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FDA Workshop on Drug Development for CFS and ME

Discussion in 'General ME/CFS News' started by Ember, Feb 21, 2013.

  1. Nielk

    Nielk

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  2. Ember

    Ember Senior Member

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  3. Sasha

    Sasha Fine, thank you

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    Their site is still under construction:

    http://naame.org/
    Ember likes this.
  4. jspotila

    jspotila Senior Member

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    NAAME is what advocates have been referring to as the Alliance over the last year. It's not yet a 501c3, but is made up of many independent advocates as well as organizations. Mary Dimmock has been leading the charge, and she coordinated the letter to Secretary Sibelius last year, and the letter to the FDA before this meeting about case definition. I'm involved, although I certainly don't speak for the group. But people have been working very hard on CFSAC, FDA, legislative and CDC fronts.
  5. Sasha

    Sasha Fine, thank you

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    Sounds like a great initiative - I think we need some coordination.

    I'm glad you're involved!
  6. Ember

    Ember Senior Member

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    What's the relationship between the construction of the NAAME website and the disabling of the Coalition's website?
  7. David Egan

    David Egan Hermes33

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    The importance of using what biomarkers we have was stated by Nancy Klimas at the FDA meeting. The vital importance of biomarkers is shown by their use in clinical drug trials which seek FDA approval. Biomarkers have been the means by which new drugs and new treatments have been developed for many decades. For example the use of Canadian Consensus Criteria (2003,2011) enables researchers to recruit people who actually have ME/CFS, not some other illness. The identification of specific biomarkers via intensive diagnostic tests enables researchers to establish a starting point for patients in clinical trials. As the drug trials progress, these biomarkers will move up or down or remain stable depending on patient responses to a drug. Further testing for biomarkers during the trials and at the end of the trials provide an end marker to objectively assess the effectiveness of a drug. This can be supplemented by Canadian Criteria and questionnaires to provide further supporting evidence. It is biomarkers which lie at the heart of clinical trials and drug development.

    Strangely, biomarkers were not used in the Ampligen trials and trials of other drugs. The FDA continues to bury its head in the sand and falsely claim there are no biomarkers and Unger's report claims similarly. This line of reasoning is false and needs to be challenged. I have researched many, many research papers and presented the following to the FDA to clear up this matter and state on record that there are biomarkers for the biological abnormalities and dysfunctions in ME/CFS. I have presented my findings on www.cfs-ireland.com/structure.htm#7
    and the following email and letter to FDA meeting corroborates this.

    " Dear Mrs. XXXXXXXXX
    Firstly I would like to thank the FDA for giving us ME/CFS patients an opportunity to express our views about ME/CFS and it's diagnosis, treatment, and management. This will hopefully bring some very important medical and scientific findings to the forefront, and facilitate medical drug deployment to address the biological abnormalities and dysfunctions found in ME/CFS.

    While patients and doctors will vary on what works in ME/CFS, one thing is consistently very clear, only a very though diagnosis of the biological abnormalities, dysfunctions and infections found in an ME/CFS patient and the treatment of these will bring about any improvement or recovery. It is the poor quality of diagnostics which is holding back effective medical treatments, improvements and recoveries. This is due in large part to governments and states not investing in new diagnostic technologies and not subcontracting out high-tech diagnosis to the private sector, which is currently ahead in the field of diagnostics for

    ME/CFS patients. This failure to thoroughly diagnose ME/CFS, is also is undermining the measurable efficacy of existing drugs, as some patients respond to drugs like Ampligen, Naltrexone, Rituximab, etc. and others do not. This is explained by the existence of subgroups which suggests significant differences between groups of patients. Thus the need for a very thorough diagnosis and an identification of all biological abnormalities,

