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Thread: CuATSM ?

  1. #11
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    What have sufferers got to lose ?

  2. #12
    Forum Member Kayleigh's Avatar
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    Unhappy

    Good point Jaxx. For many of us, our lives are slipping through our fingers like sand.
    Last edited by Kayleigh; 17th January 2019 at 22:27.

  3. #13
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    Quote Originally Posted by Admin_MND View Post
    It is positive to see such a clear example of how the money raised by charity supporters can effectively drive research to the point of a 1 clinical Phase trial. All involved should be congratulated on this achievement.
    Sadly it shows Australia can be congratulated but what part did MNDA play ? Nothing apparently

    It’s a worldwide disease so shouldn’t there be more international cooperation
    Last edited by Dis1960; 20th January 2019 at 18:47.

  4. #14
    Whenever we get a phase 1 reporting there is always a call to make drug available now.

    But the harsh reality is it is not proven yet.

    We have to wait for phase 2.

    It can’t be just made available. It’s a derived compound, and vastly expensive to make.

    Who would pay for the vast distribution?

    Why should we divert funds from other ventures etc?

    Such calls can actually delay the ultimate cure. That is the harm it can do.

    And it may be a complete false lead.

    All the trial has shown is it is safe for 24 weeks. The efficacy data is totally meaningless because there was no placebo arm.

    But it is an interesting drug.

    The Mnda are contributing to many major projects. Worldwide collaboration is happening. But we don’t need duplication.

    I want a therapy as much as anyone with mnd, but sadly we have to be rational and quite focused.
    Last edited by Onein300; 21st January 2019 at 00:07.

  5. #15
    Forum Member Kayleigh's Avatar
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    Many thanks for your interesting post Lee, and for putting foward the rational and scientific side of things.

    I appreciate what you have said about needing to stay focused on the harsh reality of situation.

    However, it is not always easy to keep a rational view of things when a diagnosis of MND can put such a strain on us emotionally.

    I find that even if my head is saying 'stay rational' about things, my heart is often breaking because of the devastation felt by not having an effective treatment or cure.

    I suppose that even if the drug is cleared for use in Australia, it will still have to go through further testing and approval for it to become available here on the NHS. These things take time - but feelings of desperation often arise, as time is not particularly on our side.

    I know that if my perspective on some things becomes 'too emotional', then the more scientific and rational folk here will soon ground me with the realities of the situation - and that is good - otherwise sometimes I could be 'clutching at straws' a bit to tightly, and pinning my hopes on things that realistically might not happen in my lifetime.

    We are all here, sadly, because our lives are affected by MND - but it is good that we all have different viewpoints and knowledge to offer - and I do love a good debate now and then!

    Take care everyone (especially if you are going out in the cold and frosty weather this week).

    Sending love to you all,
    Kayleigh x
    Last edited by Kayleigh; 21st January 2019 at 01:32.

  6. #16
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    The elephant in the room must be addressed : HOPE

    There is no hope for any of us with MND today but even a glimmer of hope for sufferers in the future would be good

    After all 30+ years of research funding by MNDA and others have little tangible results

  7. #17
    Kayleigh, morning.

    We quite rightly get emotional, afterall we are in a rather bad place.

    However, you ask a good question in here, which I think the answers to will help us all.

    You ask about whether if/when once approved in Australia would it require more trials etc?

    This is actually very important to understand and makes for an interesting discussion. I am writing a new post on my blog about international collaboration, and some bits will come out in that. However, I am going to give you my view on this question.

    The trial process is typically phase 1,2, (sometimes 2a,2b) Phase 3.

    That sounds dire doesn’t it. However, it all depends on the apparent efficacy of a drug as it goes through the phases.

    So if in the phase 2, wonderous efficacy was indicated, a drug can actually get approved after phase 2 without a phase 3.

    But also, internationally, for example in the U.K. what would have to happen is the drug company would apply for a marketing license and or NICE review board would consider the available evidence. There would be no need for more trials if the evidence meets our UK standards (or European currently!). However, if the evidence was weak, the drug showed some effect, but only marginal, it might require a phase 3 or local trials.

    I believe, but cannot be certain, that the Australian levels of proof are similar to ours, so I would suspect if it did look good, it would be a small (I say that with tongue in cheek) matter of review and approval.

    To give you the counter side, the drug Edaravone (Radicava) is approved by the FDA in the USA, but not here. The reason? Well the European levels of evidence are much stricter (and so are U.K.). There was a lot of surprise worldwide when Edaravone got approved, and as far as I can see there is zero chance of it getting approved here unless the manufacturer applied for approval and did a detailed phase 3. Neither are forthcoming. You can make what you think of that. Most neuros use the phrase “we are not missing much with Edaravone”!

    On the subject of collaboration.

    Here in 2018/19 we have an exceptional amount of collaboration, and perhaps more importantly communication. All the associations talk to each other, and trials are setup, not duplicated, for many research aspects all around the globe. This is in stark contrast to 10 years ago.

