On Dec. 16th the pre Investigative New Drug application for ovine GM1 was submitted to FDA. We should have their response in 30 days. We are eagerly waiting to hear from them as this will essentially mean a green light for ovine GM1 to move forward. This will lead to data collection for the IND application and submission so GM1 can move into clinical trials. The new year holds more grant deadlines for NIH – NINDS (National Institute of Neurologic Disease and Stroke). We continue to push ahead expecting new carrier lambs from cooperator flocks starting in Jan. The likelihood of a 2015-2016 clinical trial is looking more promising. In December we met with Dakota Lamb Growers Cooperative at their annual meeting and added 2 more cooperating flocks bringing flock numbers to 12. We should have many new carrier sheep in 2015 leading to many more affected lambs to treat HD in 2016.
GM1 Summit
On October 30-31st we hosted a GM1 summit in Brookings, SD to bring researchers together to facilitate cooperation to move GM1 into clinical trials. Drs. Shaw, Moore and Burgess from Avanti Polar Lipids, Alabaster, AL and Dr. Hersch from Massachusetts General Hospital along with local facilitators Van and Paul Fishback hammered out details to move GM1 to a preIND (investigative new drug) meeting with FDA. Funding opportunities through (NIH/NINDS – National Institute for Neurologic Disease and Stroke) are also being pursued. It was valuable time to get the group together for 2 days of face to face contact. A special thank you goes to Tara, Mike, Katelyn and Nevaeh Hansen for meeting with the group. Without first hand experience with HD it is hard to understand the urgency of this project. Kate and her family are truly inspirational in their efforts to educate others about HD and put a face to a devastating rare disease. Dr. Hersch estimated that there are 50,000 HD patients in the US that could benefit from GM1. When you live near Brookings, SD a town on 22,000, HD hardly seems rare since more than twice as many people are afflicted with HD as live in the 4th largest town in SD. Thanks to Dr. Hancock, Veterinary Epidemiologist, we now have graphic projections of how we can expand sheep numbers within the next decade to produce enough lambs to treat these patients. We currently have 10 cooperating GM1 flocks with access to over 3,500 ewes. Even without adding more cooperators it is possible to raise 30,000 affected lambs in 10 years. We are making progress on this GM1 for HD journey!
SD Sheep Growers Convention a Success
The weekend of the SDSGA convention was busy and eventful. Katelyn Hansen and her family were able to attend the meeting and Mike Hansen spoke to those in attendance about what it is like to have your 18 year old daughter be diagnosed with Juvenile HD. Katelyn is now 23 and the chorea of the HD symptoms are pronounced. Katelyn is a beautiful, courageous young woman, who with her family, is trying to find a treatment for HD not only for her but for all families who struggle with this devastating disease. As people were able to visit with Kate, her Dad Mike, her brother Mikey and the Button Boys, Landon, Shawn and Gabe (her younger step brothers) they were educated about HD and one families fight to find a treatment. The Banquet following the meeting was truly overwhelming as the generosity and compassion of the sheep producers resulted in a rollover auction of a wool blanket for Kate. After several rounds of the blanket auction somewhere between $4-$5,000 was raised for the “Katelyn project”. This generosity also fostered the name for the non-profit fundraising and promotion organization in the process of being formed “The Shepherd’s Gift: GM1 for HD” (see Facebook). Thank you so much to everyone who attended and was moved to help with this project.
Sept 27-28th SDSGA presentation
Our next opportunity to present this project is Saturday Sept. 27th at the South Dakota Sheep Growers Annual Convention. Larry is scheduled to speak from 11:15-noon at the Days Inn Convention Center in Brookings. Mike Hansen and the Button Boys are planning on helping out. The families who struggle with HD are the ones who can truly motivate sheep producers to participate. These families instill urgency to this project when they share their stories. The Button Boys and GRI will also host a booth on Saturday. On Sunday (28th) at 8:30 AM we have the opportunity to host another tour of the farm. Sunday afternoon is the Sioux Valley Chapter HDSA meeting at the Sanford auditorium in Sioux Falls. It is a place to meet more families and learn more about HD.
Larry has been busy spreading information about GM1. On Sept. 3rd he spoke to Veterinarians at the Pipestone, MN Veterinary clinic and on Sept 4th he spoke to a Parkinson’s support group in Brookings. As Dr. Hersch put it “the path forward to the clinic is right in front of us, its just gonna be one step at a time.”
Update on NIH/NCATS Proposal
We were hoping clinical trials were in the near future with the help of the National Institute of Health (NIH) -National Center for Advancement of Translational Science (NCATS) – Treatment of Rare and Neglected Diseases (TRND) http://www.ncats.nih.gov/research/rare-diseases/trnd/trnd.html.
After our proposal was accepted in January, it underwent external review, 2 levels of internal due diligence, a face to face meeting including data presentation, and another round of follow-up questions. The review board then decided to turn down our proposal. While they commented that they were impressed with the science the feedback we received focused on their concern that we could not raise enough sheep. Everyone we have talked to in SD has been baffled by this comment.
We have raised hundreds of these lambs over the last 20 years.
lambs waiting to become part of the GM1 project
The expansion of these genetics requires basic biology. The scale up is possible if there is a revenue stream for these GM1 producing lambs. Another concern was the potential production of a synthetic. While this synthesis has been attempted in the lab over the last 20 years only very small quantities have resulted and at a very high cost. Additionally, it is still a semi-synthetic requiring an expensive starting molecule. Both of these concerns can be answered and overcome.
What matters the most is that HD is a wretched disease that has no treatment. The data on GM1 is very compelling and the clinical trial needs to go forward for the sake of HD patients. We have enough GM1 in our freezers for a clinical trial. We are asking for grassroots support to help us continue on the path to a clinical trial.