Phoenix Rising tells QMUL: release the PACE trial data
Mark Berry, Acting CEO of Phoenix Rising, presents the Board of Directors’ open letter to Queen Mary University of London (QMUL) urging them to release the PACE trial data, and hopes that other non-UK organisations will join British charities in the same request...
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What research would you do if you had £1M?

Discussion in 'General ME/CFS Discussion' started by Scarecrow, Oct 14, 2015.

  1. Scarecrow

    Scarecrow Annie Gsampel

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    In view of the - I think it's fair to say - generally not well received news about the new MRC funded study at the CMRC yesterday, what research would you like to see?

    What hasn't had any attention that you think should be a core part of research?

    What needs to be replicated and why?

    Full study design not required, just your rationale.
     
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  2. Sasha

    Sasha Fine, thank you

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    What a BRILLIANT IDEA for a thread. :thumbsup:

    Wouldn't it be great if there was some mechanism for getting patients' wishes for research across to the MRC?

    I want to see a whacking great trial on rituximab or whatever needs doing so that we're not all hanging about still waiting when the Norwegian results are published in 2018.
     
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  3. Sean

    Sean Senior Member

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    We need to nail the basic character of PEM. Big baseline study, using every plausibly relevant test and measure on the books.

    Give researchers a solid basic picture to really start working with.
     
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  4. aimossy

    aimossy Senior Member

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    Julia Newtons work amped up and replicating anything relating to what Fluge and Mella are up to. Something that will actually pin down a solid finding properly that is biomedical in big numbers. I'm fed up with very interesting pilot studies not followed up. We are up against medical dogma and strategically need something that says "well explain the faulty thinking in this part of the body - talk to the hand, you have no way round this".
     
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  5. Bob

    Bob

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    I think that the research plans by Nancy Klimas and her team is under-rated, and not discussed enough by our community, and she's got some great ideas and plans that she can't get funded. So I might give her the $1m. (The OMI and Lipkin are doing top-rate work, but they have got the recognition they deserve.) Or, like Amy, I'd like to fund Julia Newton's work, including her work on muscle abnormalities. Or, like sasha, focus on rituximab. And Sean's suggestion is essential as well.

    Otherwise, I would want to see a comprehensive search for novel autoantibodies, and unusual levels/types of proteins/metabolites. Dr Lipkin is doing the latter, so I'd focus on seeking novel antibodies, or unusual levels of autoantibodies. (Except Jonathan Edwards might tell me that this would be a waste of time?)

    Also I might study the mitochondria, and latent viruses, and HERVS.

    And a detailed study of the immune system.

    $1m isn't enough. I'd need $5m just to get started!
     
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  6. Theodore

    Theodore Senior Member

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    What would be great is to fund it by ourselves since there is between 1 million to 2 millions people or more living with this condition...
     
    Last edited: Oct 14, 2015
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  7. Theodore

    Theodore Senior Member

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    By the way, the Staph Vaccine is working and has a responding rate of 60% approximately but it is no longer on the market because no one wants to fund it (When I say no one I mean Big Pharmas).

    In fact, the problem is the Big Pharmas which are not interested anymore in doing some research because of the drug patent that can last "only" between 10 to 15 years. It's not enough for them that's why the innovation doesn't come from them.

    Biotech companies which most of them are spins off from universities of other public organizations are doing the whole work and sell the drug to the Big Pharmas who are responsible of the sales of the product only. It's a win win.

    These Biotechs can raise millions from private and public investors and that's how they can conduct the research.

    I say we can act like investors, except we do it for us, for a return on health not a return on investment.
     
    Last edited: Oct 14, 2015
  8. alex3619

    alex3619 Senior Member

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    I would ask for a billion. That might be enough and what the research deserves. Rituximab has to be high on the list because its a potential cure. NK cell studies should be high, because they are potentially diagnostic. I would look at expanding the exercise testing, looking to see if something other than a CPET is needed - I suspect we only need gas analysis in severe patients. They are probably living at or over their anaerobic threshold. All the big data studies should get funded, and we need bigger better biobanks. PhD scholarships would also be of benefit. I would like to see studies looking at small biopsies of vascular tissue, trying to isolate the molecular pathology, though I would need to know how this could be done safely. I would do the same for brain tissue but there is this pesky little problem of brain damage. I would expand the search for autoantibodies, and offer huge prizes to the research team that answers each of a number of key questions, such as what causes ME.

    One million barely gets started. I would get a panel of experts together, and have them come up with a list of key findings that have not been replicated. So much of what we think we know has not been replicated. Let's find out how much of that is solid.

    On the issue of Nancy Klimas' research, she has a potential drug therapy that has been sitting there for years. There is not even enough funding for a small pilot study.
     
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  9. Scarecrow

    Scarecrow Annie Gsampel

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    Of course but the point of the budget is to focus in on what is the most vital thing in you opinion.

    I only have £1m to donate (I wish!). Persuade me why I should give it to you.
     
