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No Longer Naive in the Ways of The Beast
After having lived for years with ME/CFS, Jody Smith learned there's more to this beast of an illness than she realized, and that what might help one person may not help others ...
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Experts Reflect on the FDA Stakeholder Meeting

Discussion in 'Phoenix Rising Articles' started by Mark, May 19, 2013.

  1. Phoenix Rising Team

    Phoenix Rising Team

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    View the Post on the Blog

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  2. Simon

    Simon

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    Thanks, Joel, a really interesting piece and it pretty clear now that the FDA workshop was a landmark event.

    From the bits of the workshop I've watched, it does seem that the FDA has really listened to patients and researchers, doe now 'get' ME/CFS, and is serious about raising its game. A few random highlights for me from this piece:
    • The patient session was the real game changer, explaining the devastating impact of this illness
    • Big Decision makers were there: Both NIH Director Francis Collins and FDA CDER (Center for Drug Evaluation & Research) Director Janet Woodcock [Bob Miller]
    • Willingness of the FDA to engage with researchers/Pharma at an early stage to ensure clinical trials are set up right
    Bob likes this.
  3. Bob

    Bob

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    Thanks very much for these interviews, Joel. It's very interesting to have some post-conference thoughts from the people who attended.

    Something caught my eye that I haven't considered before:
    It might be the case that there are different biomarker profilies (e.g. cytokines, epigenetics, etc) when the condition is improving or deteriorating (waxing and waning.) It seems to make intuitive sense that 'improving' and 'deteriorating' would involve slightly different biological processes, with slightly different biomarkers.

    I think this is something that researchers need to factor into their studies, to maximise the clarity of the results.
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  4. Nielk

    Nielk

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    Thank you, Joel for these interviews.

    It's interesting to see the perspectives of the interviewees. They all seem to think that it was a positive development.

    I like what Dr. Enlander described as a friendly cooperation between major researchers/clinicians. This will definitely have a positive impact.

    Enlander stated:

    "Then there was the direct contact with the FDA principals including Admiral Sandy...and the invitation to submit new ideas by telephone or mail regarding potential new substances, was very interesting and helpful. This included instructions for new INDs (Investigational New Drug Applications)."

    I hope that researchers will take the opportunity to do so.

    Klimas answered:

    What, for you, was the highlight of the event?
    "Certainly the first day, when the patients described the severity and nature of the illness seemed to be heard well by the FDA leadership present. One CRO from a pharmaceutical company told me she thought it the best and most important part of the program."

    For those who are not convinced that it is worthwhile to give personal testimony, this should reinforce the idea that we need to be heard. The more people hear about our personal stories, the more impact we will have in how the world understands our plights.
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  5. snowathlete

    snowathlete

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    Glad you and others found it interesting. I did too!

    I agree that the waxing waning is likely important. Add that to the confections and different disease stages and its no wonder findings often conflict.
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  6. snowathlete

    snowathlete

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    Thanks Nielk. I agree that personal testimony is oh so important. I believe the more well we voice our plight, the more we will eventually get through - we need to take our opportunities!
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  7. Simon

    Simon

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    Don't know about waxing/waning - perhaps a more dramatic difference is good day/bad day esp if bad days are PEM days: Nancy Klimas has a study underway looking at immune markers that also measures whether sample were gathered on good or bad days. I'll be very interested to see the results from that work.
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  8. Nielk

    Nielk

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    I guess this needs to be investigated but, I don't believe that the waxing and waning is unique to ME/CFS. Many chronic illnesses have this variability. For example, I suffered from active Crohn's disease for over 15 years. My condition throughout those years definitely waxed and waned. What was really interesting (and fortunate for me) was that each time I was pregnant, I was in remission of the disease. The biomarker though - inflammation of the ileum is always there, even now that I seem to be in a long remission.
    Simon likes this.
  9. Valentijn

    Valentijn Activity Level: 3

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    I think autoimmune diseases especially do this.

