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C9orf72 Gene Therapy Targeting Trial UK - Phase 1

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    #1

    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 it’ll be very interesting to see the trial results when published.

    New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK - MND Research Blog
    https://mndresearch.blog/2019/09/19/new-gene-therapy-targeting-c9orf72-als-begins-phase-1-clinical-trial-in-the-uk/
    Reading Time: 2 minutes This article was written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London. Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for patients affected by a specific genetic form of ALS, the most common type of MND. ALS is a…


    Love Ellie.
    ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
    Significant bulbar impairment - No functional limbs - No speech - Feeding tube - Overnight NIV - Eye gaze user    .
    ​
    Tags: None
    #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!

    Comment

      #3
      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 balance.

      I'm staying positive and taking each day as it comes.

      Comment

        #4
        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.

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          #5
          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 balance.

          I'm staying positive and taking each day as it comes.

          Comment

            #6
            Fingers cossed that you get on the trial John and it goes well.

            Love Ellie.
            ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
            Significant bulbar impairment - No functional limbs - No speech - Feeding tube - Overnight NIV - Eye gaze user            .
            ​

            Comment

              #7
              Exciting times! I hope you are, indeed, taken onto the trial, John.
              Dina

              Trying to keep positive, but not always managing.

              Comment

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

                Comment

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