The drug, already approved by the FDA for cancer treatment, improved cognition, motor skills and non-motor function in patients with Parkinson's disease, the university researchers reported.
Note: earlier/sooner FDA approval as it is existing drug
"To my knowledge, this study represents the first time a therapy appears to reverse -- to a greater or lesser degree depending on stage of disease -- cognitive and motor decline in patients with these neurodegenerative disorders," said Dr. Fernando Pagan, an associate professor of neurology. One patient confined to a wheelchair regained the ability to walk and three others individuals who were unable to speak before the trial were able to conduct conversations. However, the researchers noted that larger and more comprehensive studies and trials would be required to gauge the drug's true potential impact.
"The use of nilotinib (AMN107, trade name Tasigna), in doses much smaller than are used to treat cancer, which is up to 800 milligrams daily, was well tolerated with no serious side effects," Pagan explains.
For the trial the doses were kept between 150 and 300 milligrams daily, he added.
According to the researchers, one of the reasons for the trial's success was that Tasigna was able to penetrate the blood-brain barrier more effectively than existing Parkinson's drugs. Moreover, they are already planning larger clinical trials with the drug for Parkinson's and other diseases including Alzheimer's.
Regarding "online contact form" Response time was about an hour for my request.
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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.
Thanks for the information. Here in UK there have been a number of press articles about this. It would seem to mesh with initiatives being promoted here about the placement of drugs which are approved for use in different (from PD) conditions which have been shown to benefit PD as a side effect. I shall certainly be looking for trials recruiting the effects of this drug.
Bah! Phooey! I've been taking Tasigna for leukemia for some six years or so. It sure didn't stop me from picking up a case of that Parkinson's bug that's been going around.
Since you were taking the higher doses, maybe the drug caused some neurons to die. Looking at a paper it "reduces astrocyte and dendritic cell number ". What dose are you taking? This work says they gave it at 1/5 to 1/3 normal doses, and in looking at the animal studies that led to this research, I see they were giving only 1/50 in human-equivalent doses. It looks like it should have noticeable benefit at only 15 mg instead of 150 mg.
The method of action is c-Abl inhibition. The connection with Parkinson's was discovered 5 years ago by John Hopkins. There have been 14 papers since then with on "c-Abl parkinson's" in the abstract or title, and none before.
Two years ago, this group tried this drug in mice.
Any nutrient or drug capable of c-Abl inhibition probably works.
The two research groups that previously published on the c-Abl connection have jumped on this treatment, testing it in mice. Here's their research papers:
These are the c-Abl inhibitors, most used for leukemia. Blocking c-Abl may help get rid of the bad a-syn, but c-Abl is also used for "neuronal plasticity, neurite outgrowth, and neurogenesis" and "required for optimal synaptic function" so taking too much of it is not good for the brain. Keep in mind that lower doses are needed for PD, so the toxicity dangers are not as great as the ones I've pointed out. Switching from one to the other may be important as the body develops protection mechanisms against them. So if you have 3 choices, you would try to alternate them every 3 months or something like that.
Nilotinib
seems to be the one of choice due to less toxicity. Price might be in the $100,000 a year range. PD patients should not expect it to be less because the Pharmaceutical might require off-label use to charge 10x more even if you use 10x less. They charge based on patient desperation and ability to pay, not based on cost to develop. It does not matter if all their costs were paid by taxpayers through research grants. Grapefruit "interferes" with it as it is a substrate of the protein CYP3A4 that grapefruit blocks. I believe this means grapefruit will make a lower dose more powerful (more bioavailable for its benefits as well as its toxicity). I did not know if the others are also affected by grapefruit. There are "competing" pharmaceuticals, but it seems they all want $100,000 to take these drugs. Be "thankful": I know a baby who had brain-damaging seizures and it was costing $50,000 a WEEK and was known to probably not work. "Dude, get this $50,000 glass vial (1 teaspoon) out of my hand before I drop it. That's not funny putting something like that in my hand and then telling me what it is."
imatinib
the original, not as good as the others. $100,000 a year for leukemia patients. Does not efficiently cross blood-brain barrier in humans.
Ponatinib
(Ariad pharmaceuticals), works good, but was halted in U.S. due to toxicity (blood clots). Still available in U.K. to leukemia cancer patients. Cost would have been about $100,000.
Bosutinib
may not be good because it stops the bad-protein removal mechanisms, even as it dissolves a-syn. Maybe at lower doses it is OK. But it worked in mice when it was being compared to nilotinib.
