At their best, drugs for Parkinson's disease (PD) only slow the progression of the disease. But now a small clinical trial has shown that a cancer drug already approved by the FDA may have the potential to reverse PD symptoms, both motor and non-motor. as well as symptoms of depression.
The new hope was announced at the recent meeting of the Society for Neuroscience by researchers at the Georgetown University Medical Center. Using a drug developed for leukemia patients, the scientists cut the dose, and gave it to 12 patients who had either advanced PD with dementia or the related Lewy body dementia.
Dr. Fernando Pagan, director of the Movement Disorders Program at Georgetown, spoke at a meeting I attended last week. He acknowledged that this study involved only a small sample, none of whom received a placebo. The gold standard element for drug efficacy trials is "double-blind," where neither participants nor investigators know who gets the drug and who gets the placebo.
He said a larger clinical trial is planned and hopefully will start in early 2016. I asked Dr. Pagan when, assuming the expanded trial went well, we might be able to begin using the drug. He estimated it would be 3 to 5 years.
For more on his potential breakthrough, see my blog post at bit.ly/1lDGMiT.
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gleeson
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Study and follow-up expanded study are at Georgetown Hospital here in Washington, DC where I live. As I said in my post here and in my blog report, I asked Dr. Pagan, assuming the follow-up confirms the original findings, when might the drug be available. He said 3-5 years.
It's never too late as long as the person is otherwise healthy. At Baptist Hospital in Winston-Salem, North Carolina they've performed Bi-lateral DBS (both hemispheres of the brain during a single operation) on a 79 year old (man). At 55 now myself, I knew I couldn't go wrong by having it done. 1st side at 53 YOA and the 2nd side at 54 YOA. I get to walk my daughter down the isle now!
That's probably the cost for Leukemia which uses 800 mg instead of 100 mg. Tasigna (nilotinib) is a CYP3A4 liver enzyme substrate which means peperine, grapefruit juice, milk thistle, and ginkgo will increase the amount that gets to the blood stream. In particular bergamottin in grapefruit juice. It's an insecure if not dangerous game to try to do this, but getting the dose down to 25 mg could be expected, but not predictable individually. That would be $281 a month. If that $9000 is the price in the U.S. then you should get it in other countries for 3 times less.
As I reported on my blog, the Parkinson's/dementia study used 150 and 300 mg contrasted with 800 mg for leukemia. So the cost for us PWP's would only be $2000 to $4000 a month
Does pharmaceutical pricing work in a way that has a straight line apportioning of cost according to dose? Or are patent drugs typically priced based on what the market will bear?
Most of the cost may be wrapped up in development, approval and marketing, with only small increases in cost being directly related to manufacture of the active component.
Don't know what history has shown, but it's worth asking the question.
GM1 with a phase 3 trial in 77 patients completed 5 years ago is already the first Parkinson's FDA-allowed drug that stops the disease from progressing. The problem is that pharmaceuticals aren't interested in it and it will take an enormous number of patients demanding it before it becomes affordable (it has to be derived from animal sources like sheep or cows).
Also ferriprox (iron chelator) is in trials with success indicated.
The nilotinib researchers need to at least publish something that supports their claims instead of going straight to the media.
Any further news here re. Tasigna? I don't understand why it would take 3-5 years. It's safe, on the market already, get it out there people are desperate
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