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carruthers209

Aducanumab is the nilotinib for Alzheimer's-they're holding their breath also

7 posts in this topic

http://edition.cnn.com/2016/08/31/health/experimental-alzheimers-drug/index.html

 

The Alzheimer's community is also holding its collective breath about aducanumab from Biogen which is demonstrating plaque clearing in Alzheimer's brains.  The plaque in Alzheimer's brains is a different protein-beta amyloid-which is also destroying the brain neurons as they collectively spread throughout the brain.  Alzheimer's starts in the hippocampus which is the short term memory and spatial part of the brain in which immediate and daily memory is stored until it moves into long term memory after a good night's sleep.  The damage in the hippocampus is why Alzheimer's patients continually repeat their statements, questions, etc. over and over.  They can't remember that they already asked the question, etc.  Anyway, aducanumab may be the answers to those prayers for Alzheimer's patients-this research is conducted on patients instead of mice so the hope is enticingly close.  Alzheimer's affects about 5 to 6 million Americans and this is the most hopeful news so far.  The Parkinson's and Alzheimer's communities might have good news over the next few years-we can only hold our breath and hope!!

Edited by carruthers209
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Please correct me if I'm wrong.

 

As I understand it, Aducanumab is a new "antibiotic" which rids the brain of plaque but it does not cure or reverse any of the symptoms of Alzheimer's. They hope/believe it will slow down Alzheimer's progression.

 

I would deduce from this that the plaque itself does not cause the symptoms of the disease but is merely a result of same and therefore could be used as a marker to confirm same.

 

Fred

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Parkinson's and Alzheimer's as well as the other neuronal diseases share (in a negative sense) some of the same frustrations-there is no cure, the actual causes can only be conjectured, and the sheer complicated biochemistry and biology of our neurons eludes definitive answers.  With Parkinson's the statement is that it's a "prion" like disease in which toxic proteins cause a cascade of damage.  The proteins misfold in both Parkinson's and Alzheimer's.  The research articles for both nilotinib and aducanumab report very similar benefits from the plaque clearing that these drugs provide and then patient improvement is reported and the rate of progression of the diseases is slowed down.  Neither is being reported as a cure.  Alzheimer's is the great fear for most people-one out of every three people after the age of 85 is projected to get Alzheimer's and the financial toll is devasting and projected to bankrupt Medicare.  My guess is that huge amounts of research money will be spent on speeding up the research and getting products to market.  If the progression of the disease can be slowed and improvements in cognitive function demonstrated, it can be the "game changer" promised in the headlines.

 

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I saw this on the news and it will be years and years before anything will come of it.

"Years and years" seems overly pessimistic. Unlike nilotinib, which has shown limited promise is a single, uncontrolled, very small study, the results for aducanumab are from larger, double-blind Phase II trials. Results were compelling enough to be published in Nature. Given the prestige of that journal, and that Alzheimer's is the most common neurological disease in the world, there's going to be no lack of funding and volunteers to start Phase III trials as soon as possible. Give Phase III three years to produce results, and if they look good, there will be considerable pressure for fast-track FDA approval. So assuming the drug passes Phase III (big assumption), it might hit the shelves in 5-6 years.

 

Sure, that's going to be too late for plenty of people. But that's always the case with any drug. The alternative is to allow drug companies to rush useless and/or lethal compounds to market, with little oversight--in the long run, that's going to kill a lot more people than might be saved by a less rigorous drug approval policy. After all, something like 90% of drugs that show promise is a petri dish or animal model fail to work in humans.

 

The good news from my point of view, other than a potential treatment for a disease I consider far worse than PD, is that immunotherapy drugs for PD (similar to aducanumab) are already in Phase III trials--that much closer to market if all goes well.

 

Nilotinib, as far as I know, is *not* a narrowly-targeted immunotherapy drug, and already carries a black box warning for use in cancer patients. I suspect that, even if it does pass Phase II (safety) and Phase III (efficacy) trials for PD, nilotinib will be a treatment of last resort for patients with very advanced disease. It's also not unlikely that the 'miracle' results seen in one tiny study (and over-hyped by the researcher, in my opinion) will disappear with high-quality double-blind studies, leaving nothing but another dead end.

 

It's also worth remembering that without solid, basic research in biochemistry, protein structure, and computer science (to build supercomputers capable of simulating complex protein folding), none of this would be possible. Research showing PD, Alzheimer's, and other neurological disorders results from misfolded proteins (are 'prion-like') took place a decade or so ago--we're seeing the pay-off now in the form of a flood of new drugs moving into human trials.

Edited by ShopGuy
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You are new to PD and relatively young so this drug and Nilotinib will probably work for you when and if  they are both finally approved. You have time on your side so of course you can be jumping for joy waiting for it to happen.  I'm speaking for people like my mother who do not have 5 years to wait for something that shows promise.  I am happy when drugs move along the pipeline but it will never be fast enough in my eyes.

Edited by miracleseeker

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Shopguy-thanks for the background on nilotinib.  Aducanumab (does this name mean something?  I can't even pronounce it) has now been fast tracked by the FDA because of the huge potential good that might result from a truly beneficial drug for Alzheimer's.  What are the other immunotherapy drugs you see as having more potential benefit for Parkinson's?  I would like to keep track of them and their progress.  I see where the Michael J. Fox Foundation is sponsoring research for a vaccination drug that might also benefit existing Parkinson's patients if it demonstrates efficacy. 

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