Whenever I was looking for something I had lost as a kid, my mom would offer the same, unhelpful advice: It’ll show up in the last place you look for it.
As irritating as the cryptic saying can be, it rings true as findings provide hope for those searching for a Huntington’s treatment. The last place anyone would look for a Huntington’s treatment is on a sheep farm in South Dakota, but that might just be exactly where they’ll find it.
There’s nothing fun about Huntington’s disease, a rare, neurological condition which appears in adults. Huntington’s is caused by an inherited genetic mutation, which interferes with production of the huntingtin protein. The disease attacks the patient’s brain. They lose more and more of their physical and cognitive functions– often causing dramatic personality changes. The disease progresses, and is ultimately a fatal condition. To learn more about this rare disease, click here.
First, it’s important to know about a crucial molecule called GM1 ganglioside, which is essential for our nerve health. It may able to treat Huntington’s, although rigorous clinical trials would need to confirm this. People and animals generally have it naturally, both in nervous and other tissues, but we also have an enzyme responsible for getting rid of the excess GM1. Huntington’s patients seem to lack this molecule. When researchers tested their hypothesis out by administering mice with Huntington’s, they saw that the mice responded surprisingly well, and began recovering.
Veterinarian and sheep farmer, Dr. Holler, is an expert in sheep genetics– he’s been focused on it for 20 years. His sheep have a mutation, that halts production of the enzyme that breaks GM1 down. As a result, they have massive amounts of GM1 in their brain– about forty times more than you’d expect in a comparable animal brain.
If clinical trials show that GM1 is an effective treatment for Huntington’s, then there would need to be a mass production of GM1. Fortunately, Dr. Holler’s sheep would have an excess of the exact molecule Huntington’s patients lack.
It’s hard to say how the pre-clinical, and then clinical trials will play out in regards to this particular treatment. However, it is exciting to see all the innovative ways researchers are approaching this degenerative disease.
