5 months ago a poster from Korea came on here and mentioned this drug. At the time I did not realize the drug had been tested for patients with Parkinson's so it crossed my radar and I moved on, but today as part of my review of drugs currently in the pipeline I came back and what I found is very impressive.
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First, this is a drug that has been around for a while as a treatment for cancer so it has a long history of safety in humans. Second, the animal research related to Parkinson's showed that this drug may act by enhancing autophagy. For those unfamiliar with my posts you need to understand that autophagy is critical to my belief in what treatments actually have near term potential to help patients with genetically inherited SCAs. Those types of SCAs are all the result of toxic proteins that build up in the brain and cause cellular death in the cerebellum. Autophagy is a fancy word meaning the drug cleans out toxic proteins, and that means Nilotinib is an excellent candidate to treat some SCAs. Most importantly though there are human clinical trials both completed and underway. The trials are in Parkinson's, but at least it's a start. Lastly, the poster from 5 months ago linked to a paper that I ignored at the time, but I now see that was a mistake. The paper reported on study done in South Korea. Doctors there gave 12 patients with various forms of ataxia low dose Nilotinib (150 mg - 325 mg per day). 3 patients had genetically inherited forms of SCA, and those patients had the best results, but 8 patients showed some improvement. Sadly that study does not meet the standard of double blind placebo control studies, but to me when combined with the history from cancer patients on safety, plus the animal studies showing enhanced autophagy, plus the results to date in Parkinson's related clinical trials this drug is now on the top of my list of drugs to watch. I also intend to contact the manufacturer and anyone else I can find to encourage research into the use of Nilotinib for SCA.
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For those that would like to read more about this drug here are several related links. I suggest starting with the article written by Simon:
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scienceofparkinsons.com/201...
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And here is a link to the paper on the Korean study. I purchased the paper so if anyone wants a copy please simply send me a PM.
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c-Abl inhibitor, nilotinib, as a potential therapeutic agent for chronic cerebellar ataxia
Partial Possible Treatment for some SCAs.
