As the 2016 HDSA meeting in Baltimore approaches, I continue to wonder how they can ignore one of the most promising therapeutic treatments for HD. In 2013, I wrote: there is a very urgent need for a treatment. GM1 has been to shown to have remarkable effects in reversing HD symptoms. We have a source of GM1. So the resulting question is why isn’t there a clinical trial in progress????
Here we are 3 years later and I’m still asking the same question. The science is all there for the bovine source (decades of it). The only thing missing is the funding to finish the testing required by FDA to show that the sheep source is equivalent to the previously used, but now unacceptable cow source. Since it is a natural molecule it is the same, we just need to collect the data to prove it. The science is there – don’t take my word for it check out the scientific literature. A search for GM1 ganglioside on PubMed will reveal a multitude of articles. Just a sampling of these articles is listed on the reference page. It continues to frustrate us that we have a source for a treatment that could halt and possibly reverse HD symptoms. For the friends who we have come to know and care deeply for time is of the essence. What is the holdup? Are the major HD organizations too wrapped up in politics? Has too much been invested in gene silencing to not look at other promising treatments? Why isn’t any possible treatment being pursued? Especially one that has been used in 100,000’s of patients. One that has been shown to be safe and effective in multi-center trials. One that could impact other neurologic diseases.