We are excited to move into the finals for the Giant Vision Business Competition. On April 26th, 2017 we have the opportunity to present GlycoScience Research (GSR) and GM1 for HD at the Governor’s Office of Economic Development Annual Conference. The day will include an exhibit, presentation, and elevator pitch. The Giant Vision competitor display booths will be open to the public from 2-4:30 PM on April 26th at the Sioux Falls Convention Center. https://www.facebook.com/southdakotagiantvision/
As stated in our business plan: This production scheme envisioned by GSR represents the first time in history that a spontaneous genetic condition in a production agricultural species has been utilized by a biotechnology company to treat a deadly and incurable genetic disease in humans. This is the GSR Giant Vision!
The following is a letter initiated by Kay Persons, one of the GM1 sheep producers. Feel free to use it to help move the project forward to help HD families and other families afflicted with neurologic diseases.
I need your help. I have several friends and relatives who have suffered from Alzheimer’s and Parkinson’s and Huntington’s Disease. Scientists in South Dakota, Larry Holler, DVM, PhD and Sue Holler, MS, have worked for years trying to get a sheep source of GM1 ganglioside to clinical trials. GM1 ganglioside is a naturally occurring molecule that has been shown to have neuroprotective and neuroregenerative properties. A cow source of GM1 has been in clinical trials for Parkinson’s disease and spinal cord injury. More recently, research evaluating GM1 for Huntington’s disease has shown reversal of symptoms in Huntington’s disease mice. New research with this sheep product is showing promising results for Alzheimer’s. The previously used cow source is not allowable by FDA due to Mad Cow Disease and the lack of traceability for this unverified source of GM1 for human medicine. The sheep source of GM1 is not only verifiable but naturally produced in much greater quantities than the cow source. The Holler’s are collaborating with Dr. Steven Hersch, MD, PhD at Massachusetts General Hospital/Harvard Medical School to secure funding through NIH NINDS IGNITE for the preclinical data that FDA requires.
Here is where your help is needed. Those of us who are so anxious for relief for patients of these neurological diseases need to have you urge the NIH to support this study. The status of this grant proposal, Ovine GM1 Ganglioside Development for Huntington’s Disease, that was submitted in February, should be determined by this summer. Time is of the essence to move forward with the preclinical data so the clinical trials can be started.
On behalf of the countless number of people suffering from these diseases, thank you for your time and effort.
ASI Lets Grow Grants are nearing completion which will include educational, promotional and training videos for sheep producers. GM1 sheep production for Huntington’s Disease: Training Videos, Record Management, and Cooperator Communications.
NSIIC 2015 grant Development of Ovine GM1 ganglioside for Huntington’s Disease – in progress HD mouse studies using ovine GM1 at Massachusetts General Hospital in Dr. Steven Hersch’s lab.
NSIIC 2016 Comparison of meat composition of lambs affected with GM1 gangliosidosis to retail lamb – newly funded.
We are continuing to apply for the larger NIH grants currently though NINDS IGNITE = NIH National Institute of Neurological Disorders and Stroke (NINDS) Innovation Grants to Nurture Initial Translational Efforts (IGNITE) which is a suite of early-stage therapy development funding programs meant to serve as a feeder program to the later-stage therapy development programs such as the Cooperative Research to Enable and Advance Translational Enterprises (CREATE) for Biologics Although there is a lot of data on the bovine GM1 they are requiring that it be repeated with the ovine product.
The Shepherd’s Gift: GM1 for HD 501c3 continues to fundraise. The Shepherd’s Shuffle, 5K and 1K, in conjunction with South Dakota Sheep Growers Annual Convention will be 9/30/17 in Brookings, SD. TSG has raised over $100,000 thus far and has supported Dr. Hersch’s research as well as pay for cooperator GM1 affected lambs. Donations are always welcome – see crowdrise.com/theshepherdsgift
Additional research funding opportunities through private HD foundations are being pursued (Bev Hartig HD Foundation) as well as potential investors.
The Governor’s Office of Economic Development (GOED) of South Dakota has been awarded a $125,000 FAST (Federal And State Technology) grant from the United States Small Business Administration (SBA) to support technology based entrepreneurs and commercialization of innovations.
“The SBA FAST project is focused on developing management teams involving business and technical expertise to not only develop innovative new products but also bring them to market,” said GOED Commissioner Scott Stern.
“Development and growth of these innovative new businesses will help to fuel continued growth of South Dakota’s economy.”
With the support of the SBA FAST grant and the GOED Proof of Concept program, “I-Corp” teams, consisting of an inventor and experienced business mentor supported by graduate and undergraduate students, will develop and execute a business plan to bring the innovative new technology to market.
The I-Corp teams selected to participate in the program will:
participate in a three-day business start-up boot camp in Rapid City, S.D., Nov. 4-6, 2016;
participate in the Small Business Innovation Research (SBIR) proposal preparation workshop, also in Rapid City, S.D., Nov. 7-8, 2016
receive support of a team of graduate and undergraduate students during the next year to implement their development plan;
provide funding for eligible feasibility studies and to also support development of SBIR proposals to federal agencies from the GOED Proof of Concept program;
will be encouraged to participate in the Governor’s Giant Vision Business Plan competition.
