mlive.com/news/grand-rapids...
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clinicaltrials.gov/ct2/show...
Exenatide is a medication originally used to treat diabetes. Most of the forms of ataxia involve a buildup of "misfolded" proteins that cause cellular death. Parkinson's Disease and Alzheimer's Disease also involve "misfolded" proteins so they have this in common with ataxia. Where the proteins build up and which cells they impact cause different symptoms in these diseases, but again they all have one element in common: protein aggregates that aren't "cleaned out". The process of cleaning out proteins is known as "autophagy".
This is what exenatide researchers know about this medication:
"Cotreatment with the GLP-1 receptor agonist exendin-4 reversed the lysosomal dysfunction, relieving the impairment in autophagic flux and further stimulated autophagy"
"The study showed that intranasally administered Exendin-4 passed through the blood-brain barrier."
"These findings provide pivotal experimental support for further study of the neuroprotective effects and clinical application of Exendin-4.
In other words, this drug improves cellular function and helps it clean out bad proteins which is exactly what ataxic patients need. Whats best of all this drug has already had human clinical trials that showed promising results in treating Parkinson's and Alzheimer's. My recommendation is we all email the maker of this drug and encourage them to consider a clinical trial in people with ataxia.
One of the major trials in Parkinson's Disease is right there in the UK!
Cheers,
Joe
ncbi.nlm.nih.gov/pubmed/289...