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Thread: Gene silencing Work in the UK

  1. #1
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    Gene silencing Work in the UK

    Dear MNDA,

    Given the recent success in treating Huntington's Disease and the breakthrough in delivering medication through the brain-blood barrier, is the MNDA aware of any work targeting the many known genetic causes of MND? I have had my whole genome sequenced for the purpose of the faulty gene being corrected.

    Graham
    Copyright Graham

  2. #2
    Forum Member MNDConnect's Avatar
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    Dear Graham,

    The recently talked-about drug that showed promising results in people with Huntington’s Disease is indeed very encouraging and we hope that future trials will show that it has the ability totreat the disease effectively. The drug itself targets the faulty proteins that are created from the ‘faulty’ gene that people with Huntington’s have, and the last trial showed that this protein was significantly reduced in people taking the drug.

    Interestingly, a recent press release by the pharma company Biogen showed similar result in MND, specifically in people with a faulty SOD1 gene. This treatment, called tofersen, overcomes the blood-brain barrier by being injected directly into the spinal cord, and binding to a stretch of the gene which contains the mistake, preventing a faulty protein from being made. The early-stage trial showed a reduced number of this toxic protein in people who were receiving the drug and a bigger follow-up trial is now being rolled out to show the effectiveness of the drug in a larger number of people. It is hoped that other types of MND could also be targeted using this therapeutic mechanism (called antisenseoligonucleotide), and a clinical trial for people with a mistake in the C9ORF72 gene is now starting to recruit participants. You can read more about this study on our blog here: https://mndresearch.blog/2019/05/07/...ase-1-2-trial/

    If you have any follow-up questions you can contact research@mndassociation.org directly.
    Kind regards
    Ruth

  3. #3
    Forum Member Lynne K's Avatar
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    Thanks Ruth. If the trial starts in the UK with people who have the C9ORF72 faulty gene please let me know how I can be included. Many thanks. 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
    Forum Member Ellie's Avatar
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    Hi Lynne,

    Antisense was one of the topics covered in Tuesday's ALS Unfiltered's webinar - it's not yet available to view back, I'll keep an eye on it for you.

    If you wanted to keep on top of when that particular trial may be recruiting in the UK, check the following site https://clinicaltrials.gov/ct2/resul...&Search=Search


    You can add in BIIB078, C9orf72 or Biogen as filters for that specific Antisense trial (BIIB078 is the drug's code name)
    Atm, it won't find any results for the UK, but here's the info, inc eligibility criteria, for the current US & Canada trial: https://clinicaltrials.gov/ct2/show/...lerosis&rank=1


    Take care.

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

  5. #5
    Hi Lynne

    A more general study into C9orf72, i.e. not a specific antisense drug trial, is recruiting in the UK now.

    I posted about it on April 6th. It’s the C9orf72 Cohort Study being led by Prof Kevin Talbot of the Nuffield Department of Clinical Neurosciences, University of Oxford. Three groups of people are being recruited: those with MND caused by a mutation of the gene, those with sporadic MND and no mutation, e.g. me, and healthy subjects.

    https://www.hra.nhs.uk/planning-and-...-cohort-study/

    The aim is to collect all sorts of neurological data and physical samples to try to understand the processes at work in this type of familial MND.

    The project has ethical approval for 300 participants. They would be happy with 100 or a minimum of 60. Currently they have about 20, I think.

    If you’re interested you could email Prof Talbot or the research coordinator at the JR, Lynn Ossher (lynn.ossher@ndcn.ox.ac.uk).
    It would be good if you were on the MND register beforehand.

    Doug

  6. #6
    Forum Member MNDConnect's Avatar
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    Hi all

    The plans to include UK centres in the C9ORF72 study are currently being put together and once we know more, we will publish this opportunity on our website: https://www.mndassociation.org/resea...t-in-research/

    Best Wishes

    Rachel

  7. #7
    Forum Member Lynne K's Avatar
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    Thanks, Ellie, Doug and Rachel. 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.

  8. #8
    Forum Member Lynne K's Avatar
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    Quote Originally Posted by Doug Carpenter View Post
    Hi Lynne

    A more general study into C9orf72, i.e. not a specific antisense drug trial, is recruiting in the UK now.

    I posted about it on April 6th. It’s the C9orf72 Cohort Study being led by Prof Kevin Talbot of the Nuffield Department of Clinical Neurosciences, University of Oxford. Three groups of people are being recruited: those with MND caused by a mutation of the gene, those with sporadic MND and no mutation, e.g. me, and healthy subjects.

    https://www.hra.nhs.uk/planning-and-...-cohort-study/

    The aim is to collect all sorts of neurological data and physical samples to try to understand the processes at work in this type of familial MND.

    The project has ethical approval for 300 participants. They would be happy with 100 or a minimum of 60. Currently they have about 20, I think.

    If you’re interested you could email Prof Talbot or the research coordinator at the JR, Lynn Ossher (lynn.ossher@ndcn.ox.ac.uk).
    It would be good if you were on the MND register beforehand.

    Doug
    Hi Doug, thanks for the info, but I'm up North so no chance of me travelling to Oxford, 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.

  9. #9
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    HI Ruth and Rachel,

    Thank you for the prompt reply.

    That is excellent news for the people with the C9orf72 gene fault in the Oxford catchment area that are fortunate enough to be able to enrol on the trial. This is the very best opportunity yet of beating this wicked disease.

    My gene defect has not yet been identified, however there are UK scientists working on it I believe under the MINE project umbrella. My gene defect has links to a form of PD and PSP. I would be most grateful if you could advise on how my gene defect may be identified?

    Graham
    Copyright Graham

  10. #10

    Project MinE

    If youíre unfamiliar with Project MinE (https://www.projectmine.com) this recent paper in EJHG gives a good overview. Unusually for Springer Nature, itís open access so itís easy to download the pdf.

    https://www.nature.com/articles/s41431-018-0177-4

    If nothing else, it highlights the huge scale of the problem scientists are facing in these whole genome sequencing projects. Needles in haystacks donít come close!

    Doug

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