Nilotinib/Tasigna revisited

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.



vow JP66, are you a specialist doctor of medicine, or just a buff (somebody very keen to follow it all). No idea on meds, as my maternal grandpa passed on 8 years ago, and do not have AN ACTIVE INGREDIENT of his meds…Let’s just say all his meds were very strong, sorted his shaking, but side effects with digestion and constipation like hell…I am personally at the beginning of sca2, so NOTSHAKING VISIBLY YET. Could you write to me the active ingredient, pls.?I am in Britain and the pharmaceutical industry differs from Europe (my origin), so I never go by the names of any meds, as they seem to change depending on manufacturer…too true my gait is wide, dizziness, articulation, non existant….hand coordination I am still threadin a needle as practice…But IMPATIENT, FRUSTRATION WITH MYSELF…on a bad day, on a good a mere inconvenience…

Ladybird, be very careful about taking anything not prescribed by your GP or Neurologist. It’s possible there could be an interaction, not all medications or complimentary therapies will be suitable for every type of ataxia. The last thing you want is to have a reaction and bring something else into play :slightly_smiling_face: xB

Thanks Beryl for your concern.I am not on any meds or NOT PLANNING to take any apart from my antihistamine for general maintenance of lower level of histamine…My neurologist would know, if anything helping. Not even my gp dares to go any further, then the usual bog standard antibiotics, paracetamol, cough medicine…I know how far I can go, preferably not far from my bed:) Although annoying all this ataxia stuff, but accepted and there is nothing to solve anymore…It is what it is, I am making the best of what’s left…

Hi Ladybird,
First thank you for reading my posts. I very much enjoy knowing that people pay attention. Next please forgive my lack of understanding, but I am unclear about what you asked. If you are asking about my Alternative Therapy then I must emphasize 2 important points. First what I am doing is completely unproven and so it is very important that you talk to a doctor before trying anything on my list. Second, if that IS what you are interested in then I am very happy to provide information that you can take to your doctor so he can help you decide if anything on my list might be helpful to you. You can send me an email request at p e c k j a at a o l dot c o m if I got that right. Otherwise please forgive my misunderstanding :grinning:Joe

I do not necessecarily believe that our neurologists or other doctors know all that we want them to. We are our own best advocates, and it makes sense to discus whatever we find out with our doctors.