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Updated NIH reponse to CFSAC recommendations

Discussion in 'General ME/CFS News' started by viggster, Mar 29, 2016.

  1. viggster

    viggster Senior Member

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    Highlights:

    Confirms that RFA's (PLURAL) are coming soon:

    "Revised NIH Response: The Trans-NIH ME/CFS Working Group is in the final stages of putting together a comprehensive research strategy for ME/CFS research that will include new RFAs. Clearly, as stated in the initial response, biomarker research will be critical to understanding the underlying causes and mechanisms of disease in ME/CFS. Until the new initiatives are in place, investigators can submit applications for peer review in response to any of the standing NIH grant mechanisms."

    And the NIH is working to develop the back-end for a patient database that could be used by all researchers:

    "Revised NIH Response: Working with the CDC and the ME/CFS clinical, research and patient community, NIH intends to develop Common Data Elements (CDEs) that will then be utilized for all clinical studies and clinical trials. In conjunction, a Data Coordinating Center (DCC) that would provide a standardized platform for storing and sharing data from clinical studies and clinical trials would significantly improve the ability to compare results across studies. The Trans-NIH ME/CFS Working Group is exploring the cost and feasibility of a DCC as part of the research strategy moving forward."

    http://www.hhs.gov/advcomcfs/recommendations/08182015-agency-responses.html#
     
    Last edited: Mar 29, 2016
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  2. Justin30

    Justin30 Senior Member

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    POINT 3: Advance Treatments and Therapeutics:

    This response by the NIH is not acceptable in my mind. The funding of and starting of both Rutuximab and Ampligen studies should commence immediately. They should be highlighting and tasking key experts Drs. To assist them in the process. Both OMI and Simmaron could be used. I admittedly dont know how this whole process takes place but should be of top "PRIORITY" Getting a serious drug like Rutuximab approved will get the whole world looking ME in a new light, that light is the disease is Serious, life-altering and fatal!

    As for Rutuximab: NIH should be contacting Fluge and Mella getting as much info as possible and starting clinical trials ASAP. NIH should be contacting and assisting reasearchers and Dr. to start phase 3 trials immediately for FDA approval.

    Quite frankly it is a joke to have to rely on a substantially smaller country like Norway to get things moving...I commend Norway and the Drs working their just rediculous to see the US drag their feet. Step up NIH and Help! (No offence to Norway, please dont take this the wrong way).

    As for Ampligen: they should review the Patterson Subset review his biobank and biomarkers and select patients as soon as the NIH starts their study. I know this is FDA stuff. But the NIH should be pushing hard for this.

    I choose not to get barried in the NIH beet around the bush type attitude; we'll do it in the future. GET something going right away!

    I Want definitive responses with regard to treatment based on what is avaialbe in the literature now!

    NIH has smart people look at the studies select some drugs ex:

    Select all cohorts from ME/CFS Clinics that meet CCC/ICC Criteria only.

    Test:
    - low dose IVIG
    - Test Cychlophosphamide
    - test bellubimab less strong version of rutuximab
    - there are so many that have proven to work in small cohorts that need to be reviewed.
     
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  3. aimossy

    aimossy Senior Member

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    Nice to see more formal confirmation! Thanks for highlighting.
     
    Simon likes this.
  4. BurnA

    BurnA Senior Member

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    Its good to read, i'll take anything positive out of it that i can. However i feel some of the replys are sidestepping the questions.

    7. Provide Research Funding Commensurate with the Burden of Disease: To facilitate the above goals, CFSAC recommends that the Secretary work with HHS agencies to ensure that total research funding is commensurate with the epidemiologic prevalence and economic burden imposed by this disease. Based on disease prevalence, equitable funding is estimated to be $250,000,000 per year.

    CDC Response: CDC has established cooperative agreements to collaborate with external researchers as well as supported an intramural program using appropriated funds. Currently nearly half of CFS funds coming to CDC are used to support extramural programs. One such example is the ongoing Multi-site Clinical Assessment of CFS study that involves 7 ME/CFS clinics across the United States.

