This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Neurology

Sirtuin 1 (SIRT1)

In vitro and mouse studies suggest inhibiting SIRT1 could help treat myelination disorders. In a mouse model for lysolecithin-induced demyelination, genetic inactivation of Sirt1 increased remyelination compared with Sirt1 activation. In a mouse model for experimental autoimmune encephalomyelitis (EAE), Sirt1 inactivation delayed paralysis. Next steps include testing pharmacological inhibitors of SIRT1 in animals.
Elixir Pharmaceuticals Inc. and Siena Biotech S.p.A. have the SIRT1 inhibitor EX-527 in Phase I testing to treat Huntington's disease (HD).

SciBX 6(21); doi:10.1038/scibx.2013.525
Published online May 30, 2013

Findings unpatented; licensing status not applicable

Rafalski, V.A. et al. Nat. Cell Biol.; published online May 5, 2013;
doi:10.1038/ncb2735
Contact: Anne Brunet, Stanford University School of Medicine, Stanford, Calif.
e-mail:

anne.brunet@stanford.edu