Bafetinib
(INNO-406) newer, looks good, may not be available. Efficiently crosses blood brain barrier. Molecular weight is 576. Tested in PD mice:
(Bristol-Meyers). looks good except can have several fluid on lung build up in 20% of patients on the high doses needed for leukemia. 15% lower molecular weight than bafetinib (488), so may cross blood-brain barrier. It has a lot of studies on it for PD, but I noticed one seems to claim it did not work as well as bafetinib. U.S. gov is trying to stop indian manufacturers from making it in order to maintain Bristol-Meyers patent rights. $133,000 a year for leukemia patients. $1,500 a year in India if they are getting around the patents. Same situation for rasagiline, $50 a year in india, several thousand here. Almost all of Bristol-Meyers cost in developing it came from tax payers through NIH research grants and a 50% tax CREDIT (which can lead to tax REFUNDS).
Green tea extract, olive oil, and fake-curcumins are so far the only compounds I can find that also inhibit c-Abl.
If you've read my posts you know I believe there is a connection between PD and cancer. Viruses and mitochondria dysfunction are two connections. This c-Abl connection is another: "The c-Abl kinase protein is one of the most studied targets in the fight against cancer and is a hotspot for drug development because it participates in several solid tumors and is the hallmark of chronic myelogenous leukemia."
I keep repeating the lack of oxygen increases PD progression. It turns out intermittent hypoxia (as may occur every night when we sleep) increases c-Abl.
Curcumin: a potential neuroprotective agent in Parkinson's disease.
Curcumin exhibits antioxidant, anti-inflammatory and anti-cancer properties, crosses the blood-brain barrier and is neuroprotective in neurological disorders. Several studies in different experimental models of PD strongly support the clinical application of curcumin in PD.
Turmeric contains curcumin, the polyphenol identified as its primary active component and which exhibits over 150 potentially therapeutic activities, which include antioxidant, anti-inflammatory and anti-cancer properties.Curcumin is unique in its multiple actions in protecting dopamine neuronal loss through regulating enzymes breaking down dopamine and hitting multiple signals and genes “turned off or off “. Curcumin also reduces inflammation. Curcumin formulations can be the novel frontier in PD therapeutics.
end item
While natural supplements and alternative therapies are becoming more main stream, clinical trials using natural compounds, including curcumin, to treat neurological diseases have been disappointing thus far.
Novartis ($NVS) remains in the fight over a patent on blood cancer treatment Glivec/Gleevec, while reports suggest the Indian government has mulled a compulsory license on Roche's Herceptin (trastuzumab).
In the case of BDR, the aim was to sell dasatinib at about INR8,100 ($127.83) a month, compared to INR100,000 ($1,535) by BMS. The Indian Patent Office in 2013 had also rejected a compulsory license application by BDR, saying it failed to try the proper channels to obtain a voluntary license from the patent holder.
First, there's a lot of money involved. At these prices only 5 patients need to take it for 5 years to pay for the researchers to (justly) become multimillionaires (if it works as well as they say). And if it does not work so well? Well, they still become millionaires. They have nothing to lose. The pharmaceuticals need to direct the profits from only 5 to 10 patients to reward the researchers who previously had no agreement with them so there is no conflict of interest. Then the pharmaceuticals and wall street can get the profits from thousands more patients (billions of dollars) before these patents run out. This means the researchers are guaranteed "good old boy" support from any one of the 4 pharmaceutical companies that own one of the drugs I listed. There is going to be a push to get this through before the patents run out. Off-label use should allow it to happen quickly. Maybe anti-inflammatories or a combination of green tea and olive oil would work as well, but there is not enough financial motive for the research to be done in humans. For at least a decade research in animals has shown green tea works wonders in PD. Money is why it has not been formally tested in humans.
It took only 2 years from the first animal testing to get this drug tested in humans. It takes 20 years to go from animal research to human testing for natural compounds. Money is the difference, not efficacy.
Second, the 2 videos from these very intelligent researchers is amazingly useless. Have you seen the before and after images of "cures" for wrinkles? That's what it reminds me of. The before video of the lady does not show her holding a spoon, but clearly they could have done that. Why not show an after video of her getting out of bed and into a chair? I wonder if they DID make a before video of her holding a spoon, but decided not to include it. The after image of the man cuts the video off where you can't determine if his gait has actually changed. So the videos do not demonstrate any difference that I can see.
Third, the research paper does not seem to be available. Technically speaking, no research has been done in people until they get it published.
I was just at the docs with my boyfriend with his PD and I asked about this drug and his doc says it can cause instant death and she would not even think of about prescribing it. The pharma people have neglected to mention that fact I guess
The follow up question is "at what dose?" A very small fraction of the regular dose is used in Parkinson's patients. In the Georgetown study no one died of angeles thing that was clearly caused by the drug. Stroke and heart attacks occur in this elderly population regardless of treatment with tasigna/nilotinib.
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