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[Buddhahead]

Mount Sinai preclinical program flags a potential drug strategy for MS in Fierce Biotech Research, March 2, 2015 | By John Carroll

Excerpts:
"Testing a theory that the molecule that transports proteins from the nucleus of a cell to the cytoplasm is altered in neurodegenerative diseases like multiple sclerosis, a group of investigators at the Icahn School of Medicine at Mount Sinai say that they have successfully completed an animal study that tested drugs that could block the molecule and perhaps repair nerve cell damage that occurs in MS.

That molecule is called XPO1, or CRM1. Using money from the NIH and the National Multiple Sclerosis initiative Fast-Forward, the researchers tested two chemicals--KPT-276 and KPT-350, from Karyopharm Therapeutics ($KPTI)--and found that it prevented the target protein from carrying "cargo" out of the nucleus of nerve cells in a mouse model. The shutdown helped prevent the structural damage of the key cells, which are damaged by MS when an autoinflammatory process attacks the myelin sheaths that protect nerve cells.

The compounds also stopped inflammatory cells from multiplying, according to a statement on the work, thereby reducing inflammation. Mice with hindlimb paralysis demonstrated signs of recovery in the study, though as with all mouse studies it should be noted that rodents can have amazing recoveries that never apply to humans. The results were published online in Nature Neuroscience on Feb. 23."

Article at:
http://www.fiercebiotechresearch.com/story/mount-sinai-preclinical-program-flags-potential-drug-strategy-ms/2015-03-02?spMailingID=11682764&spUserID=NzczNzQyMjg0MDgS1&spJobID=463229527&spReportId=NDYzMjI5NTI3S0