Tasignia

[maier1959]

Hello, has anybody more information about the therapy with nilotinib, one study with eleven Patients showed very good results (improvent in 10 patients). Open Study not doubleblind). If the results are correct, the "holy grail" of parkinson therapy.

gumc.georgetown.edu/news/Ca...

To me it seems likt a therapy with antibiotics, you need no double blind studies to find a proof for the effect. (If you have 10 Patients with bacterial pneumonia without therapy 8 or 9 will die, with antibiotica none or one).

How long will it take until this medical drug, which is already approrved for the therapy of CML will be available for parkinsons patients?

[whack-a-mole]

Yes, from the point view of the CEO and team at Novartis Pharmaceuticals (manufacturers of Tasigna, nilotinib, imatinib) this is certainly the "holy grail" for their industry. Imagine the billions upon billions they now stand to make off the millions of desperate Parkinson's sufferers throughout the remainder of their lives!

But for those of us 'sufferers' who continue to hold out hope for something that will halt or reverse the progression of our ever worsening symptoms, this is NOT the "holy grail" by any stretch of the imagination.

"The cost of nilotinib treatment is £31,711 [US$45,675] per year, assuming a treatment regimen of 400 mg twice daily." nice.org.uk/guidance/ta241/...

This drug involves committed dependency at great expense and must be taken religiously throughout the life of the patient (cessation results in accelerated regression).

This is sheer nirvana for the pharmaceutical company gaining rights to market the drug, but a nightmare scenario for most patients. Regenerative medicine remains our greatest hope for ultimate freedom from the disease (and from the pill vendors).

[maier1959 in reply to whack-a-mole]

Hello whack-a mole.

If the results are confirmed the therapy of parkinson´s disease will change from symtomatic to more curative treatment, The therapy will we more effective and "regenerative " medicine will loose more believer. The price is about 4 times lower, and will go down in future, lately when the patents finish. Dopamineric therapy (f. e. Requip) has also a high price and a lot of side effects (I had a pleural effusion under thserapy with dopaminagonist).

The dependence to a drug plays no role, I am dependent f. e. on Dopa, as most patients after 12 years of parkinsons.

[park_bear in reply to maier1959]

Dopamine agonists are truly dangerous drugs.  They commonly cause impairment of the postural regulation of blood pressure, the result being orthostatic hypotension (stand up, faint, fall down). 

[stevie3 in reply to park_bear]

I had read a great deal about the adverse side effects of DAs and was very reluctant to take them, particularly as I've i)a history of compulsive behaviour in my youth and ii) issues with blood pressure. But the quality of my life was being impaired and so earlier this year, I slowly started on ropinirole. I began with the tiniest dose and tirated up very gradually, under the watchful eye of my GP and I monitored by blood pressure daily. My husband monitored my behaviour! I was prescribed 8mg slow release by my neurologist but my GP encouraged me to experiment and I'm sticking at 4mg for now. Apart from a little nausea at the start, and some tiredness, I have experienced no ill effects. I started to take them during a week when I didn't have to do anything major or any driving. I feel much more like myself than I have for some time and I'm able to get on with life much more easily. I think we all respond to different medications individually and it's important to be aware of the potential side effects but also the potential benefits of the judicious use of medication. I'm very fortunate because my GP treats me like a grownup, and our relationship like a partnership in managing my health. I know that isn't everyone's experience.

[park_bear in reply to stevie3]

Glad it works for you. Monitoring blood pressure daily and having someone keeping a watchful eye on behavior are both essential if one is going to try a DA. Good for you and your team that you went about this the right way.

[stevie3 in reply to park_bear]

Well, that post from me was a year ago! I can report back that I ended up on 6mg ropinirole and it's working very well, in tandem with the supplements and exercise, I'm glad to report no side effects from the DA and my blood pressure is OK - I'm on some medication for my BP which has always been what my GP calls: 'comically high' but is well controlled. Interestingly I have a very low resting heart rate.

[silvestrov in reply to park_bear]

Here is an interesting article about DA entitled:

Parkinson’s medication blamed for sexual offences

This article is from the University of Oxford, Practical Ethics, and it goes way beyond looking at the behavioral effects of DA into legal issues. It is a thoughtful article.

blog.practicalethics.ox.ac....

[FMundo]

Its a total bafflement to me why everyone (Michael J Fox included) is sitting on their hands on testing out Nilotinib for efficacy. Its is a good possibility that, in addition to being "safe," it is efficacious. Will it cure Parkinsons? No. Will it slow progression? Probably. Will it stop progression? Possibly. Will you need to take the drug for the rest of you life? Yes. Will you get answers to these questions in your lifetime (if you have PD, probably no).

Certainly the researchers at Georgetown who were trumpeting the drugs efficacy (symptoms halted, people not walking were walking, not talking were talking, etc, etc) ... as the design of their tests - to "test for safety" it most certainly was inappropriate to be crowing about the drug's efficacy. Ah well I'm sure the temptation to secure notoriety and add to one's resume was irresistible. One would think that Clinical Test funding would be "no problem" . . . Novartis (with patent rights to the drug) should logically be leading the charge for testing a drug that would have value for tens of millions of people. They are, meanwhile, making a pile of money selling it at astronomical prices to patients with cancer. Perhaps application for low doses for people with Parkinsons threatens that cash cow in some way I haven't been able to discern.

You can acquire Nilotinib from Canadian Drug distributors for far less than $45K a year... more like 12K. You could join 200 folks with PD who are taking the drug and not waiting around, as doctors recommend, for Clinical Tests to begin. Well, ladies and gentlemen (MD's in particular), its been a year now, NO FURTHER TESTING... And the "testing" .... if and when it ever gets started will, no doubt, be to resolve the question "What are the long term effects of use of the drug?" So it will take a number of years to complete the testing. The people "waiting for results will, alas, no longer be waiting.

The choice is, quite simply yours to make (as to whether or not to take the drug) . . .

[drozdown]

Lots of bad info in the previous posts.

1. The dosage in the phase 1 test was 150mg/day capsules and 2 ea 150mg/day capsules. So the test subjects took either 150mg/day or 300mg/day. These doses are well short of the 600 to 800mg/day the cancer patients take.

2. Georgetown Medical will be starting phase 2 testing in Jan2017. MJFF also doing a phase 2 next year.

3. Phase 2 testing will NOT be to determine the long term effects of taking nilotinib (Tasigna). No need to, as cancer patients have been taking it for NINE years.

My wife, a 22 year parkie, has been taking 150mg Tasigna for 8 months. She feels much better with the Tasigna. Not much change in balance and gait though. Stiffness and tremor much better. Internal feeling much better.

[Paddler4 in reply to drozdown]

I have had my husband on 200 mg a day for 16 months now for PD with Lewy Body Dementia. It may have slowed progression, but the LBD is progressing more than the PD. I am thinking of doubling the dosage.

[Bretray in reply to Paddler4]

Thank you for sharing. I am considering this medication for my mom. In what way is the LBD continuing to progress?

[park_bear in reply to drozdown]

Glad your wife is doing better. One other thing missing from previous posts is about 40% of patients have skin reactions to nilotinib. I personally have an adverse skin reaction to even subtherapeutic dosages so this is not an option for me.