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.