Is Michael J Fox organization holding up key PD research?

Is Michael J Fox organization holding up key PD research?

I have been wondering why is taking SO LONG to get the next Clinical Test underway for Nilotinib, an anti-cancer drug drug which in a small test, appears to slow or stop progression of Parkinsons. With initial (albeit a very small test) of the drug that was completed with results reported OVER A YEAR AGO... MJFF has been waffling about getting a test started and now suggests they'll be starting a test in 2017.... when initial test results were reported to the general public in August, 2015 !

Apparently, behind the scenes there has been a major dustup (turf war ?) going on between the initial research group at Georgetown University and MJFF. It really calls into question how serious is the commitment of MJFF to quickly get a cure for PD. For those of us suffering with disease... "time is a-wasting...." Here is a link to the debacle:

22 Replies

  • The article tells Moussa's side of the story. That is not the whole story. Nilotinib is pretty toxic stuff - 40% of patients develop skin rash at the 600mg/day dose. Granted the PD study used 150-300 mg/day, but I can't even tolerate 40mg once a week without getting an unacceptable skin rash. In the PD study Moussa et al only reported two minor adverse skin reactions during the treatment. Maybe that was so, but it just doesn't seem typical.

    Here is MJFF's response to that article:

    "We and our skilled collaborators are committed to answering critical scientific questions about the drug through a study of the highest quality to provide clear data for patients and physicians. While multisite, double-blind, placebo-controlled clinical trials require immense work to plan, our coalition’s ambitious goal of launching a trial in 2017 reflects our commitment to move rapidly *without compromising scientific rigor or participant safety*.

    Unfortunately, the greatest impact of this story may have been to hype the drug before it has been fully demonstrated to be effective and, crucially, *safe* for people with Parkinson’s. We regret that Dr. Charbel Moussa has chosen to level accusations against us in the press. But we bear him no ill will. Last month we granted him $290,000 for research on a separate topic. As in all his work on behalf of people with Parkinson’s, we will be rooting for his continued success."

    MJFF wisely chose not to get down in the gutter and level any direct accusations. That said, the phrases I marked off with *'s hint at what the problem might have been from MJFF's perspective.

  • The "behavior" of the Michael J. Fox organization of greatest concern to those people suffering from Parkinsons' Disease is not the "fairness" of treatment of Dr. Moussa. Nor do we care whether or not one or both parties "climbed into the gutter" for a mud slinging contest.

    What we DO care about is about, as I pointed out, the fact that SO MUCH TIME has elapsed since the results of the initial test were published. There is simply no excuse for so much time to go by - What's it going to be, TWO YEARS! by the time the new test is underway?

    The fact that the first test was improperly done (and the results inappropriately broadcast to the world) only makes it more important that a rigorous test be initiated promptly.

    Do you seriously think that people with PD buy the argument that it takes a year or two to design a good clinical test? More plausible is the idea that when competing organizations focus on "who's going to get the credit" energy gets diverted from the most important objective... finding effective therapies for PD in as short a time as possible. The article states the situation accurately I believe.

  • I am one of the people with PD. I am not some interloper hanging out here for jollies. I am also an investor in biotechnology stocks, and the sad fact is these things take an egregiously long time. If you are going to uncritically accept one side of the story that's your business but you are not going to end up with an objective viewpoint.

  • Our task as PwP's is to push organizations (researchers and those people who fund them) that play a part in finding a solution to Parkinson's at FLANK SPEED. To accept things "as they are" ("these things take an egregiously long time") is, from my perspective, unacceptable. If it takes time to design tests well that's one thing. If delay is due to "egos and 'who gets the credit'" then we have an entirely different situation. That is the situation I believe we have here.

  • You want to push fine. These sorts of accusations: "calls into question how serious is the commitment of MJFF " will get you put on ignore by the very people you seek to push.

  • FMundo you are correct, it is all about egos. I guess if I was studying this drug for a dozen of more years I'd feel like Dr Moussa to. One other thing is money.

    I volunteered for the next trial a few months back. Got my answer yesterday. GU in Phase 2 trial, will require 15 visits. Therefore they will be using local PWP. Hopefully a similar trial will get to my locale (Albany NY) soon.

    Keep the faith and keep moving!

