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!

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