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Thread: C9orf72 Gene Therapy Targeting Trial UK - Phase 1

  1. #1
    Forum Member Ellie's Avatar
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    C9orf72 Gene Therapy Targeting Trial UK - Phase 1

    This is a big deal for some of our (favourite ) Forum Members.

    Phase 1 of its clinical trial started this week, so very early days, but itíll be very interesting to see the trial results when published.

    https://mndresearch.blog/2019/09/19/...ial-in-the-uk/

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

  2. #2
    This is early days but this along with the biogen SOD1 Trial are in my mind the key trials ongoing in the world today.

    Rock on!

  3. #3
    Forum Member Lynne K's Avatar
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    Thanks Ellie. I read all about it previously but forgot. Appreciate the reminder. I read it again. There's other research going on but no other to my knowledge for C9ORF72 in the world. I get the ALS news letters.

    I hope that the participants of this trial stay safe and the scientists can learn from it. Obviously I hope that it's the first of 3 associated trials and that it turns out to be a useful treatment but I'm keeping my feet firmly planted. 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.

  4. #4
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    I hope to get on the Phase 1 trial at the National Hospital for Neurology in central London. I was phoned by one of the doctors there yesterday. I plan to visit them in the next few weeks for an informal chat. This is indeed a big deal for those of us with the C9orf72 mutation. They are running 6 cohorts with 8 people in each. Cohort 1 is given a very low dose of drug, Cohort 2 gets a higher dose and so on. The cohorts run in series. The aim, as I understand it, is to assess people's tolerance to the drug.

  5. #5
    Forum Member Lynne K's Avatar
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    Good luck John. I hope that it doesn't make you feel ill. Take care. 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.

  6. #6
    Forum Member Ellie's Avatar
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    Fingers cossed that you get on the trial John and it goes well.

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

  7. #7
    Forum Member Gillette's Avatar
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    Exciting times! I hope you are, indeed, taken onto the trial, John.
    Dina


    Trying to keep positive, but not always managing.

  8. #8
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    Thank you, all. I'll post updates.

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