    dysfunctions and infections present in a patient order to (i) establish the subgroup (ii) the treatment methods to be used (iii) the biological markers to track in order to measure progress or lack of it. This should also be undertaken before, during and after all research trials so as to have objective evidence tracking the progress or lack of progress of certain treatments. Most research into ME/CFS has not used biomarkers for the abnormalities, dysfunctions and infections found in ME/CFS patients, and did not use Canadian Criteria (2003, 2011) but used subjective questionnaires and outdated, subjective and conflicting diagnostic criteria which led to people with other illnesses being recruited, and produced confusing, contradictory and ineffective results. This has frustrated the efforts of the FDA, CDC, NIH and many drug companies for several years.
    I ask the FDA to consider liasing with other government bodies including the CDC, the NIH and the US Department of Health with the objective of establishing a new system for
    (i) diagnosing ME/CFS. The Canadian Criteria (2003, 2011)
    (ii) measuring biomarkers for the abnormalities, dysfunctions and infections found in ME/CFS patients. Some ideas are presented here www.cfs-ireland.com/structure.htm
    (iii) treating these abnormalities, dysfunctions and infections. Some ideas are presented here www.cfs-ireland.com/structure.htm
    (iv) tracking progress in doctor / GP settings and in research trials. Some ideas are presented here www.cfs-ireland.com/structure.htm http://www.cfs-ireland.com/prioritisation.htm
    (v) standardisation of research methods and critera for ME/CFS across America and the world, so that replication of studies is more precise, consistent and accurate
    (vi) stating what constitutes improvement and what constitutes full recovery in objective scientific terms
    (vii) a biological database tracking all stages of recovery for ME/CFS using biomarkers and objective scientific evidence
    (viii) The FDA could work with Biovista’s ME/CFS progam ( http://biovista.com ) in the USA. Biovista is engaged in research and data mining of thousands of medical drugs (including some which are currently out of use) and finding matches between them and the symptoms, abnormalities and dysfunctions found in ME / CFS. They are compiling a list of these medical drugs with the objective of having these dugs re-purposed for ME/CFS treatment.
    Some of these ideas, and scientific and medical facts are discussed on the Cross-border ME clinic proposal at http://www.cfs-ireland.com
    This new system, numbered (i) to (viii) above would impart greater precision to drug development, drug re-purposing and drug use in ME/CFS patients. This would enable the FDA and other Federal bodies to make swift and effective progress in relation to ME/CFS. This would benefit ME/CFS patients in the USA and over time, by diffusion of new standards, criteria and knowledge, benefit ME/CFS patients all over the world.
    I have registered to attend the FDA meeting via webcast.
    Thanks again for your efforts on behalf of ME/CFS patients and their families.
    Best Regards,
    David Egan."
  8. jspotila

    jspotila Senior Member

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    Absolutely none that I am aware of.
  9. Ember

    Ember Senior Member

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    Congratulations to Jennie on her appointment to the FDA’s Patient Representative Program!:balloons:
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  10. Sasha

    Sasha Fine, thank you

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    jspotila, Nielk and Bob like this.
  11. jspotila

    jspotila Senior Member

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    WillowJ, Sasha and Nielk like this.
  12. Ember

    Ember Senior Member

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    Two days after the FDA workshop, Mindy Kitei wrote, “While patients had only two minutes to talk, FDA officials waxed lyrical about the lovely baby grand piano in the hallway, should any patient care to play.” Mindy's two minutes have been posted now on the CFS Patient Advocate blog:

  13. Bob

    Bob

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    I appreciated Mindy's contribution, but I don't agree with this assessment of the event. Patients were being consulted during the entire conference, and had many opportunities to contribute in relation to specific questions, such as symptomatic experiences, and treatment experiences. Also, everyone has the opportunity to contribute via a docket. I've noticed that they even accept video contributions. We don't yet know what the outcome of the conference will be, but I can't fault their attitude so far.
    jspotila, Blue, Valentijn and 2 others like this.
  14. Nielk

    Nielk

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    I agree with Bob. Although it's true that the first two minutes of testimony were pretty short, there were plenty of opportunities in the discussion part to add as much as one wanted. I feel that we are so used to being abused by government and officials, that we automatically complain that, they are dismissive of us and they don't really want to hear us. I don't feel that this was the case here at the FDA.
    jspotila and snowathlete like this.
  15. snowathlete

    snowathlete

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    I expect many of us had the same initial thought regarding the piano comment, but at the same time it's important to remember that the FDA representatives are only human, and when you're the person giving the opening talk at a large meeting like that it is pretty easy to say something pointless and unthinking like that. It can be pretty nerve-racking and there is a temptation to try and break the ice with a comment like that.

    I think we have to see the meeting for what it was, and still is: an opportunity to be heard.
  16. Ember

    Ember Senior Member

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    I struggle to watch the the FDA videos because I can't abide the spectacle of government departments “collaborating,” playing footsie with the CDC case definition. A decade after the publication of the CCC, patient groups petitioned the FDA to separate ME from CFS, but the FDA failed to acknowledge any difference save a difference in the name.

    The CDC case definition has been destroying lives for two decades. What's happening to patients every day is nothing short of criminal.

    Like Nielk, I have painful memories. When I was diagnosed with CFS, I complained of intermittent paralysis in my leg muscles when I jogged. I was encouraged to exercise. I described a new startle response that flooded me with “electric” sensations. No doctor ever commented. I asked why minimal stress was precipitating an escalating response that I couldn't shut off. I've never heard that symptom better described than by Howard Bloom last week.

