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Fluge & Mella's pre-trial study highlights life-changing potential of rituximab

Discussion in 'Phoenix Rising Articles' started by Sasha, Jul 1, 2015.

  1. Sasha

    Sasha Fine, thank you

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    This is a massive surprise to me - in all these years on PR that people have been discussing rtx, I hadn't been aware that this was even a theoretical possibility in the UK. I don't think I've ever seen anyone mention it.

    That's promising.

    In some trials, data are so clear about the benefit of an intervention that they stop the trial before completion because it would be unethical to continue the study.

    I know that the follow-up period in the Phase III trial that's now ongoing is 24 months but if benefit is clear earlier, would there be a possibility of the trial being stopped early and rtx being rolled out faster?
     
  2. Sasha

    Sasha Fine, thank you

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    This is from November 2011 on the MEA website - I don't know if there's more recent guidance.

    This whole thing of off-label use confuses me. My GP has for years prescribed me, on the NHS, a drug for sleep which I now find is an off-label use for it: and yet I can't get rtx on the NHS for my ME.

    Is that because the risks are higher, and/or the costs?
     
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  3. Sallyagerharris

    Sallyagerharris

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    Having read and I appreciate it is early days that so far rituxamib seems to work better with younger patients with ME it is helpful and hopeful to know that there may be possibilities to potentially be able to get proper access to treatment.

    The hope of course is via the NHS for everyone but time and money are huge factors in getting any newly approved drug let alone with the challenging history ME has and continues to have.

    One of the many sad things about ME for me is not having capability to raise awareness more, raise funding ... I do a tiny bit with the energy I have but do "envy" people who can run marathons or who have normal energy to campaign. This illness seems particularly harsh with the physical, mental and emotional restrictions for so many of us.

    I would rather have a shorter but more quality of life but with some capability "to do" (i.e. To be well enough to give rather than taking and particularly do stuff with ME) but do realise that we need to see what happens as the Norweigan trials continue.

    Sorry bit of a vent
     
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  4. Scarecrow

    Scarecrow Revolting Peasant

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    Don't quote me on this and I'm not sure if I'm going to be able to find the source but I think F&M have ruled out an early unblinding. They're going to see it out to the bitter end.
     
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  5. Scarecrow

    Scarecrow Revolting Peasant

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    Yes, this was true in the Phase II but the number of participants was too small to have any significance. As you stated later in your post, we just need to wait and see what happens in the larger trial. :)
     
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  6. MeSci

    MeSci ME/CFS since 1995; activity level 6?

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    That label is for Non-Hodgkin's Lymphoma and Chronic Myeloid Leukaemia. You won't get Tumour Lysis Syndrome unless you have a tumour. I think we have discussed the differences between warnings/risks for ME (and doses?) and cancer elsewhere.

    EDIT - sorry - Chronic Lymphocytic Leukaemia - getting my words wrong at the moment - I knew it was Lymphocytic as it is what my friend has and she is taking a drug that can cause TLS.
     
    Last edited: Aug 8, 2015
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  7. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    I think that is unlikely, partly because Drs Fluge and Mella are very keen to play things exactly by the book to avoid any compromise of the results. However, things may change in other ways. The publication of the follow on data is an important extra piece of confidence building.
     
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  8. Izola

    Izola Senior Member

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    Oh dear, Violetta. I can't possibly read all that misery. It's good I spent my money on mine and other's kids! iz :D
     
  9. Izola

    Izola Senior Member

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    I'm so sorry about your friend. iz
     
  10. panckage

    panckage Senior Member

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    Is there an easy way to give myself cancer so I can try this? Maybe eat mcdonalds every day or something? :D
     
  11. Jo Best

    Jo Best Senior Member

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    Just cross-referencing to this thread - http://forums.phoenixrising.me/index.php?threads/dr-%C3%98ystein-fluge-to-give-public-talk-in-norwich-uk-26th-jan-2017.47946/#post-791642
    Invest in ME Research have arranged for Dr Øystein Fluge from Haukeland University Hospital in Bergen, Norway, to give a public lecture in Norwich in January 2017. http://www.investinme.org/ce-news-1611-04.shtml
    This is connected with the IiMER UK rituximab trial planned to take place in Norwich (hub of the IiMER Centre of Excellence for ME) - http://www.investinme.org/ce-news-1611-03.shtml
     
    Last edited: Dec 8, 2016
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  12. Jo Best

    Jo Best Senior Member

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  13. joshualevy

    joshualevy Senior Member

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    I don't know how things work in other countries, but in the US, that would be very unlikely for at least three reasons:

    First, phase-III trials for drugs are usually double blind. If a trial is double blind, and properly done, and there is no intermediate check by independent researchers, then there is no way to even know the trial has been successful until the data is unblinded. That happens at the end of the trial. Before that, no one even knows if it has been wildly successful or not. Does anyone know if the RTX has an intermediate analysis as part of the protocol? I did not think so.

