After reading someone else's post about this medication being used in clinical trials for PSP and other neuro-degenerative diseases and seeing that it had already been cleared by the FDA (we are in the U.S.) for a cancer treatment, I have asked our neurologist to prescribe it for my husband as an off label treatment. Physicians in our country can do that. I sent this correspondence along with a copy of the article about the study (in Human Molecular Genetics magazine, May 10, 2013, by Hebron, Lonskaya and Moussa) to the physician by mail so I can't expect a reply for at least a week and maybe longer. If the drug works it is NOT a cure. We are hoping for more physical control in his limbs. Thanks to whoever posted that link. I will let everyone know what, if anything, happens.
Carolinesimmons
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carolinesimmons
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If you obtain permission to use Nilotinib, I would be extremely interested in the results (as most would on this forum). In the USA it has a black box rating. The quantity administered would be very much lower than that used in the cancer treatment. I wrote the following about this compound a few moths ago on this site....I have not edited my old comments, so they may not be up to date ..........
PeterJones thought I might be interested in this research. Well I've had a quick look and it sounds very interesting. The following is my understanding and I don't know if anyone really wants to knows the "ins and outs" of the research. The news item does say "potential" treatment, so we would have to wait and see, hopefully with optimism.
Some think that the death of neurons (and other brain cells) in PSP (and related diseases) can be attributed to the formation of "garbage" (called neurofibrilliary tangles plus other bits) as described in this research news. They have been successful in mice in "removing" (by a process called autophagocytosis) this garbage without destroyng the neurons (by using a drug called nilotinib - used normally to actually kill cancer cells in a certain type of leukaemia).
It sounds like a very useful clinical trial, and they say it can remove the tangles (caused proteins that have "gone wrong" and destabilised the neuron, causing cell death- those proteins are called tau in PSP/CBD and alpha synuclein in Parkinson's, and other proteins for other neurological diseases).
They have to use a small amount (about a thousand time less than for killing leukaemia cells) because they only want to remove the garbage without damaging the neuron itself.
It sounds exciting, but one must be reserved until it has had full clinical trials. The drug itself has some serious side effects.
I see a few reservations (but I'm not an expert in this field), and I don't want to sound negative but I guess I will!!!! The researcher admits that this drug should be used "early" in the disease, and we know that the tangles (garbage) can start 15-25 years before symptoms, so how early is early.
Then some feel the tau protein that forms into garbage (in PSP/CBD) starts out doing so as defence mechanism (that is, it's good and not bad). Then there is the problem of being successful in mice. If everything that has been successful in mice translated to success in humans we would have many cures by now. These research studies are often very controlled, and quite unlike the many complex mechanisms making up neurological diseases in the human brain. Finally, other current research has shown in autopsy PSP that some areas of the brain had lots of tau garbage, and little neuron death, while others had very little tau garbage and lots of neuron death. Such researchers think there are more factors that cause the PSP status than the conventional thinking of being tau garbage alone.
I was thinking of deleting all this because it sounds pessimistic, but someone may want to see how I approach such news as potential treatments for PSP.
If anyone reads this and sees errors in my statements, I would really welcome their input.
Let's trust this potential treatment does what it says in the clinical trials and we all see a light at the end of the tunnel particularly for those newly diagnosed with PSP and similar conditions.
Well, Strelley, you do sound a bit pessimistic. But that having been said, I am glad to have more input into the potential use of Nilotinib as I only knew that a much smaller dose was being used - not how MUCH smaller we were talking about. I hope our neurologist will not only actually reads the original article in Human Molecular Genetics but will actually call Dr. Moussa at Georgetown University where the clinical trials started.
My expectations are very low anyway and expect if there were to be any improvement, it would only be in limb movement. My husband can, with some difficulty, raise a glass to his mouth and use a walker with assistance. That's it. He is under hospice care and I don't know how any use of a medication like this might impact that. But, as you can well imagine, we grasp at any little straw that comes along.
By the way, what is "black box rating" and what are the "serious side effects" of Nilotinib?
Don't stop using this sight to keep us all as informed as we can be. Your knowledge is invaluable to us! Thanks.
Sorry for sounding pessimistic. I think it results from my attempt to be realistic with the facts that have been presented. As I have already mentioned, I am not expert in these matters, but I do try and assess the "science" presented in such research, and search for the cautions expressed by the researchers themselves.
We do not have black box warnings in Oz, but they are used in the USA by the FDA as the sternest warning on medications that are still on the market. It is an alert to serious or life threatening risks. In the case of Nilotinib it can cause heart problems that may be fatal (plus other side effects like liver and pancreas problems plus bleeding on the brain). Having said that, its use in treatment of a certain type of leukemia had safety approval through clinical trials. As mentioned in my previous post, this medication will be used at a much lower dose in Parkinson's clinical trials.
I think it is understandable to try anything that may improve the symptoms of PSP/CBD (and Parkinson's). Like the researchers, I think the benefits of such medications as Nilotinib would be better assessed in the earliest stages of these diseases.
