BioCentury
ARTICLE | Distillery Therapeutics

Neurology

May 16, 2018 5:56 PM UTC

Patient sample and mouse studies suggest inhibiting GSK3B could help treat Rett syndrome. In postmortem cerebellum tissue samples from Rett syndrome patients, levels of GSK3B were higher than in samples from unaffected subjects. In a mouse model of the disease, a tool compound GSK3B inhibitor increased survival and mobility and decreased disease progression, breathing abnormalities, tremors and inflammation in the cerebellum and pons compared with vehicle. Next steps include testing undisclosed GSK3B inhibitors in patients with Rett syndrome.

Jeil Pharmaceutical Co. Ltd. has the GSK3B inhibitor JGK 263 in preclinical testing to treat Alzheimer’s disease and amyotrophic lateral sclerosis...