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Time from diagnosis to death causing problems with clinical trials

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  • Lynne K
    replied
    Thanks Ellie. It was great. Lots of info and well explained. Lynne
    Last edited by Lynne K; 20 May 2019, 13:47.

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  • Doug Carpenter
    replied
    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

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  • Ellie
    replied
    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.

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  • Dis1960
    replied
    It is still available to watch ( 91 mins ) at https://www.als.net/als-webinars/archive/05142019/

    Leave a comment:


  • Lynne K
    replied
    I missed the webinar. I'd planned to log in but we were out. Lynne

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  • Dis1960
    replied
    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

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  • Doug Carpenter
    replied
    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

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  • Ellie
    replied
    Thanks for posting that Doug.


    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...

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  • nunhead_man
    replied
    Good morning Doug

    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

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  • Doug Carpenter
    replied
    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

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  • Gillette
    replied
    Hi Doug,

    I am happy to be a guinea pig - there may as well be something useful that comes from having MND.

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  • shrew
    replied
    Mick was diagnosed last July in Preston. He only sees the Respiratory department. When he had his Rig done in january a lady came and asked him to be part of a study, he agreed. She asked when he was next at clinic to see a nurse, we said mid March. She said 2 weeks before due to come she would send out paperwork for us to complete a diary of everything that he had eaten, we heard nothing!

    Leave a comment:


  • Doug Carpenter
    replied
    Biomarkers

    Hi Andy

    Yes, it can be difficult for researchers to assemble cohorts for trials – see my reply to Dina above. My view is we should help them to help maybe not us but future generations of MND patients.

    The P75 test is attractive because it’s easy to collect and analyse samples. And there is some evidence

    https://www.nature.com/articles/s41598-017-05430-w.pdf

    https://n.neurology.org/content/neur.../1137.full.pdf

    that it correlates with progression. However, from the published data, I think the correlation with the ALSFRS-R is weak. Nevertheless, it’s yet another potentially useful biomarker.

    There’s a very good recent review paper on biomarkers:

    https://www.ncbi.nlm.nih.gov/pmc/art...r-10-00191.pdf

    from the folks in Queensland that recognises the multifactorial nature of the problem.

    On a separate topic, I’m just hoping I’ll live long enough to learn the outcome of the IC14 monoclonal antibody study at the hospital there.

    Doug

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  • Doug Carpenter
    replied
    Hi Dina

    You raise an important point.

    In my experience of the researchers at the MND clinic at the John Radcliffe Hospital in Oxford – yes, they are proactive. I was asked on diagnosis if I would take part in various studies and have done so for the past couple of years.

    One of the reasons I was banging on recently about everybody should sign up on the MND register was because this is the place where researchers can find potential subjects if they don’t come across them at clinics.

    Well done for being a guinea pig!

    Doug

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  • Gillette
    replied
    Hi,

    Does anyone have any idea whether staff at MND clinics routinely ask patients if they would be interested in volunteering for trials and/or studies?

    When I received my diagnosis I was an in-patient and had already offered myself as a guinea pig for student doctors and student nurses. It was a natural progression to offer myself for any studies/trials. The research team did all their necessary tests while I was still an in-patient. Now they automatically ask me if I would like to take part, if they have any others that I might be a 'fit' for.

    The research team also go to the MND clinic to see if they can sign people up.

    I'm just wondering if clinics need to be more proactive in their recruiting?

    Leave a comment:

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