This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Ataxia

GABAA receptor

In vitro and mouse studies suggest GABAA receptor agonists could help treat motor dysfunctions associated with Angelman syndrome, a neurogenetic disorder caused by deletion or inactivation of genes on chromosome 15. In a mouse model of Angelman syndrome, the GABAA receptor agonist gaboxadol restored normal signaling in cerebellar brain slices and reversed cerebellar ataxia. Next steps include testing the effects of gaboxadol on other brain functions.
Merck & Co. Inc. and H. Lundbeck A/S discontinued development of gaboxadol in insomnia after Phase III trials showed that the molecule's profile did not support further development.
At least 13 companies have GABAA agonists in development stages ranging from preclinical to marketed for various neurological indications.

SciBX 6(1); doi:10.1038/scibx.2013.19
Published online Jan. 10, 2013

Unpatented; unavailable for licensing

Egawa, K. et al. Sci. Transl. Med.; published online Dec. 5, 2012;
doi:10.1126/scitranslmed.3004655
Contact: Atsuo Fukuda, Hamamatsu University School of Medicine, Hamamatsu, Japan
e-mail:
axfukuda@hama-med.ac.jp

Contact: Kiyoshi Egawa, Massachusetts General Hospital, Boston, Mass.
e-mail:
cdh67560@par.odn.ne.jp