  • Hi park_bear,

    I'm very curious as to how you came to be taking 40mg of nilotinib per week (my understanding is that the smallest Tasigna capsule is 150mg). Were you involved in some kind of tolerability trial?


  • I will message you on that.

  • We discussed this a couple of months ago. Here's the link in case anyone wants to read about it (again). You will have to search through it though, to find the relevant bits:

    More recently there has been some good news. A small company called Inhibikase Therapeutics has received FDA approval to run two Proof-of-Principle studies involving nilotinib (Tasigna) and dasatinib (Sprycel).

    As they say in their press release, they are developing two Abelson tyrosine kinase inhibitors that are as much as 60-times more potent than imatinib (Gleevec), and as much a 50-times more penetrant into the brain than nilotinib.

    Their strategy makes sense to me. Their 6 month trial can determine efficacy sooner than the trial of MJFF et al. If the result is positive, they will have learned something useful (i.e. "[better understand] which patients with Parkinson's Disease are more likely to benefit from this [Abelson kinase inhibitor] therapy" ). Alternatively, if the result is negative, they can stop wasting money immediately.

  • Jeff, wow, not only is this great news about a potential new drug they seem to register the importance of time with the planned release of not one but two "simultaneous planned clinical trials, double-blinded, placebo-controlled clinical studies in Stage 2 and Stage 3 Parkinson's patients will be conducted over a 6 month period. These trials will evaluate the ability of these drugs to slow the progression of or even begin to reverse the course of Parkinson's Disease in patients."

    Running simultaneous studies and shortening the length of the study, at least at the onset, makes so much sense, agree!

  • Ditto, I'm impressed and agree that this firm grasps the importance of time when it comes to getting Clinical Trials up and running.

  • I have chronic myeloid leukemia in addition to PD and have taken all three of these drugs: Gleevec, Sprycel and Tasigna. They are not without serious side effects. One, with Tasigna, is prolonged Q-T syndrome which can spur instant death. I get frequent EKGs and blood work. Just sayin'. Plus, it hasn't done thing one to curtail my PD. I wouldn't get too worked up about it.

  • Beckey I am sorry to hear that you have been dxd with both myeloid leukemia and PD. Managing the medications must be challenging. You have a unique perspective. I am curious about any details of your reaction to Nilotinib you care to share.

  • FMundo, I appreciate you sharing your thoughts on such an important aspect of getting to a cure for PD: How do we speed trials? This certainly is a topic worthy of our attention and focused activities.

    When I was first diagnosed with PD earlier this year, I read a book called "The Frozen Addicts" authored by a neurologist some call the father of PD. Interestingly, the latter third of the book deals with the competitiveness built into the clinical trial process. I learned it is not unusual at all for a larger organization, for example the NIH, to get involved immediately when they learn of a possible valuable drug that can speed a cure. This should not diminish the effort of the group or individual engaged in the initial clinical trial (who might feel disinfranchised from a solution they may feel they own) because finding a cure for any disease is not about ego gratification but rather helping those suffering from the disease. What is positive about the competitive nature of launching multiple trials at different organizations is that it should speed the process. While the competitive process can be challenging, it is designed to work to the benefit of the process overall.

    Moussa may have done something remarkable by using Nilotinib for PD but he may have also slowed the process with a bit of a sloppy first trial (based on criticism we see in the press) and he / Georgetown may have generated some confusion in the industry by showcasing their dramatic results early. All of this may be true, but I don't care about the mechanics of how we got here really. There is enough good in the story that we (Georgetown, MJFF and why not perhaps the NIH as well) could race to a successful launch of phase II and get to some conclusive data. A few questions surface in my mind, such as what is the typical time to get from phase I to phase II to phase III completions, what are the traditional expectations for clinical trial timelines and what can be improved? This is a great question for the group - our power is in numbers. How can we message our sense of urgency in speeding trials, together, as a group?

    Yes, of course, multisite, double-blind, placebo-controlled clinical trials require immense work to plan but have we not achieved a format to streamline this process yet? Do we not have the processes in place to already be conducting phase II trials on Nilotinib by now? Could phase II and phase III trials be run simultaneously for example? It seems that - in general - with all the advances in medicine and science - for example mapping the human genome - we could match with the rigor to determine how to speed test / trials of potential drugs that can have life altering benefits.