    AHRQ response: Consistent with its mission, AHRQ will welcome grant proposals as they relate to the dissemination and implementation of patient centered outcomes research related to ME/CFS.

    NIH Response: The NIH recognizes that ME/CFS is a chronic condition that imposes significant limitations and health concerns for the individuals with the disease. Public health needs are a critical factor in NIH’s funding decisions, but scientific merit, portfolio balance, and budgetary impact are also important considerations. Often, the funding level is driven by the number of high quality applications that are received. NIH encourages the ME/CFS research community to submit grant applications utilizing new and innovative approaches to study the disease.

    Revised NIH Response: The goal of the Trans-NIH ME/CFS Working Group is to stimulate new research strategies in order to increase funding for ME/CFS, increase training of young investigators to do research and provide clinical care for individuals with ME/CFS, and attract and interest new investigators from other research areas to do research on ME/CFS.
     
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  5. Justin30

    Justin30 Senior Member

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    Why doesnt the NIH just say and "WE HAVE DECIDED TO SUPPORT RON DAVIS AND HIS NOBEL LEAREAUTES AND ASSIST THEM IN FINDING SEVERELY DISABLED PATIENTS AS WELL AS FUND THE REST OF THE OMF STUDY"

    Or did I miss this....

    For some reason I feel that these severly ill will showcase the degree of disfunction and damage from the disease.

    There are many hear on PR that fit the Bill its time to send out Drs to test them!!!!!

    This goes for housebound patients as well!!!
     
  6. Sasha

    Sasha Fine, thank you

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    Valentijn likes this.
  7. viggster

    viggster Senior Member

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    So do you want a treatment trial to start immediately, or do you want a structure where patients and ME experts can help design the study? You can have one or the other. I don't think you can have both.
     
  8. jimells

    jimells Senior Member

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    Well for one thing they would have to get the approval of 23 different institutes. :bang-head:
    This is bureaucratic bungling by design. So exactly how many people will have to "review and approve" everything touched by the Trans-NIH Group? Hundreds? Thousands? This is the perfect design to make sure that authority and responsibility is so widely diffused that no one will be responsible for anything,

    I remember reading someplace that the chair of the group will rotate among the different institutes, just in case someone in the world doesn't understand that no one in NIH will touch this program with a ten foot pole, except under duress.
     
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  9. BurnA

    BurnA Senior Member

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    This is a recipe for disaster. What is someone going to focus on, their tasks that they will have to be accountable for next year, the year after and so on, or the tasks that nobody will even remember that they were accountable for for 6 months. ?
     
  10. jimells

    jimells Senior Member

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    Exactly. And it is By. Design. I'm nobody special and it is obvious to me. Does anyone seriously believe that the professional bureaucrats who clawed their way to the top of NIH can't see it as well?
     
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  11. Justin30

    Justin30 Senior Member

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    Well I would actually prefer if they would would start simultaneously. I believe there to be enough preliminary data to get phase 3 trials up and running.

    Ampligen is an immune modulator and antiviral-with ME Expert Experience

    Rutuximab a B Cell Depleter-with ME Expert Experience

    Than there is the NIH Study. Which I feel if They were to study the patients with responses and work backwards as well as forwards through the borage of testing we will get to a cause of the dysfunction much sooner.

    I would like to see both and dont see this as unrealistic to ask for both.....considering the amount of suffering....latest stats I bet would be around (1.5mill sick 15% are severe) = 225,000 people suffering in dark rooms or stuck inside....and I bet you the stats are much higher in the US considering in Canada as of 2011 450,000 had been diagnosed. Im not even going to memtion population differences.

    @viggster we have so much suffering with this disease that treatments need to be made available sooner than later...people have waited 30 or more years for treatment. We need one!

    I believe by having 1 serious treatment will spark the interest of researchers to get this thing moving.

    I think having patients and ME experts design the study is given considering that they really hold the key to the answers in their body and brains.

