Nilotinib - lastest update - not the best news
 

Unfortunately, the never ending saga of Nilotinib continues on. There had been several stories floating around about problems between MJFF and the Georgetown research team as the reason that GU couldn't obtain funding for a larger phase 2 study. I didn't think the problems were as bad as that reported in this article. Nevertheless, the GU trial is still not fully funded and will continue to be delayed. The MJFF trials are probably a year away from beginning, if not more. Meanwhile, the PD community waits, twiddling their fingers with frustration, as to why it takes so long to proceed with a trial for a well known drug that has been approved and on the market for years. I am willing to bet that I could get 100 volunteers in one week, who would sign waivers and begin taking the drug immediately. It's very hard to maintain patience when one has a progressive degenerative disease. It's much easier if you just fund or do the research, your life is not on the line.

Parkinson's disease study caught in feud involving Fox Foundation

Kind of sad they all can't be on the same page which is helping all of us that suffer. Hopefully they will get it together without greed and glory leading the way. Thanks Tup.

Eric

Discouraging to say the least. Seems there are internet sources and buyer groups for those inclined not to wait 5 plus years for nilotinib to be approved.

In the video [1], showing a patient walking before and 6 and 8 weeks after starting nilotinib, it is interesting that in all cases he is leaning to his right. I assume that the conditions, e.g. time since last dose, are the same on all three occassions. I associate a lean, caused by dystonia, to be a reasonable measure of integrated exercise or conversely the effectiveness of the therapy. It could be argued that a progression delaying drug doesn't need to show improvements immediately, but over 8 weeks I would have hoped to see something more. Given that, I am disappointed by the results.

As Blackfeather says there are people making group purchases. One of which is on the LongeCity forum. Over the last few months they have arranged a number of group buys, at a much reduced price. Interestingly, none of them are reporting great benefits so far.

The problem with assessing progression delaying drugs is the resolution of the measuring procedure. Suppose you had a drug that unbeknown to you stopped progression, but did not reverse it. For the sake of argument, suppose that the annual decline is 12% or, approximately, 1%/month. Then, to see an effect after a month you need to be able to measure with at least this accuracy. You can improve accuracy by taking more frequent observations or increasing the group size.

Reference

[1] Parkinson's disease study caught in feud involving Fox Foundation

[2] Nilotinib Group Buy - Page 15 - Brain Health - LONGECITY - Page 15

John

More Nilotinib news from MJFF

Parkinson's Disease | Foundation Statement on Activities Related to Nilotinib for Parkinson's Disease

Letters to the Editor post STAT article
 

As others have stated, we at MJFF felt this nilotinib story was intentionally one-sided. I wanted to share MJFF's Letter to the Editor of the Boston Globe: https://www.bostonglobe.com/opinion/...1NM/story.html


Additionally, a separate note from Boston-based experts Simon and Schwarzschild is here:

Hype about Parkinson’s drug is unwarranted

WE WERE disappointed that the tone of the Stat article “The patients wait” further spread the unwarranted hype about nilotinib. For example, nilotinib is referred to as “the most promising new treatment for Parkinson’s disease in decades,” and in the online version, videos are shown of a patient before and after treatment with this drug.
The published study involved only 12 patients, without a placebo group. The placebo effect is strong in Parkinson’s disease, and so no conclusions regarding symptomatic benefits can be made from the doctors’ and patients’ reports of clinical benefits. Parkinson’s disease patients have many reasons for hope, but exaggerating the significance of early clinical data is counterproductive. These words are not without consequences. Some doctors are now prescribing nilotinib to Parkinson’s disease patients despite a lack of sufficient data regarding efficacy or long-term safety in these patients.
On the other hand, nilotinib is indeed worthy of further study. The focus should be on how to move forward with the best-designed, scientifically rigorous placebo-controlled study as a step toward more definitively determining whether or not nilotinib will be of benefit for Parkinson’s disease patients.

Dr. David K. Simon
Associate professor of neurology
Harvard Medical School
Director
Parkinson’s Disease and Movement Disorders Center
Beth Israel Deaconess Medical Center
Dr. Michael A. Schwarzschild
Professor of neurology
Harvard Medical School
Director
Molecular Neurobiology Laboratory
Massachusetts General Hospital
Boston
The writers are members of the Linked Clinical Trials scientific committee established by the Cure Parkinson’s Trust, have received funding from the Michael J. Fox Foundation, and conduct clinical trials for Parkinson’s disease.