    Now of course there is competition, but in reality scientists are pure thinkers. So they all read each other’s papers, written to exacting standards, and any “major” advancement will be taken up by scientists globally. One of the first tenants of scientific proof is repetition. There are many many arms of new research that suddenly teams around the globe suddenly start to begin research on.

    But just to re-iterate, there are many other areas of research going on in the UK, USA, Europe, Australia etc. The real breakthrough is just as likely to come from the UK as anywhere now.

    Key to future research is funding.

    One final comment. Remember the ice bucket money raised in the U.K.? It was about 7m. That is ALL now effectively gone and committed to research! So the search for new funds is always key.

    We will get there.

    Rock on all.

    Oh and the phase 2 for CUatsm is going to take place in the USA and Australia. In the U.K. there are two phase 2 trials in process on other treatments.

  8. #18
    Quote Originally Posted by Dis1960 View Post
    The elephant in the room must be addressed : HOPE

    There is no hope for any of us with MND today but even a glimmer of hope for sufferers in the future would be good

    After all 30+ years of research funding by MNDA and others have little tangible results
    I would have to disagree on results. There has been significant progress and the mnda have been instrumental in that progress. Remember MND is an extremely complex disease. But also, we are now at a stage where the vast data/work/progress is reaching a tipping point. It might be tomorrow, next week or 5 years, but there is NOW real hope of a significant leap forward.

    For us with the disease now, that is no reassurance, but we have to think of future generations. I know I do. My mid twenties son for example. I don’t want him to develop MND, and the research now we hope can prevent it in the next 20 years.

    We need more awareness and more funds. Research is so so expensive. And the relatively small amount we got in the ice bucket challenge (7m) is but a sniff at the money we really need.

    For example 130m was spent on cjd research following the scare 20 years ago in the U.K. and probably 4 people died from it, and no one in the last 10 years has been infected. And yet this amount was spent! So awareness of disease with decision makers is vital.

  9. #19
    Quote Originally Posted by Admin_MND View Post
    Good afternoon all,

    Thank you to Ellie for sharing the research teams recent blog regarding CuATSM.. We also wanted to give a brief overview to all users of the forum.

    Recent press coverage of results of the Phase 1 trial of Copper ATSM (CuATSM) have reported that the compound produced a 70% reduction in rate of progression and an improvement patients who took the drug at 72mg/day.

    As an Association we recognise this trial as an important first step in the drug development process. However, we must remain realistic when interpreting and reporting data from this small, Phase 1 study. Phase 1 studies are designed to determine what dose of a new drug is safe for people to take. It is too early to confidently report on the effectiveness of the drug although passing this safety milestone is an important development.

    CuATSM is not yet at a stage where a clinician can prescribe it as a treatment. Drug development is a long journey, where any drug has to pass important rigorous checks before approval as a medicine. Historically, many promising drugs have failed at this first (Phase 1) stage.
    We are hopeful that CuATSM will prove to be an effective therapy in the future. The next and important stage Phase 2 - is to perform a robust trial on a larger number of people to really see if it can alter the disease progression. This randomised, blinded, placebo-controlled trial, is planned for later this year in Australia. We will keep the MND community updated on any news from this study.

    It is positive to see such a clear example of how the money raised by charity supporters can effectively drive research to the point of a 1 clinical Phase trial. All involved should be congratulated on this achievement.
    I advise all on this forum to read the detailed MNDA blog. It is very precise. Thank you MNDA for posting.

  10. #20
    Forum Member Kayleigh's Avatar
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    Many thanks Lee, for your very detailed and informative answer to my question about the approval of CuATSM for use in the UK, if it is approved in Australia. Also, many thanks to the MNDA for their very helpful blog.

    The information has helped me to focus my thoughts on the realities of the situation about the approval process. In future, I will be more mindful of the complex process that is involved before a drug can be approved. Also, I will no longer presume that just because a drug has been approved in another country, it will soon become available for us here on the NHS. However, you made a very interesting point Lee, about there being similarities between the Australian process and process of approval here - and, hopefully, this will bode well for us here if CuATSM is approved in Australia.

    There is, of course, the other hoop to be jumped through - even if approved, will it be deemed cost effective enough for it to be funded by the NHS? As it is administered orally, (rather than more expensive methods such as intravenously) I would presume that's an advantage in terms of cost-effectiveness - and hopefully, the drugs companies would supply it a price that the NHS could afford to pay!

    I have kind of given up on Edaravone being made available on the NHS. However, sometimes it does cross my mind - what if I lived in America, and the cost was covered by my health insurance company, would I go ahead with taking Edaravone? I would probably disregard the seemingly rational opinion of many scientists, who have questioned its effectiveness due to lack of proof. My heart would probably rule my head on this one, with the emotional response of "what have I got to lose?".

    "Where there's life, there's hope". I gleen hope from the fact that many drugs trials are currently taking place, and hopefully these will have a positive outcome for future generations, if not for us. Also, I hope there will be more international collaboration, so that the time it takes for drugs to be approved for use in more than one country will be greatly reduced.

    Best wishes,
    Kayleigh x
    Last edited by Kayleigh; 21st January 2019 at 16:25.

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