    Bob and Sasha like this.
  10. SOC

    SOC

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    I would fund immune and mitochondrial research on moderate-severe patients only. I'd insist on CPET testing to confirm that patients in the study actually had PEM, given that's the closest test we have for it so far.
    I think this may well be true. The report of my last (submaximal) CPET test said I was hypermetabolic at rest before the test even began, so my guess is that severe patients will show sufficient abnormalities without the 2-day CPET. The 2-day CPET may be necessary to identify mild patients, but possibly not moderate-severe patients. If we can find a way to avoid maximal exertion testing, we could get more severe patients in these studies, which is crucial, imo.

    I think we need to get at the core of PEM. That is the identifying feature of ME. It's not fatigue, so simple fatigue research is not the way to go. The most likely sources of PEM are in the immune system and/or the mitochondria, so that's where the money should go. Once we can identify what makes PWME different from patients with other illnesses, then we can start looking into what might be causing that difference -- pathogens, autoimmunity, cardiac, or neurological abnormality, genetics. Ninety percent of the problem with current (and past) ME research is that it is highly likely that the cohorts of mild patients most researchers study probably contain a very high percentage of non-ME patients, so the results are inconclusive.
     
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  11. MikeJackmin

    MikeJackmin Senior Member

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    A million isn't much.

    There's a problem-solving technique called "working the bottom of the flow chart". You start from your desired outcome, even if it is unlikely, and work back to see what you need to get there. It's a good trick sometimes, especially when you are willing to take a risk and get the most bang for your buck.

    My guess is that the game really changes when we find an easy-to-assess biomarker that not only distinguishes us for healthy people, but from folks with non-CFS fatigue. Easy tests are those that use blood or saliva or urine, and have accepted reference standards, and are generally available. That's a fairly small set, especially after we exclude many tests which we already know will not distinguish us from healthy controls.

    Look at that set, select the best of the lot, and use them to compare us against folks with non-CFS fatigue. You don't need a huge number of people, you can probably find CFS folks who have been pre-screened for other studies, there is no blinding and no followup, so it ought to be reasonably inexpensive. You could run a lot of tests and if you get lucky you'll blow the roof off.
     
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  12. *GG*

    *GG* Senior Member

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    I think Dr. Baraniuks research in the D.C. area is along these lines. FYI

    GG

    PS I am hoping to make it, but think I am only going to attempt this in the spring, and of course I am going to have to have someone to drive with me. I did the CPET test in 2010 already, so perhaps this could be valuable as well? I plan to mention it, if things work out for the study!
     
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  13. Helen

    Helen Senior Member

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    That´s what the Ron Davies´group will do. They will run every available test on each patient that they´ll study.
     
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  14. MEDad

    MEDad

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    I'd go for a phase 3 trial of cyclo in mild to severe ME (ICC) and start recruiting participating centers. As soon as the results from the norwegian phase 2 trial are ready next spring I would try to treat as many as possible. Never mind the exact disease mechanism if we can get a treatment faster. Rituximab as plan B
     
    Last edited: Oct 14, 2015
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  15. Scarecrow

    Scarecrow Annie Gsampel

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    My first thought was that the CMRC might be responsible for that as they sprang from an MRC initiative. However, patients don't get a mention in the CMRC Charter's Aims and Objectives, except in the reference to 'well phenotyped patient cohorts'. So that means any input from patients would need to be made direct to the MRC.

    https://www.mrc.ac.uk/about/how-the-public-can-get-involved/public-involvement/
     
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  16. BurnA

    BurnA Senior Member

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    If we only have 1 million I agree - we need to focus on treatments and not diagnosis. More phase 3 trials of anything and everything !
     
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  17. alex3619

    alex3619 Senior Member

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    Better diagnosis means better research and better identification of potential responders. Problems with phase 3 trials are one of the biggest reasons drugs that work are not approved. If too many do not respond, and nobody knows why, or there are too many side effects, then bureaucracy creates road blocks. If on the other hand we can remove people who have something different wrong with them, then the findings will be more focused and have fewer failures. So most drugs will have a higher success rate, and a higher chance of being approved.

    We definitely need more drugs tested though. There are quite a few now that the theories suggest might work.
     
    SOC likes this.
  18. Antares in NYC

    Antares in NYC Senior Member

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    Well, I would use that money to launch a major PR campaign on all media about the realities of ME/CFS, and start a major fundraising effort targeting potential donors with deep pockets, so we can turn that million into 1 billion. The PR and goodvertising effort should also shame the governments of major industrialized nations (and shame them hard!) for neglecting research funding and treatments for the disease. The goal would be to change the public perception of the disease and make the public demand that governments properly fund research for this devastating illness.
    That would be my take.
     
    Last edited: Oct 14, 2015
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  19. BurnA

    BurnA Senior Member

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    Great idea ! Here's hoping that the first few RTX responders ( when it's available ) can help shame the governments and some factions of the medical community too.
     
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  20. BurnA

    BurnA Senior Member

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    I agree. It's just with 1m to spend I'd take my chances on a treatment trial ( well designed obviously :) ) The bigger picture is diagnosis/biomarker no doubt but I think I'd need more than 1m to make a dent in that field.
     

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