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Old 03-21-2013, 10:04 PM
Larry Holler Larry Holler is offline
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Join Date: Mar 2013
Posts: 3
10 yr Member
Larry Holler Larry Holler is offline
New Member
 
Join Date: Mar 2013
Posts: 3
10 yr Member
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I will try to bring GM1 ganglioside into perspective so all can understand where this potential treatment stands today. GM1 ganglioside was pioneered by an Italian pharma company called Fidia. They produced GM1 and many derivitive of GM1 including Liga 20 (a derivitive that is useful for Parkinson's) from cow brains havested at slaughter. Unfortunately, when Mad Cow disease came along, they went out of business and the clinical trials they had in progress limped to their conclusions, including the Parkinson's trial. GM1 ganglioside cannot be synthesized and attempts at semisynthetic molecules have also not proved currently feasible. We have worked for many years to develope a alternate animal source that the FDA will accept for ganglioside production. We have a flock of sheep that have a genetic condition called GM1 gangliosidosis. Affected lambs accumulate 40x normal levels of GM1 ganglioside in their brain and other tissues. Our GM1 is produced for research use by a company called Avanti Polar Lipids. We have previously worked with a VC group to develope our model for GM1 production for use in Parkinsons, but the venture fell apart when it became apparent that they were more interested in money than working to get the chemical approved for Parkinson's. We still haven't given up, and have continued to struggle to maintain the progress we made while working with this group. We have also started to explore working on Huntington's disease as it an orphan disease and approval is slightly more straight forward. Preliminary data with ovine GM1 in HD mice showed that mutant Huntintin protein returned to normal in two week on intraperitoneal treatment. Hopefully we can convince the researchers doing this work to continue to complete the rest of the studies to present to the FDA. Unfortunately, we are stuck trying to find them grant funding to finish the work. The sialidase treatment that has been funded is interesting, but the reality is it would take decades to ever get it approved, even if it does work. GM1 ganglioside has been in phase II clinical trials for applications including spinal cord injury and Parkinson's disease, so all that is really need is to show the sheep GM1 is the same as the cow (which it is) and hopefully the FDA will speed thing up since so much work has been done on GM1 and the only thing that is really missing is a safe source that the FDA will like, which we have. Every animal in our system has complete health records from birth to death and we are certified in the USDA Export Monitored Scrapie Program (disease similar to Mad cow). I would give you a link, but evidently I can't so use a search engine and check for sheep and GM1 ganglioside and you should be able to find us. Our company is called Glycoscience Research if you are interested in more information about GM1 ganglioside. Thanks.
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