I wrote a post a while back in which I mentioned this drug, but never followed up with the later research I uncovered. This drug has been used to treat cancer for years, and recently was studied as a treatment for Parkinson’s disease. The human clinical trial in Parkinson’s was positive, but corporate politics got involved and the second Phase II trial was delayed, but is finally beginning. Meanwhile a group of doctors in South Korea gave low dose (150 mg - 325 mg per day) Nilotinib to 12 patients with different forms of ataxia. The 3 patients that responded the best all had genetically inherited forms of ataxia whose pathology involves mutant proteins. Part of the animal research that led to the study of Nilotinib in patients with Parkinson’s showed that the drug was effective at enhancing autophagy which means it helps clear out bad proteins from the brain. I intend to write an email to the makers of Nilotinib and encourage them to examine it’s potential for people with genetically inherited forms of SCA. Aside from the fact no research is being done here for ataxia treatment with Nilotinib, this drug costs $4000 to $5000 a month.
Also, I purchased the paper written by the Korean doctors. If anyone is interested in a copy please feel free to send me a PM.
The main pathophysiology of the major causes of chronic CA syndrome, SCA 1, 2, 3, 6, 7, and 17, and DRPLA is abnormal intracellular protein aggregation that is mainly intranuclear and is caused by CAG trinucleotide repeat expansions (Koeppen, 2005, Schöls et al., 2004). MSA-C also results from cytoplasmic accumulation of α-synuclein in neurons and glial cells (Gilman et al., 2008). Therefore, nilotinib may exhibit a modifying effect on CA from those etiologies by facilitating autophagy-mediated clearance of abnormal proteins.
In addition, activated c-Abl tyrosine kinase is a key element of cell inflammation and cell death processes and is provoked by various neuronal stresses, including intracellular Ca2+ accumulation, glutamate toxicity, overproduction of reactive oxygen species, mitochondrial dysfunction, and DNA damage, which are common pathomechanisms underlying genetic, degenerative, autoimmune, and idiopathic chronic CA.