Special Sheep May Hold Promise for Huntington’s Disease Treatment
Ag Product for Pharmaceutical Use
Article by Tricia Wempe, MA, HD Translational Research Advocate
What would you do as an HD community member if you met a scientist(s) who thinks he/she has a prospective therapeutic drug for HD – and/or possibly a cure? I hope your answer would be similar to mine – start supporting them in any wa
y a person can! A four member cohort of scientists: Dr. Holler (South Dakota State University), Dr. Hersch (Harvard/Massachusetts General), Dr. Frey (University of Minnesota), and Dr. Shaw (Avanti Polar Lipids, Inc. – AL) are leading the way to get GM1 for HD into human clinical trials. For the past 2 decades the Holler’s have maintained a flock of sheep that contain a specific genetic trait that results in the accumulation of a natural molecule, GM1 ganglioside. Dr. Holler originally got excited about the idea of pursuing ovine (sheep) GM1 application to HD because data was published in Spring 2012 that showed GM1 had reversed HD symptoms in a mouse model within 60 days(http://en.hdbuzz.net/072). And in addition, he knew of published data that showed GM1 had already been in human clinical trials for application to Parkinson’s Disease (PD), Alzheimer’s Disease (AD) and other neurological diseases (http://www.glycoscienceresearch.com/references-2/ ). Safety in humans had already been proven. For the laypeople in the HD community, this is all very exciting! Why is there not a human clinical trial yet? Short answer: Politics and money. Long answer with some history: Last January in conjunction with Dr. Hersch a project proposal was submitted to The National Institutes of Health (NIH) National Center for Advancement of Translational Science (NCATS) Therapeutics for Rare and Neglected Diseases (TRND) to help get GM1 from basic science research into clinical trials for HD. While the proposal made it through multiple rounds of scientific scrutiny, it was rejected in late April 2014. After reviewing current trends in the US sheep industry, the NCATS reviewers concluded that although they were impressed by the science, they determined it was impossible to raise enough sheep to treat HD patients, and that it may be necessary to wait until the scientific community figures out how to synthesize the molecule. The scientific bureaucrats in the federal government simply rejected the proposal because they didn’t think enough sheep could be produced. According to Agricultural Lamb Producers that do this for a living, this deduction is completely asinine reasoning. Currently there are about 30,000 HD patients in the US. Using current estimates, it would take 1 to 2 sheep to treat an HD patient for 1 year. To date, Dr. Holler currently has capacity to treat approximately 300 HD patients. However, money is needed so that Dr. Holler can ramp up the number of sheep to 30,000 sheep. A good faith initial estimate is that he needs about 1- 2 million dollars over the next 2 - 3 years to help get GM1 into human clinical trials and into the pharmaceutical market. These scientists need our support through the HD grassroots level to help GM1 get its turn to prove itself in a human clinical trial. And for approximately 2 years, that is what I have been doing on a volunteer basis. And we need more grassroots support. If you are motivated and want to help, please contact Dr. Larry Holler, Sue Holler, or myself directly if you have any questions or simply want to learn more. Families who struggle with HD, we urgently need a treatment! And please do what you can to help get GM1 for HD into human clinical trials. GM1 needs its turn to prove itself! Tricia Wempe, MA [email protected]
Larry Holler, DVM, PhD [email protected]
Susan Holler, MS [email protected]
Glycoscience Research Inc. www.glycoscienceresearch.com
