Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Musculoskeletal disease

Myotonic dystrophy type 1 (DM1)

Glycogen synthase kinase 3b (GSK3B)

Mouse studies suggest inhibiting GSK3B could help treat DM1. In a mouse model of DM1, activated GSK3B was upregulated prior to the development of muscle weakness. In the mouse model, the nonspecific GSK3B inhibitor lithium increased muscle strength and decreased myotonia in skeletal muscles compared with no treatment. Next steps could include clinical testing of lithium or other GSK3B inhibitors in patients with DM1.
Jeil Pharmaceutical Co. Ltd.'s GSK3B inhibitor, JGK-216/263, is in discovery to treat Alzheimer's disease (AD) and amyotrophic lateral sclerosis (ALS).

SciBX 5(48); doi:10.1038/scibx.2012.1259
Published online Dec. 13, 2012

Findings unpatented; licensing status not applicable

Jones, K. et al. J. Clin. Invest.; published online Nov. 19, 2012;
doi:10.1172/JCI64081
Contact: Lubov T. Timchenko, Baylor College of Medicine, Houston, Texas
e-mail:
lubovt@bcm.tmc.edu