FDA PIND (pre investigational new drug) application

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.

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7 Responses to FDA PIND (pre investigational new drug) application

  1. Tricia Wempe says:

    This is an awesome step forward!
    Totally God!
    Talk soon, —TW

  2. dan evans says:

    this is great news let god lead the way for this soon

  3. Do you have any interest in expanding your cooperative flock network into the state of Virginia? Dear family friends raise sheep, I have other precious ones with HD, and I am considering jumping in here where a difference can be made.

    • admin says:

      Hi Margaret,
      We have others in Virginia that are also interested. We would need to figure out the logistics to get you the genetics but we are thankful you are interested. We keep moving forward, but it sometime feels like we are always fighting uphill. But the pre-clinical data for GM1 looks very good, its been proven safe and right now, these lambs are the best source available. So we keep pushing. Come visit in South Dakota some time. Larry Holler DVM, PhD

  4. adam white says:

    Hi Larry and Sue. Just wondered as a new PD sufferer how the process is going to date on GM1? There’s a lot of talk on the web and folk just want to get better. Have you any hope for the future? Yours from the UK.

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