  • P-oui

    Some work that has been done in this area may be of interest to you. On another thread FMundo said regarding participation in a clinical trial "...for many tests, they are blind and there is a 50% chance you will receive a placebo. I don't like those odds . . ." This highlights a problem.... how do we get trials moving if we all want someone else to be the participant?

    Clinical trials are necessary to see how effective or safe certain treatments, interventions or diagnostic tests are in humans. They are also important in gaining information about a disease, how it manifests and the clinical course that it takes.

    Drug development is not an expeditious process. For a new treatment to get from the idea stage to the pharmacy shelf it takes time, usually 10–15 years .....

    Part of the problem, is the timely and successful recruitment of study subjects. As clinical trials have become more rigorous they have also grown larger and more complex, and the work burden for study staff is increasing. For those who choose to volunteer, the last decade has seen a more selective and more stratified approach to the inclusion and exclusion criteria applied to clinical trials. This narrowing of the goalposts by an estimated 58% is likely to be responsible for volunteer patient enrollment rates dropping by 21% and retention rates falling by 30%.

    The challenges facing patient recruitment are complex, currently up to 30% of the timeline for the drug development process is spent on suitable subject enrollment. In fact, 45% of clinical studies are delayed because of difficulty enrolling participants.

    The full report is here :

  • It's not easy is it. I think I read that MJFF site has improved participation for trials just by making it easier to figure out which trials are out there that are appropriate. That was a great service to PWP. MJFF has truly done a great job with the clinical trials page ... I've posted a few trials that do not require travel, etc., such as the 23andme PD study. NIH offers a clinical trials site but I find it more difficult to navigate. HealthUnlocked can be amazing in terms of sharing info with each other on what goes on while participating in a trial. I always wonder how the participants are feeling during the trial. Given what MJFF accomplished just making it easier to participate by finding a trial, I feel optimistic about improving and speeding the trial process. Thanks for sharing this Hikoi. I always appreciate reading your informed shares.

  • Glad it was of interest p-oui. As regards trial participants commenting on how the trial is going for them I think most trials will not allow that sort of discussion because as we know that expectations, positive or negative can affect individual results. We do however sometimes get comments when the trial is finished.

  • You raise interesting questions p-oui

    The time line should be shortened in investigating meds that already have been approved for treating other conditions but I don't see how you can run phase 2 and phase 3 simultaneously when each builds on the previous results

    the emphasis in Phase 1 is on safety,

    the emphasis in Phase 2 is on effectiveness. This phase aims to obtain data on whether the drug works in people who have a certain disease or condition. It involves blinded trials with placebo. the number of subjects in Phase 2 studies ranges from a few dozen to about 300.

    Phase 3 studies begin if evidence of effectiveness is shown in Phase 2. These studies gather more information about safety and effectiveness, studying different populations and different dosages and using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.

  • Hikoi check out jeffreyn's post above. I read the article after I posted my comments and was intrigued about Inhibikas' approach aimed at speeding the trial process. I would like to get your thoughts on this. Also, I posted this morning titled "Why 23andme represents an important new approach to speeding a cure for PD and other diseases". Brin, a founder of Google, offers his interesting take on medical research which might also help us think 'outside the box'.

  • P-oui

    I would need to read more to feel fully informed because I don't know what running the 2studies simultaneously mean, is it one study for each drug ? It's not clear is it? I read the stage 2and 3 to refer to the stage the patient is at not the phase of the trial process but am I right.

    I think that The article is reporting that the company has approval to run proof of concept trials which are very early trials and It hasn't started recruiting yet - so that will take a year I expect.

    While being appalled at the drawn out process and I agree with moves to shorten it, shortening the length of a study is not always to our advantage. I suspect we may have missed out on good treatments because the benefits were not immediately seen or the poorly constructed trials gave negative results or the wrong criteria were used for selecting participants, (something that may work on older onset PD may not on young onset for example. )

    Sorry this isn't meant to be negative, just some questions that came from reading the article.

    23andMe - I joined a while back, it's not without controversy but I support it. It's a unique opt in rather than recruitment and selection process.

    It's easy to find fault with any organisation but I am very grateful to MJFF for all it does, especially raising the profile of Parkinson's worldwide.

  • Agree Hikoi, I will stay tuned and let you know any new developments I hear of...

  • Where is M13? No side effects,reverses Pd?

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