This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Epilepsy

Estrogen receptor

Mouse studies suggest estradiol or related drugs could be used to treat infantile spasms syndrome (ISS). Estradiol mediates long-term gene expression changes through the estrogen receptor. In a mouse model of ISS with deficient interneuron development, estradiol given in an early postnatal window decreased motor spasms and seizures compared with vehicle or treatment at a later time. In the mice treated in the early window,
neuropeptide Y
(Npy)+ cortical interneuron and choline acetyltransferase-positive striatal interneuron numbers were restored. Next steps include studies to refine dosing levels, duration and timing of estradiol treatment.
More than 20 companies market ligands for the estrogen receptor for various indications.

SciBX 7(6); doi:10.1038/scibx.2014.179
Published online Feb. 13, 2014

Unpatented; mouse model available for licensing from Baylor College of Medicine

Olivetti, P.R. et al. Sci. Transl. Med.; published online Jan. 22, 2014;
doi:10.1126/scitranslmed.3007231
Contact: Jeffrey L. Noebels, Baylor College of Medicine, Houston, Texas
e-mail:
jnoebels@bcm.edu