HDBuzz

A recent article in HDBuzz had misinformation about GM1 and our project. Scientists are trained to review data and draw conclusions based on the statistical analysis of data. We put forth data that is well supported in the scientific literature.  On the Glycoscienceresearch.com reference page we have listed some conclusions about GM1 and the scientific journals that published the supporting data.

 GM1 has been shown to be SAFE in human clinical trials – see results of 5 year PD study by Schneider, Spinal Cord Injury study by Geisler and Stroke study.   These studies validate the safety of GM1 after its use for up to 5 years in thousands of patients using doses of up to 1 gram of GM1.  The only reported side-affect was a slight increase in lipemia in serum (similar to eating a big Mac).  Clinical trials were in progress and showed promising results. Schneider and colleagues demonstrated that GM1 ganglioside treatment enhances neurologic function significantly in PD patients. Originally GM1 (bovine source) was approved for use in Europe for treatment of peripheral neuropathy and was used in hundreds of thousands of patients.  When BSE (mad cow disease) was diagnosed in Europe this source was banned.  With the ban on unverified source material (slaughter plant cow brains) there was no longer a source of GM1 THUS research progress stalled.  For many years synthesizing GM1 was tried but to no avail. Only very small quantities of semi-synthetic GM1 could be made in the lab and then at a very high cost due in part to the expensive non-synthetic starting material.

The mechanism of action (why it worked) in HD is known, Ganglioside GM1 induces phosphorylation of mutant huntingtin and restores normal motor behavior in Huntington disease mice, as Dr. Sipione states in the title of her Proceedings of the National Academy of Science paper.  In Neurology Today, Tom Valero interviewed Dr. Sipione.  The resulting article is Injecting Ganglioside GM1: An Experimental Approach for Huntington Disease That Soon Might Be Ready for Human Trials. Unfortunately, she only had a small amount of bovine GM1 and couldn’t continue research without a source of GM1.

GM1 gangliosidosis occurs in humans and other species including sheep.  GM1 itself is NOT toxic in this disease and does NOT cause the destruction of nerve cells. The pathogenesis of GM1 gangliosidosis is much more complicated than the mere accumulation of GM1.  GM1 administration, even in gram quantities, has been shown to be safe.

The delay in research for GM1 for HD is due to funding.  Although FDA had positive pre-Investigative New Drug feedback, the next step for the IND application will take close to $1.50M.  Then funding is needed to move into clinical trials (estimated $4M).  Although the bovine source has been through this process FDA will require the ovine source to go through the same testing.

The Shepherd’s Gift is the non-profit formed by HD families (not the Hollers – we are not on the board) since the organizations that are promoted to help HD families did not respond to requests to help get GM1 into clinical trials.  Additionally, pharmaceutical companies supporting current HD medications were not interested either.  One reply stated that GM1 was not in their strategic plan.  The Shepherd’s Gift: GM1 for HD mission is to promote ovine (sheep) GM1 for the treatment of Huntington’s Disease (HD) and/or other neurological diseases through education, advocacy, and fundraising.

GM1 has volumes of data behind it and a research track record of over 20 years.  The missing component, since the bovine source became unacceptable for pharmaceutical use, has been a verifiable source. A requirement that this specifically bred line of sheep can fill.

And YES – WE WOULD BELIEVE IT!! BASED on a review of the scientific literature. The use of GM1 for Huntington’s disease is one of the most promising therapeutic approaches.