    Second, except in unusual situations, the US requirement is two separate phase-III studies for marketing approval. Therefore, unless it is AIDS-in-1983, you need two phase-III studies for approval. So even if the first one really is wildly successful, you need the second one.

    Three, drug approvals are generally done by pharma companies. That is not a law or a rule, but in the last 10 years, I've never heard of a university research team doing the paperwork required for drug approval for a new indication (ie. public marketing approval). Obviously, they do IND (Investigative New Drug) approval paperwork, so they can start their trials, but they are not similar. Marketing approval requires a ton of specialized paperwork.

    (There is a separate issue, which is will the US FDA accept this trial at all? The primary end point is defined it such a way that the FDA may simply ignore it. The secondary end points look better (to me), but usually the FDA focuses on the primary end point, so I'm not sure what they would do in this situation.)

    Obviously, none of this impacts "off label" use.
     
  14. viggster

    viggster Senior Member

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    Clinical trials in the US can be stopped early if there is a clear benefit detected, although it is rare. The cases I know about are in cancer care. In big, government-sponsored cancer clinical trials, a group called a 'data safety & monitoring board' (DSMB) can review data as the trial is ongoing and if a clear benefit or a clear harm strongly emerges, end the trial early. I have no idea how things work in Norway but I doubt a smaller trial like this would have a DMSB looking at data early.

    Here's a piece that explores the pluses and minuses of stopping trials early. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC516093/
     
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  15. Snow Leopard

    Snow Leopard Hibernating

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    To be frank, we have one major shot at this, best to let it go full term, since that is best practise. I think stopping early due to efficacy is suggested in cases where the people in the control group are dying! (or vice versa for harm)
     
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  16. Jo Best

    Jo Best Senior Member

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  17. deleder2k

    deleder2k Senior Member

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    You can receive two infusions 1grams infusions of Mabthera at Kolibri Medical in Norway for $9500 (+ consultation fee at your first appointment). Two infusions should be enough to see whether you respond or not. I am not sure if it is recommended though. Say you do two infusions at 1 gream each. After 10 months you start to recover. You feel extraordinary for 4-5 months. Then you deteriorate quickly. The B-cells has returned, and so has the autoantibodies. At this stage you will probably need a full start dose of two infusions for $9500 + maintenance infusions for say 3 times. That is $7000 more. If you have an enormous effect after say 9 months, I would do one more. That is what they are doing at the current Haukeland trial. Longer B-cell depletion leads to much longer remission. Some feel fine after several years. I think I remember that 50% or so had gradually increasing symptoms of a relapse after 2-3 years. If you read the open label phase 2 study you can see some data about this.

    It is a big risk, but that risk is mostly money wise. I don't know what to recommend. If cash was not an easy I would do it. If doing this treatment means that you have to stop paying your medical insurance, or that if is means that you have to get a quick personal loan with a 15% APR, you better reevaluate it. My guess that the results will be revealed in 1.5 years. Unless you have a lot of greenbacks lying around I would wait. But it all depends on what life you have.

    Tough decision for many of us to take, or not to take.
     
    Last edited: Dec 18, 2016
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  18. Kati

    Kati Patient in training

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    If a drug is approved for a designated disease, in many countries, then this drug is covered by the country's insurance or the private insurances.

    Personally, access to mainstream medicine is better for patients communities who have been left behind for decades, because it makes mainstream medicine legitimate. We very much need access to treatments that work. It all starts with the approval of one drug. Then there will be more.

    Mold avoidance stuff is very much 'out there'.
     
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  19. deleder2k

    deleder2k Senior Member

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    If RTX is approved for everyone with ME then every Norwegian citizen with ME can obtain it for free. Today one can claim up to 90% of costs of other kinds of drugs, even though they are not approved for the condition. With respect to Rituximab, it doesn't work like that in Norway. Rituximab is classified as a cancer medicine The Regional Health Authority (there's four of them in Norway) will be in charge. For patients that means they need to get treated with RTX at a hospital. If else they will have to cover the cost entirely. It is expensive. Very expensive - but still much cheaper than getting treatment in San Francisco. They charge around 2.5 times more or so if I am not mistaken.
     
  20. Gingergrrl

    Gingergrrl Senior Member

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    I think these are two different topics like apples and oranges (mold/mycotoxin illness and Rituximab). For me it is not one or the other. I had a viral trigger, and then exposure to severe toxic black mold as an additional trigger, and both contributed to the downfall of my health. Moving away from the mold, getting rid of our belongings, and doing mold testing/treatment was crucial as is now treating the autoantibodies with IVIG and hopefully later RTX. For me they are not mutually exclusive and not in competition with each other, they are just two different issues.

    I think that is true if someone were purely a private pay patient at OMI but if someone got insurance to cover part of the cost, or got financial aide from Genentech, it would be cheaper. Am not saying either would actually occur, just that it would be worth trying.
     
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