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.
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.