Nilotinib in Parkinson's

[shinything]

https://gumc.georgetown.edu/news/Ca...tor-Skills-in-Small-Parkinsons-Clinical-Trial

I read this a couple days ago, and can't help but think this would also be beneficial to treat ALS. It specifically mentions that it reduces plaque build up. When I google nilotinib along with tdp-43, other research/reports indicate that it removed soluable and unsoluable plaques.

Why is this not given to people with ALS?

I created this account specifically to bring it people's attention tion, because it seems like this should be available as an option and prescribed.

I have been viewing these forums since my father was diagnosed, and I have found so much helpful information. I haven't had the opportunity to share anything until I ran across this.

 

[lgelb]

Re: Thoughts on using Nilotinib to treat ALS

First, this was an uncontrolled trial w/ 12 patients (no control group) so as in ALS trials that have seemed successful and then failed, differences between patients and progression may have played a role.

Second, PD relates to the loss of dopamine-producing cells, not the central problem in ALS.

Third, it's a chemotherapy that has a number of potential side effects, some potentially fatal. It's not a drug to just hand out in any disease, including ALS. And, as you may recall, the list of drugs and devices that have actually worsened ALS manifestations in some PALS while at first seemingly helping is not insignificant.

 

[shinything]

Re: Thoughts on using Nilotinib to treat ALS

"Currently there is no cure, nor is there an effective treatment, to ease the suffering of ALS/FTLD patients. The only drug for the treatment of ALS is riluzole (rilutek), which is believed to extend survival by 3–6 months via possible glutamate mechanisms (12,13). The lack of ‘druggable’ protein targets to prevent aggregated protein toxicity is a primary reason that no effective therapies have been developed in FTLD/ALS. We demonstrated that some brain-penetrant tyrosine kinase inhibitors (TKIs) may be a potential alternative strategy to ameliorate the pathology associated with protein aggregation in neurodegenerative diseases (14,15). TKIs, nilotinib and bosutinib, are clinically effective and well-tolerated FDA-approved treatments for chronic myeloid leukemia (16,17). Importantly, nilotinib and bosutinib lead to parkin ubiquitination and increase its endogenous level, thereby activity (14,18), and TDP-43 regulates parkin expression (3). Inversely, TDP-43 depletion down-regulates Park2 mRNA in human stem-cell derived motor neurons in sporadic ALS, and TDP-43 expression affects parkin levels in postmortem brains of ALS and FTLD (19). "

from here: Parkin-mediated reduction of nuclear and soluble TDP-43 reverses behavioral decline in symptomatic mice

I believe this was one more ALS oriented, and also indicated nilotinib as an alterntaive strategy.

This paper is from May 2015.

 

[gooseberry]

Yes I read this info too. They used 150-200mg vs the 800 mg used for chemo. Parkinsons patients at Georgetown all showed a response, some significant in reduction of disease. However, within a few weeks of stopping the med, the decline began again.....at a very quick pace. The chemo monthly dose costs $10,800.

What I find exciting about this drug is the researchers are starting to find some pathways to treat degenerative disease. It is a start!

 

[lgelb]

It may be. ALS R&D is littered with promising mechanisms. As with others, it will likely depend on how early/to whom it is given.

There is an inducement to suggest from in vitro data that any given class could help a vast array of neurodegenerative dz; such hopes lead to grants, contracts and investors. No one disputes that repurposing chemo is worth a try; there is an immunomodulator that was used for RA that is now used in MS, for example. But there were several other immunomodulators tried for CNS d/o that did not pan out, or caused unacceptable toxicity. It's just not a great DIY project for all.

 

[findalscure2015]

Truly believe this will be a very effective treatment for ALS!
Please stay positive and don't give up!