It has been a busy month. Sept 1st started with a booth at the South Dakota State Fair and an opportunity to talk with Governor Dennis Daugaard about the project ant the impact it could have for South Dakotans, not only those with neurologic disease but also those in rural areas. Sept. 16 brought the new SD Secretary of Agriculture, Mike Jaspers, to the farm for a tour and meeting. Interest continues to grow as we continue to make contacts and bring awareness to the project about the humanitarian, as well as the economic impact, it could have on the state. Sheep producers continue to inquire about joining the project. Sheep producers from Ohio, with careers in the medical field, as well as an Oregon sheep producer whose sons have HD have contacted us. We have shifted gears for grant opportunities to Small Business Innovation Research and Technology Transfer. We are working with the Graduate Education and Applied Research Center at the South Dakota Public University and Research Center (University Center) to develop necessary protocols for Current Good Manufacturing Practice (cGMP) of GM1, to comply with the regulations enforced by the US Food and Drug Administration (FDA). They will also be able to help with genetic testing. Additionally, we have been busy with sheep, marketing, and processing lambs, adding fall lambs to the flock, and putting up corn silage for winter feed.
We continue to push forward for the families we have gotten to know, as they deal with the cruelest disease known to man. GM1 deserves its chance to be tested!
As the 2016 HDSA meeting in Baltimore approaches, I continue to wonder how they can ignore one of the most promising therapeutic treatments for HD. In 2013, I wrote: there is a very urgent need for a treatment. GM1 has been to shown to have remarkable effects in reversing HD symptoms. We have a source of GM1. So the resulting question is why isn’t there a clinical trial in progress????
Here we are 3 years later and I’m still asking the same question. The science is all there for the bovine source (decades of it). The only thing missing is the funding to finish the testing required by FDA to show that the sheep source is equivalent to the previously used, but now unacceptable cow source. Since it is a natural molecule it is the same, we just need to collect the data to prove it. The science is there – don’t take my word for it check out the scientific literature. A search for GM1 ganglioside on PubMed will reveal a multitude of articles. Just a sampling of these articles is listed on the reference page. It continues to frustrate us that we have a source for a treatment that could halt and possibly reverse HD symptoms. For the friends who we have come to know and care deeply for time is of the essence. What is the holdup? Are the major HD organizations too wrapped up in politics? Has too much been invested in gene silencing to not look at other promising treatments? Why isn’t any possible treatment being pursued? Especially one that has been used in 100,000’s of patients. One that has been shown to be safe and effective in multi-center trials. One that could impact other neurologic diseases.
The MSPA was a great opportunity to spread the word about the project. Two families who are living with HD were present and once again made us realize how important the success of this project is. More than 200 people packed the room as Larry and I presented. We truly appreciated this opportunity to spread the word about this grassroots venture. Michigan Farm Bureau (MFB) was there to film our presentations and also interviewed one of the families. Thank you Mike and Heather Ludlam for you enthusiastic participation and willingness to share your personal story. .
An article in HDBuzz http://en.hdbuzz.net/199 reported on GM1 for HD. Unfortunately they did not talk with GRI or The Shepherd’s Gift before they published. As a result of their rush to get their version online there is misinformation in the article. I have explained the discrepancies on the HDBuzz correction page. The take home message is that GM1 has decades of research data and has been proven safe in hundreds of thousands of patients. The missing link is a verifiable source. Once the bovine source was banned due to BSE, research was greatly hindered. We are pursing a verified source of GM1 to provide a treatment for HD and eventually other neurologic diseases. We are disheartened that “researchers” don’t fully review the scientific literature and report it accurately. The first component of any graduate degree is a thorough evaluation of the scientific literature. We have a reference page to validate our information on GM1. Don’t take our word for the potential therapeutic benefits GM1 has for HD. Please review the literature for yourself.
On January 23 we received feedback on our pre-IND proposal submission. The FDA said the sheep source for GM1 is acceptable! We will now move ahead with the next step toward clinical trials which includes ADMET (absorption, distribution, metabolism, excretion, and toxicology) studies, finishing pre-clinical animal studies, finalizing GM1 the recipe, and planning a clinical trial. All this work will eventually be put into the form of an IND (Investigative New Drug) application. To learn more about the process see:
The NIH/NINDS (National Institute of Health/National Institute of Neurologic Disease and Stroke)/CREATE (Cooperative Research to Enable and Advance Translational Enterprises for Biotechnology Products and Biologics (CREATE Bio)) grant was submitted by Feb. 11th. http://www.ninds.nih.gov/funding/areas/translational_research/CREATE-bio.htm.
The FDA pre-IND approval of the sheep source for GM1 ganglioside production was necessary for us to have a chance with this grant submission. Hopefully, the Create funding will keep us moving forward. Unfortunately, without some type of outside investment, the pace will be much slower. In Isaiah 40,verse 31, we are told, “But those who trust in the Lord will find new strength. They will soar high on wings like eagles. They will run and not grow weary. They will walk and not faint”. The path forward to the clinic is laid out right in front of us. It’s just gonna be one step at a time. All we have to do is trust.