This leukemia drug is being researched for application in Parkinson's by Dr. Charbel Moussa at Georgetown. His theory that PD is a vascular disease (defective blood brain barrier) is a radical departure from the decades-old alpha-synuclein/misfolded protein theory, but given what I think are very positive results from his work with PD patients and this drug, may be correct.

I tried to get this drug for my husband several years ago, from many doctors, but no one was willing to off label it for us. Dr. Moussa's work with Nilotinib was in its infancy, and none of the doctors I approached was familiar with it, or him, and thus completely unwilling to script it for us (even if we signed a waiver of any liability and full consent).

With this newest update on Dr. Moussa;s research, hopefully that will change (also with the "right to try" law now on the books). I can't help but wonder if this drug could have saved my husband's life. I'll never know, but am putting this out there so that those who come behind us can, armed with the new research demonstrating beneficial use of this drug for PD, discuss it with their doctor and perhaps be allowed to try it.

Here is one of the articles announcing the newest research results from Dr. Moussa's lab:

Parkinson’s Disease Trial Reveals Defects in Blood-Brain Barrier

If the Parkinson's of the 75 "moderately severe" patients in this study was not only stabilized when Nilotinib was taken, but improved, is that not a cure?

I cannot wait to see how Dr. Moussa's next larger trial-Phase III I think it will be-of this drug goes.

Hang in there, everyone

Less good results from a trial reporting last year:

"In this 6-month, multicenter, randomized placebo-controlled clinical trial of 76 participants with moderately advanced PD, nilotinib at 150-mg and 300-mg daily doses met prespecified safety and tolerability criteria. There was no evidence of symptomatic benefit of nilotinib on any measures of PD disability and there was trend toward worsening in the motor function in active treatment arms;"

"Efficacy of Nilotinib in Patients With Moderately Advanced Parkinson Disease
A Randomized Clinical Trial"

Efficacy of Nilotinib in Patients With Moderately Advanced Parkinson Disease: A Randomized Clinical Trial | Movement Disorders | JAMA Neurology | JAMA Network

John

Hi John,

Thank you for sharing that article, but to me, I don't buy it. I appreciate science's cautious approach to advance, but I think Dr. Moussa's promising work is such a radical departure from the established line of research (and "accepted" theory) in PD/dementia that it represents quite a threat. I anticipate a tremendous amount of push back because if he is correct, pharma stands to lose billions of dollars in PD drug sales, researchers all over the world could be out of a job, and what would happen to all of those Parkinson's Centers of Excellence?

I am reminded of the ridicule, even villifying, of Dr. Peter Jannetta (highly esteemed neuro but sadly now deceased) when he was working on what is now the gold standard procedure for trigemina neuralgia. He was correct, and he persisted despite professional ridicule. Countless patients have been saved immeasurable suffering by the procedure he invented and pioneered, and which is now known worldwide as The Jannetta Procedure.

It is perhaps more than coincidental that both Dr. Moussa and Dr. Jannetta approached things from a vascular point of view. Dr. Jannetta's famous procedure involves moving blood vessels away from the trigeminal nerve, as the blood vessel(s) pulsing against it is apparently the cause of the problem.

In his later years, Dr. Jannetta treated a patient with both well-established PD AND trigeminal neuralgia. He performed his Jannetta Procedure, and when the lady woke up, both her pain and spasms from TN, AND her PD symptoms were gone. It made some news, and I tried to get this procedure for my husband but Dr. Jannetta passed away and no one else was remotely interested or willing to try this surgery for my husband (not faulting that-it is brain surgery and would be experimental at that).

The point being that Dr. Moussa's work represents a massive upheaval and disruption in the way PD is both researched and treated. I have researched PD and dementia for almost twenty years now. I think that there are a lot of very powerful forces that will not only not welcome Dr. Moussa's work, but will actively try to discredit and discourage it. Billions of dollars, annually at that, is made from PD treatments, research, therapies, etc.

I am not at all surprised the article you cited came from JAMA...and so soon after Dr. Moussa announced his earlier results! Simply amazing (sarc).

Sorry to be so skeptical. IMHO I believe Dr. Moussa is on to something very promising and faces an uphill battle to get his results recognized, just like Dr. Jannetta. I hope he is able to succeed

lurkingforacure,

You are right to bring up Dr Jannetta.

Here is an excellent thread on this forum from 2012 which discusses the implications of his work for Parkinson's.

Regarding Dr. Jannetta's recent article on PD...

John

I am curious, Johnt, what your take on Dr. Jannetta's MVD for PD is, as well as Dr. Moussa's work-which involves not surgery to physically repair a vascular issue, but a drug which affects a protein which improves the vascular function of the BBB?

I wonder if both can be correct, that is, some people's PD is cased by an unnatural entanglement of blood vessels around the peduncle, while other's is caused by a defective BBB that needs some fine tuning to allow the wastes out and the glucose and nutrients in.....and there are probably many more causes of PD, since everyone's PD is so different.

If we remember that most people don't get PD until their later years, it does match up with the fact that our blood vessels and their function decline as we age. Varicose veins can appear, visibly and often bulging, which is a very physical change, and blood flow and pressure are affected. If one were to make a horrible analogy and compare leg blood vessels to those in the brain, it would beg the question of what would you do if you got a varicose vein in your brain? Isn't that basically what you would call a "distortion" of blood vessels around the peduncle? Whether they are tangled up with each other or have bulged and shifted around with age doesn't really matter-the problem is that they are not the same size, shape, and elasticity, or in the same place, as they used to be and are causing a problem.

It would be so interesting to know if Moussa performed MRI's of the 75 patients in the study he just announced the results of, and if so, if any of them had any "distortions" around the peduncle.

With the Tesla level of imaging we have reached now, T3, having an MRI of every person diagnosed with PD should be standard procedure. I wonder what that would show

"The failure of Nilotinib to provide a clinically meaningful benefit in PD patients in two double-blind studies is discouraging, but may be explained by the fact that nilotinib does not accumulate in the brain at concentrations sufficient to inhibit c-Abl."

https://movementdisorders.onlinelibr...1002/mds.28858