Take care.
T.
• in reply to
Dear Strelly, I think your observations are spot on. My wife suffers from PSP and I am obviously also very keen to hear about any new developments regarding a possible treatment that could alleviate some of the symptoms, even if it is not a cure. But not at a huge risk. My recommendation regarding nilotinib is 'let's wait and see'. It is a very nasty sort of drug with horrendous side effects.
I am going to see Dr James Rowe on the 20th of this month who is running the Neuroscience Department at Addenbrooke's Hospital in Cambridge and who specialises in CBD / PSP and similar neurological disorders. So I will discuss this matter with him and let you know what he has to say.
We have had over the past two years spectacular failings with drugs that where meant to have an effect on PSP and other neurological disorders. The Davunetide drug trials spring to mind which crippled the Canadian pharmaceutical company Allon Inc. in the process. It was on of the spectacular flops that should sound as a warning not to become over optimistic.
Unfortunately we have to realise that even a condition like Parkinsons has not resulted in an effective treatment despite the fact that it has been around for a long time.
It is only now that Governments are waking up to the fact that neurological disorders are costing society a fortune and only because of that they are now starting to pump real money into research. So we are still in the infancy stages of it.
Although it is a sensible approach to look at existing drugs to see if they might have a beneficial effect on an illness (for obvious reasons), it is much more important to find out what triggers the disease and what is its pathway. At this moment the medical world has not a clue, they cannot even classify the disorders properly. So unless we see some progress in fundamental biochemical research we continue with the classical machine gun approach and the chances of success are minute. Obviously it would be nice if somebody would hit the jackpot. Gerko
• in reply to
Hi Gerko
Good to hear from you. I would be VERY interested in the opinions of the Addenbrooke specialists in PSP/CBD.
I agree with you about the need for better progress in fundamental biochemical research. While researchers have discovered a multitude of faulty processes involved in these neurological diseases, along with some compounds that prevent some of these faulty mechanisms, they have not discovered the initiating causes. It is very complex but hopefully they'll soon crack the problem.
I'm guessing that the solution to PSP (and other tauopathies) will lie in the Alzheimer's research. I think this has the most money being thrown at it! More recent Alzheimer research is highlighting the tau problem and not just the beta amyloid problem.
Thanks, as ever, Strelley, for giving us the benefit of your considerable knowledge. Just reading your posts makes me feel less powerless! (And that is a rare and wonderful feeling these days.) Cautious pessimism seems much more appropriate for this drug than the opposite, but of course I'm very interested to learn what carolinesimmons and Gerko discover. Thanks to you both, too. With an outlook as dire as the one we are all facing, taking a chance with a potentially dangerous drug doesn't necessarily seem like a bad decision. Many of us are experimenting with diet and supplements like CoQ10 - and all we've been promised is that that can't hurt. Hope is a fine thing. EC
Thanks to Strelley and Gerko for keeping my feet on the ground and I'm determined to keep my head out of the clouds. You both sound so smart and in tune with the science behind everything. I'm fairly new to all this and I probably got carried away. I see my husband locked in this non-working body and it tears my heart up - so I grasp at straws.
But let me tell you the wonder of it all. This morning I went with our pastor to take communion to my husband. We (the pastor and I) were just talking about this and that and the conversation turned to sports. We were trying to come up with the great basketball player from the University of Virginia during the days when my daughter went there. WE kept say "what IS his name?" Out of the blue, my husband said "Sampson." And the pastor said, "Yes, Ralph Samson!" So that PSP - wounded brain is still right on when it comes to memory and intellect. It's just so hard for him to get it out.
It's things like this that keep me going. I KNOW he is still there.
I understand how this awful disease exacts its toll on both you as a carer and your husband, as the sufferer. We have to hang on to those "bright moments" that your hubby showed when recalling that basketball player's name.
My wife's speech is now very slow and sometimes I find it hard to hear some of her words. However, her memory is really marvellous. Far better than mine. She remembers small details of our life together as we raised our children, that I have absolutely forgotten. She remembers birthdays of both family and friends.
As PSP extracts so much life out of our loved ones, we try and enjoy what remains, and attempt to keep them as comfortable and pain free as possible.
The Georgetown study used 150 and 300 mg. THey were treating Parkinsons, and have not started testing with PSP patients yet. medstargeorgetown.org/2015/...
This drug, and the immunosuppressant Rapamycin appear to work by convincing the Metabolism that the cell is not receiving enough nutrition, so the cell stops Growth, and Triggers Autophagy (recycling of unnecessary cell parts). ncbi.nlm.nih.gov/pubmed/200...
At higher doses these medicines trigger apoptosis (Cell-Suicide) because the cell is seen as too damaged to be allowed to grow/reproduce...
May I ask what you are paying for the drug. I did a search, and requested cost from every place I found. The best price I was able get wasn't for the generic, but the original drup Tasigna. I'm paying $700 US per box of 28 capsules. I get it from India. When my wife had to have some surgery, the hospital force us to have the drug tested. It turned out to be the real thing.
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