    My fiance came down with Type I Diabetes a year or so ago, and even though damage to insulin-producing cells is supposed to be permanent, his need to use insulin waxes and wanes, while his diet remains the same.
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  10. Marco

    Marco Old blackguard

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    FWIW Bob fluctuating cytokine levels are already known to be associated with various 'state' symptoms in 'psychiatric' diseases such as the manic and depressive phases of bipolar disorder or psychotic episodes in schizophrenia.

    No reason a similar picture won't be found with ME/CFS.
  11. Bob

    Bob

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    I agree: I wasn't suggesting that fluctuations were unique to ME. I meant that perhaps ME's unique biomarker profile might change slightly as the illness is improving or deteriorating. So there might be a 'waxing' biomarker subset, and a 'waning' biomarker subset. As Mary Dimmock pointed out, it's another variable that could/should be factored into biomarker research.
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  12. Bob

    Bob

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    Thanks Marco, I wasn't aware of that. It's not entirely surprising, but it is interesting.

    I think there has already been some research which shows cytokine profile differences before and after exercise, in ME patients, or am I imagining that?!
  13. Sasha

    Sasha Fine, thank you

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    Great job, Joel - I think this is one of the most interesting articles I've ever read on Phoenix Rising.

    It's one thing to watch a meeting - I watched most of it - and quite another to get the informed viewpoint of people with special knowledge and the inside track. Fascinating.
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  14. snowathlete

    snowathlete

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    Thanks Sasha. As most of what is writen came from the people who took part, it was really interesting for me to, as a reader. I agree it's fascinating to get this angle on it. If people like it then hopefully we will do it again following other conferences/meetings etc.
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  15. Marco

    Marco Old blackguard

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    If I recall correctly there was a paper by Broderick, Fletcher and Klimas etc (probably not in that order) that carried out a 'network' analysis of cytokines in ME/CFS and cancer related fatigue I think and noted that differences between ME/CFS and controls only became apparent after an exercise challenge. Hope I got that right.

    Sorry for the slight hi-jack Joel. It good to get feedback from some of the participants and good also that the general feeling was positive which was what I felt after reading the transcripts. It did appear to me that the FDA folks were genuinely interested in better understanding ME/CFS.
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  16. Shell

    Shell Senior Member

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    My son was warned that for the first year to 18mths after dx things can be very rocky. It was explained that once he started using insulin, his pancreas would "rest" and then try and then would fire up occasionally until it finally died. This was certainly true for my son. He had some awful hypos in the early days, but now he has a better hold on things because there's no "extra" insulin being thrown in. Doing the DAFNE course has helped as well. Basically he weighs his carbs and injects accordingly.
    Hope your fiance gets to the stable point soon.
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  17. Shell

    Shell Senior Member

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    I do hope the positive feedback means the FDA will move towards taking this seriously. If ME is autoimmine for example we know already that auto diseases are on the increase quite rapidly. One little thought I have on this is, they know auto diseases are on the increase. Let's take type 1 diabetes for example. There is a push to stop type 1s having the test strips and even the insulin they need because of cost. It's not just here in the UK that the pressure is on. Channel 4 among other joined in the "type 1s are draining the NHS, let's go back to pig insulin" campaign.
    As ME is already a dismissed disease; that has got to be a cheaper option for Govts, so I wonder, even if the FDA do start to change tack whether they will crash into the politcal wall?
  18. alex3619

    alex3619 Senior Member

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    Exercise challenge seems to induce the differences in CFS, and immune challenge in fibromyalgia. Its less clear with ME, it may involve both.
  19. alex3619

    alex3619 Senior Member

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    Cost benefit issues at treating ME have been a bugbear since the 1990s. Its part of the appeal of BPS approaches. It depends on the treatment. A cure will probably be funded even if its moderately expensive. An expensive ongoing treatment will probably not be funded, even by most private insurance. Everything in between will be up to advocates to fight for.
  20. jimells

    jimells Senior Member

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    Another great article, this definitely belongs on our 'front page'. The unofficial meetings are where the action is, and not just in ME/CFS; it is encouraging to read of them.

    I am dismayed that after decades of misery our government leaders are just now bothering to find out how severe our illness really is. They didn't need a two-day meeting to find this out, just an hour or two of reading the literature or even a phone call to one of our esteemed clinicians should've been enough.
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