    So I cant chose one or the other...but we will be dealt what we are dealt unless one or more of the organizations pick it up and do something with it.
     
    NL93 likes this.
  12. searcher

    searcher

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    Hi @Justin30 - Although I have stepped back from daily work, I can promise you that organizations are speaking with NIH about the urgent need for treatment trials. As I am sure you know, NIH said they were looking into doing Ampligen and Rituximab trials-- http://www.meaction.net/2015/12/21/nih-considering-ampligen-and-rituximab-trials/-- and have been speaking with folks in Norway and experts in the US. Unfortunately NIH does not often do Phase III trials (that is usually the responsibility of drug companies) but it's my understanding that they were trying to find ways to make trials happen, whether at NIH or externally. Hemispherx issued a recent press release recently saying that they are speaking with the NIH about Ampligen--- https://globenewswire.com/news-rele...-Neurological-Disorders-and-Stroke-NINDS.html
     
    Last edited: Mar 30, 2016
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  13. Justin30

    Justin30 Senior Member

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    @jimells and @BurnA make serious points about the working group. The NIH have used this tactic before....A Working Group...potentially flawed by design...as it has been in the past...

    The problem is even in their revised response to CFSAC they keep pushing things off to future dates....accept the study.....

    They want every Center at NIH to have a crack at it before they asign it to somewhere...its rediculous...if you ask me....more ACTION less talking!

    Its infectious (usually) = Infectious disease
    It is Neurological = Neurology
    It is Immune = Immunology (Inate Immune System)

    The stupidest thing I just cant get over about this NIH study is why is there not a huge component dealing with Dyautonomia, blood volume, tilt tables, biopsyies for SFN....Why have none of the organizations brought this up????? This could explain most of it....just like the overlapp and stats related to POTS.

    Why is their not a large Rhemetological compoment to this???? Rutuximab is working for 2/3s of patients....strange...this is why I said clinical trials at the same time as the NIH study cause you can work both angles front to back and back to front.

    If I had to put money on it it would be that an infection, toxic overload, stress response wacks the Autonomic, Peripheral and Central Nervous systems which would =

    Disturbed Cognition
    OI
    Exercise Intolerance
    Endocrine Dysfunction
    GI problems both Dybiosis and Motility issues
    Muscoloskeletal pain

    The reason we cant figure it out is because we the human race knows but a fraction about how the brain and CNS work......

    MS, Alzhiemers, Dementia, SCA types, etc havent been solved!

    This goes for Immunological/Rheumatological illnesses as well

    Lupus, RA, Sjorgens, Sarcardoises

    They have treatments but they are still not curable

    What we need is treatments at this point because of the debilitating nature along with more research....

    We have the most devasting disease on the planet in its severe form. Inrelenting suffering that wont kill you for year after year. Not having or taking a risk ro feel a bit better is inhumane.
     
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  14. Justin30

    Justin30 Senior Member

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    @searcher I have read this I know in governments things move slowly. I did my Univeraity Practicum at a health authority. After engaging in that endevour I decided to pursue other avenues of business.

    I guess what I would like to hear the NIH say is that with regard to clinical trials that they make a promise.

    Something along the lines that we at the NIH will engage the pharmeceutical companies to start phase 3 clinical trials within the next year.

    This is a long shot but get an open commitment. Their response are very laxed and open for to much speculation.

    This is why you see so much unrest amongst the community.

    Here is a good one:

    We at the NIH agree to supporting a yearly funding increase of $250,000,000 to match the burden of the disease. We at the NIH will use this funding to conduct internal research on ME/CFS, support clinical trials, develop centers of excellence in universities around the country, provide RFA funds and support financialy all other ongoing studies of ME/CFS.

    Im sure you could at more to this but you get it......Its a COMMITMENT.
     
  15. jimells

    jimells Senior Member

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    Is that by federal law or by tradition? If they don't know how to organize one they could probably get a few pointers from Professor Edwards or Dr Fluge or Dr Mella.

