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Thread: Time from diagnosis to death causing problems with clinical trials

  1. #11

    Biomarkers

    Another very comprehensive review of biomarkers - including imaging studies - has recently been published by the SITraN team at Sheffield.

    https://www.frontiersin.org/articles...019.00291/full

    Doug

  2. #12
    Forum Member nunhead_man's Avatar
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    Good morning Doug

    Quote Originally Posted by Doug Carpenter View Post
    Another very comprehensive review of biomarkers - including imaging studies - has recently been published by the SITraN team at Sheffield
    Thank you - as you say, really good summary.

    Key line seems to be "Despite excellent attempts in each field, single useful biomarkers of ALS are as of yet out of reach"

    What we do have is markers that allow some segmentation of those diagnosed for research study - e.g. the breathing test that has led me to be accepted for the for the REFALS trial, which is looking at the effects of oral levosimendan on the respiratory function in patients with ALS.

    Mind you, I was on the edge of that as its almost 4 years since I saw the first signs of MND and they are not accepting people who were showing signs more than 4 years ago.

    So I guess there is progress but with the caveats about selecting folk clinical trials above.

    Warmly

    Andy
    Warmly


    Andy

    ​Diagnosed 03/2015. Limb onset (arm) sporadic ALS/MND.
    MND hitting - now 50% left arm and 90% right arm, plus other bits including left shoulder

  3. #13
    Forum Member Ellie's Avatar
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    Thanks for posting that Doug.


    Quote Originally Posted by nunhead_man View Post

    Key line seems to be "Despite excellent attempts in each field, single useful biomarkers of ALS are as of yet out of reach"
    And with every failed attempt to find even one valid biomarker, it look as if MND is more of a syndrome...
    ​Diagnosed 03/2007. Sporadic Definite ALS/MND Limb Onset.
    Eye gaze user - No working limbs - No speech - Feeding tube - Overnight NIV.

  4. #14
    Quite right, Ellie.

    I’m sure there are some mechanisms that will be found to be common across several types of MND – and possibly other neurodegenerative diseases. But as far as initial triggers are concerned, for example, I expect many different ones to emerge and different disease pathways found that will need to be explored.

    Researchers face a daunting task!

    Doug

  5. #15
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    One point made in the webinar was that ALSFRS-R is somewhat subjective so a better indicator would eliminate subjective bias.

    I hadn’t realised before the webinar just how many genes are implicated in inherited MND

  6. #16
    Forum Member Lynne K's Avatar
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    I missed the webinar. I'd planned to log in but we were out. Lynne
    ALS diagnosed November 2017, limb onset. For the 4 yrs previously I was losing my ballance.
    I'm staying positive and taking each day as it comes.

  7. #17
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    It is still available to watch ( 91 mins ) at https://www.als.net/als-webinars/archive/05142019/

  8. #18
    Forum Member Ellie's Avatar
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    Hi Lynne,

    It's now available to watch, I posted the link for you on the webinair thread https://www.als.net/als-webinars/

    You need to register to watch it.

    Love Ellie.
    ​Diagnosed 03/2007. Sporadic Definite ALS/MND Limb Onset.
    Eye gaze user - No working limbs - No speech - Feeding tube - Overnight NIV.

  9. #19

    ALSFRS-R limitations

    Hi Dis1960

    Yes, you’re right. The ALSFRS-R is a rather crude metric. A bit like using the FTSE-100 as a measure of the health of the whole economy.

    Monitoring ones own ALSRFS-R, i.e. normalising out the subjective bias, can be useful. But comparing scores between patients is fraught with difficulty.

    If you key your symptoms into one of the oldest (1996-2007) and biggest (>6000 patients) ALSFRS databases – the University of Massachusetts CARE program – you’ll see on the plot at the end the huge spread of scores across the population.

    https://www.outcomes-umassmed.org/als/alsscale.aspx

    Bear in mind too that not all ALSFRS-R points are equal! A one-point change in my score may not mean the same as a one-point change in your score, depending where we are on the scale.

    Getting rid of subjective bias when trying to quantify something that is essentially qualitative is notoriously difficult. The ALSFRS-R is a quick and crude statistic, but clinicians like it. You just have to be aware of its limitations and interpret the numbers with care.

    Doug

  10. #20
    Forum Member Lynne K's Avatar
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    Thanks Ellie. It was great. Lots of info and well explained. Lynne
    Last edited by Lynne K; 20th May 2019 at 14:47.
    ALS diagnosed November 2017, limb onset. For the 4 yrs previously I was losing my ballance.
    I'm staying positive and taking each day as it comes.

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