Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Ataxia

Potassium channel KCa2.2 (KCNN2); KCNN3; ataxin 2 (ATXN2; SCA2)

Mouse studies suggest positive allosteric modulators (PAMs) of KCNN2 could help treat spinocerebellar ataxia type 2. In SCA2-expressing transgenic mouse models of the condition, Purkinje neurons showed aberrant firing patterns. In cerebellar slices from those mice, NS13001, a PAM of KCNN2 and KCNN3, normalized Purkinje neuron firing patterns. In the mouse model, a PAM optimized for potency against KCNN2 increased motor performance compared with pretreatment baselines. NeuroSearch A/S plans to test NS13001 in other models of cerebellar and/or episodic ataxia.

SciBX 5(44); doi:10.1038/scibx.2012.1165
Published online Nov. 8, 2012

Patented by NeuroSearch; available for licensing

Kasumu, A.W. et al. Chem. Biol.;
published online Oct. 26, 2012;
doi:10.1016/j.chembiol.2012.07.013
Contact: Ilya Bezprozvanny,
The University of Texas Southwestern Medical Center, Dallas, Texas
e-mail:
ilya.bezprozvanny@utsouthwestern.edu
Contact: Palle Christophersen, NeuroSearch A/S, Ballerup, Denmark
e-mail:
pc@neurosearch.com