johns Hopkins University School of Medicine, Baltimore, Md.
e-mail:
rreeves@jhmi.edu