    I've been wondering if there has ever been any calls for a study at the Clinical Center and I stumbled over this in the CFSAC recommendations

    So here is a recommendation from CFSAC to use the Clinical Center for treatment trials. I assume Rituximab would fit with this recommendation. And the NIH answer to this is: NO.
     
  16. searcher

    searcher

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    I don't think it is due to law, but primarily due to cost, complexity, and tradition. A Phase III trial is incredibly large and expensive, and I doubt could be run solely out of the Clinical Center due to the number of people who need to participate. I think they are usually run out of multiple locations and take years. As you probably know, the basic science for drug development is often funded by NIH, but bringing a drug to market is almost always funded by drug companies (who also reap the rewards.) Rituximab is going off-patent (if it isn't already), so there is no incentive for Roche/Genentech to fund a Phase III trial.

    A Phase II trial for Rituximab may make more sense as I believe it would require less patients, would be faster, and at least will prove safety and effectiveness for patients, but the problem is that I don't think Rituximab passing Phase II for ME would force insurance companies to pay. If NIH is involved in a clinical trial someone told me that NeuroNext (https://www.neuronext.org/) could help-- they are currently helping with a Phase II trial on Rituximab for MG-- but I think they still require external researchers/drug companies to participate.

    Ampligen appears to be a different story since Hemispherx has strong financial reasons to try to get it approved.

    One other issue is that our clinical experts are already stretched incredibly thin so are not able to participate in all the initiatives on which we need their expertise. We desperately need Centers of Excellence and many more trained experts.
     
    Last edited: Mar 30, 2016
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  17. Justin30

    Justin30 Senior Member

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    Ill take phase II trials but they really need to engage in something and publically state it.
     
  18. BurnA

    BurnA Senior Member

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    I don't think phase II would be worth anything and perhaps might be a waste of money. In order to get approval phase III multicentre trials are generally the norm. It doesn't really matter if it's called phase II or III, the cost is in the large no. of patients at multi sites which requires a lot of administration.

    Phase II is a like a stepping stone to phase III but as that work has been completed by Fluge and Mella there would be no real value in repeating it, from what I can see.

    In theory there is nothing to stop the FDA from approving RTX based on a successful Norwegian trial but I don't know how likely this is.
     
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  19. dancingstarheart

    dancingstarheart

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    Here is an idea to get around the excuse of loss of patent for Rituxumab, that Pharma won't risk funding a costly phase 3 trial.

    How about the brand new fully humanized version of Rituximab? It basically works same way as Rituximab. So it should have less side effects since it is fully humanized.

    "Obinutuzumab is a fully humanized monoclonal antibody that binds to an epitope on CD20 that partially overlaps with the epitope recognized by rituximab.[2]"

    "GlycArt's technology platform allowed control of protein glycosylation; the cells in which obinutuzumab is produced were engineered to overexpress two glycosylation enzymes, MGAT3 and Golgi mannosidase 2, which reduce the amount of fucose attached to the antibody, which in turn increases the antibody's ability to activate natural killer cells.[5][6]"

    https://en.m.wikipedia.org/wiki/Obinutuzumab

    Note: increases anti body's ability to activate Natural Killer cells. The very thing that many with ME have problems with.

    Pharma would be more likely pay for trials on this. It is already currently approved for CLL and as backup to follicular lymphoma.

    My oncologist had told be to there was hope for new treatments once Rituximab stops working as treatment for me. He said obinutuzimab was hopeful.

    Unfortunately my ME got worse, but I have been sick with it for 27 years. I am all for research being done on this. Norway has already made headway in finding immune abnormalities with subset that responded well to Rituximab.

    There are many advantages to doing Rituximab and obinutuzimab testing.
     
    Justin30 likes this.
  20. dancingstarheart

    dancingstarheart

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    Another possibility is NIH extramural funding Rituximab trials in Canada. NIH could also help fund Norways or UKs trials, because NIH has been known to fund international research. Like the one HIV research done in China that Senator Bill Cassidy mentioned at the Fall appropriations meeting with Collins/NIH.
     
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