    I'm mostly bedbound now. I couldn't care for my mother who exhibited ME symptoms but wasn't diagnosed because of her age. Her caregivers didn't change their bathing practices despite her cries of protest. In the end, she refused to eat. These things didn't need to happen. And they're happening all over the world. It makes me want to weep, and sometimes I do.
    mezombie and allyb like this.
  17. Bob

    Bob

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    Ember, I do understand where you are coming from. Many of us have had prolonged distressing personal experiences. I'm sorry to hear about your mother's experiences. It sounds like a horrible situation that you were both forced into.

    And what went on under Reeves at the CDC, and what goes on in the UK, is profoundly unacceptable, to say the least. And their actions have had a profound impact on the way we have been treated.

    I watched the FDA videos with an open mind, but not really expecting to see or hear anything new.
    But what I personally sensed was a willingness to engage, to listen, to hear, and to take us seriously.
    All the time, symptoms were talked about in medical terms, and not in psychiatric terms.

    Perhaps I am being too optimistic, or naive.
    I think it's too early to tell where this new venture by the FDA will lead.

    One thing that I noticed is that the FDA program, and the CDC program, are being led by women.
    It seems to be an all-woman team at the FDA.
    Perhaps this will lead us away from the old boys networks that perhaps inhibited the field in the past.
    And perhaps it will lead us away from ego-led policy.
    The theme seemed to be collaboration, openness, and engagement.
    And the officials came across (to me) as professional, empathetic, and sincere.

    They talked about case definitions, and my interpretation was that it was widely agreed that research could be carried out with any case definitions, and any subsets, using any biomarkers, and using any endpoint measures. The attitude seemed to be one of getting answers, and not sticking rigidly to any preconceived ideas or expectations, or to any past rules or formulae.

    The FDA officials asked the ME research community to come to them with their research proposals, and they would help them design them, and implement them. They said that help and funding was available.

    Also, the CDC is currently carrying out research with regards to diagnostic criteria, definitions and subsets, using the well-known US clinicians.
    Perhaps something will come of that. It seems to be a serious attempt to understand and define the illness, including subsets. It's three decades too late, but at least they seem to be making a start now.

    I'm not telling anyone how they should feel about the FDA conference, or the CDC.
    I understand why people feel cynical, and we have been repeatedly disappointed, and let down, over decades.

    It's just that I personally sensed change at this conference.

    Also, on the private research front, there is en enormous amount of work taking place at the moment.
    Perhaps it will be some years before we see any major fruits from these developments, but I do sense that a profound change is taking place.

    Even in the UK, things seems to changing. Although the new UK research collaborative got off to an unfortunate public-relations start, it builds on past work carried out by Prof Stephen Holgate at the MRC, in which research funding has been committed to serious biomedical research. Stephen Holgate (who chairs the UK research collaborative, as I understand it) is a serious figure, who has already proved his sincerity and his credentials.

    Anyway, this is just my take on it. I do feel that there is reason to be optimistic at the moment. But I acknowledge that we've had many false starts in the past. And it may still take years before any of this comes to fruition or provides answer for us. And perhaps I'm being naive.
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  18. Nielk

    Nielk

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    Ember - I'm so sorry about your mother. I feel your pain and frustration. I wish I could undo it.
  19. Ember

    Ember Senior Member

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    You hit a raw nerve in me, Bob. The ICC was published almost two years ago, and Mindy, bless her heart, spoke of the ongoing carnage. Where is the urgency, Llewellyn King asks: “The hell I write about...is here and now. It is the hell of those who live with a disease that is incurable, has inadequate therapies, indifferent government attitudes, social stigma and no strong public voice.”

    Thanks, both, for your kind words. Thanks too, Bob, for your faith in the fairer sex. After the Ottawa conference, Dr. Carruthers described Dr. Unger as a pleasant woman. (I think those were his words.) I wasn't impressed. I haven't found in myself yet the requisite compassion: “Father, forgive them for they know not what they do.”

    Mr. King closes his recent ME/CFS Alert with congratulations: “I want to congratulate you for your optimism. We need some optimism in this business.” I sometimes wonder, though, if we aren't "so used to being abused by government and officials" that we're prepared to settle for far too little. Almost two decades ago, Dr. Peterson observed, “If I had a disease that was totally disabling, I would be much more angry and aggressive than most of the patients I know.”
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  20. Ember

    Ember Senior Member

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    So do I, Nielk. So do I.

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