GM1 ganglioside improved symptoms and slowed their progression during a two and a hal
 

http://www.newswise.com/articles/pro...roves-symptoms

Newswise — PHILADELPHIA—Treating Parkinson’s disease patients with the experimental drug GM1 ganglioside improved symptoms and slowed their progression during a two and a half-year trial, Thomas Jefferson University researchers report in a new study published online November 28 in the Journal of the Neurological Sciences.

Although the precise mechanisms of action of this drug are still unclear, the drug may protect patients’ dopamine-producing neurons from dying and at least partially restore their function, thereby increasing levels of dopamine, the key neurochemical missing in the brain of Parkinson’s patients.

The research team, led by senior author Jay S. Schneider, Ph.D., Director of the Parkinson’s Disease Research Unit and Professor in the Department of Pathology, Anatomy and Cell Biology and the Department of Neurology at Jefferson, found that administration of GM1 ganglioside, a substance naturally enriched in the brain that may be diminished in Parkinson’s disease brains, acted as a “neuroprotective” and a “neurorestorative” agent to improve symptoms and over an extended period of time slow the progression of symptoms.

What’s more, once the study participants went off the drug, their disease worsened. The study enrolled 77 subjects and followed them over a 120-week period and also followed 17 subjects who received current standard of care treatment for comparison.

“The drugs currently available for Parkinson’s disease are designed to treat symptoms and to improve function, but at this time there is no drug that has been shown unequivocally to slow disease progression,” said Dr. Schneider. “Our data suggest that GM1 ganglioside has the potential to have symptomatic and disease-modifying effects on Parkinson’s disease. If this is substantiated in a larger clinical study, GM1 could provide significant benefit for Parkinson's disease patients.”

Gm1
 

I talked to my neurologist at Jefferson about the GM1 this Monday. We discussed this prior to my appointment on Monday thru e-mails. He also talked to Dr. Schneider. What I was told was that they may have a next phase planned soon, and my neurologist said Dr. Schneider will get back to both of us...

This appears like another "breakthrough" drug, but why the delay?
 

I was recruited for this trial 12 years ago when first diagnosed. I declined for many reasons. This trial to my knowledge hasn't been financed by pharma but by
NIH and academic research sources so that that and the production of the substance itself has slowed progress on this treatment. All of which supports Peggy's thread on making more noise.

Katherine

GM1 Rx
 

This looks like a breakthrough treatment. Finally, something that favorably alters the course of Parkinson's.

Does anyone know whether GM1 Ganglioside is approved for treatment of any other conditions so that a doctor could prescribe off label for Parkinson's? Or could it be prescribed as an experimental drug by a doctor?

There is a clinical trials.gov record (with phase II data) at
http://clinicaltrials.gov/ct2/result...&Search=Search

Also a list of other clinical trials for other conditions. It was also being studied as a treatment for Huntington’s.

The Parkinson Pipeline Project treatment database was following the progress of GM1 Ganglioside for a number of years. See:
http://apexutf.shellprompt.net/pls/a...RN_PAGE:185,12

The latest news was that as of June, 2006, the treatment phase of the study was closed to recruitment. Then 6 years to analyze and publish the data? I agree the delays were likely due to a lack of a commercial sponsor. But this was one of the rare successful phase II trials. You’d think there would be some interest in developing it further?

A bia
 

There has been little hoopla or fanfare on these results which is odd. The lead researcher, Dr. Jay Schneider, is receiving plenty of local press. It looks like recruitment for Phase III is posted on his Thomas Jefferson U's

http://www.jefferson.edu/jmc/departm...unct-ther.html

Funny, they do not include the name of the substance under study? It is not listed at clinicaltrials.gov??

I was cautiously optimistic to read this quote from a local paper:

Over a 2-1/2 year period, not only did we see an improvement in their symptoms,” Schneider says, “but the progression of their symptoms was so slow that they weren’t even back to where they were at baseline.”

This falls in line with what Ceregene and Amgen were showing...that neurons are more impaired or clogged up as I think Girija has said. The researcher I spoke with from Ceregene basically said they were surprised by results as they implied neurons were functionally impaired or at least some were.

Laura

GM1 ganglioside could be the best thing since levodopa for PD patients! It really is so strange why there isn't more national news about the successful results of this phase II trial.

Is there even going to be a phase III trial? How can PD patients like ourselves make a difference into getting this treatment into further trials and expedited approvals? Is there an expedited FDA review process for such groundbreaking treatments like this? I read there's concern about the product being derived from cow and sheep brains and also some patent issues with the Italian manufacturer, but there must be a way to help this process along.

The researcher Jay Schneider is now studying sialidases as a way to boost endogenous levels of GM1. He has a grant funded from the MJF Foundation. But this research could take a decade. So why not get GM1 safety issues sorted out now to help PD patients now while in parallel working on sialidases and other related research?

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.