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    #16
    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

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      #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.

      Comment


        #18
        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.

        Comment


          #19
          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.

          Comment


            #20
            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; 21 January 2019, 15:25.

            Comment


              #21
              Originally posted by Kayleigh View Post
              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? - but that's probably a separate issue.

              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
              Just take every day as it comes. Edarvrone to me is horrific. You have to be linked up to a drip every day for two weeks!! And then no real proven benefit! So a treatment that makes you less mobile for 2 weeks a month!!! And we are immobile anyway!

              That’s some burden! It needs to be a pill for any small or marginal drug.

              I know it is an obvious fact, but we are living in the best time for having this disease. The power for social media and the community will come when there is a true breakthrough. Once a true treatment is found, old approval processes will fall by the wayside and that is when we should start petitions etc etc. But not just yet.

              Let’s keeping rocking guys!

              Comment


                #22
                Thanks Lee. I think I'll forget about Edavarone, and keep my fingers crossed for the other possibilities becoming a viable reality.

                In the meantime, your advice to 'keep on rocking' sounds good to me! Now, where did I put that Status Quo CD?

                All the best to you and your family,
                Kayleigh x

                Comment


                  #23
                  Thanks Lee for your very helpful posts.

                  Just to flesh out the discussion a little around drug discovery –

                  In general, drugs need a target. That means research has to be done to understand how a disease works, with the aim of identifying some step in the process where pharmacological intervention might be disruptive and successful.

                  As a result of painstaking research by medical scientists over many years, we now know a hell of a lot more about MND than we did two or three decades ago. But so much is still unknown. Research teams around the world are still putting together the MND jigsaw. And the MNDA is helping to fund that, along with comparable organisations in other countries.

                  By coincidence, I had a brief insight into some of the Australian research last July when I was at Macquarie University for a conference on another field of science entirely. I was given a tour around the MND labs and met many brilliant, dedicated people doing amazing research. They were, however, very happy to acknowledge that they were still a long way away from understanding many of the cellular and molecular processes involved in our disease, especially in sporadic MND.

                  Ever since I was diagnosed, I’ve volunteered to help with the various projects being conducted by equally brilliant researchers at the John Radcliffe Hospital in Oxford. Up to now this has mainly meant having blood and cerebrospinal fluid taken at intervals. But in a couple of weeks’ time it will involve an hour in an MRI machine wearing a “hat” covered in sensors and doing various tests while my brain is scanned in real time. None of this will lead directly to a new wonder drug, but it may help to shed a little more light on how MND progresses.

                  There is also the possibility that research on other neurodegenerative diseases could throw up signposts for MND scientists. As I understand it, CuATSM was originally used as a contrasting agent in the imaging of hypoxic cancer cells.

                  One day…….

                  Doug

                  Comment


                    #24
                    Doug,

                    Good stuff. I took part in one 3 years ago. It was fun. Make sure you get a copy of the scan of your brain. I asked them and they were happy to give it.

                    I show it to people who think I am an idiot, Hahaha!

                    Yes you are right we are a long way off knowing the precise pathology actions but boy are we moving fast now. The combination of many trials, the genetic project projectmine and the seeds of gene therapy we are getting much closer.

                    I suspect we will get a more effective slowing treatment before anything. In reality if we could slow by 50% (a real 50% not just an alsfrs rating) and doubled lifetime, that would be a major achievement.

                    Lee

                    Comment


                      #25
                      This is exactly what I was saying - but in more detail - about drugs and targets. And about common factors between different neurodegenerative diseases:

                      https://mndresearch.blog/2019/01/23/...-from-glasgow/

                      Nobody should doubt that much splendid research is being done.

                      Doug

                      Comment


                        #26
                        From The International Alliance of ALS/MND Associations Scientific Advice Council

                        Some feedback on the CuATSM Trial...

                        Dear members,
                        As you may be aware On January 7th, a media release was published stating that CuATSM slowed disease progression by 70% in the Phase 1 clinical trial, resulting in an international outcry for people living with ALS to access CuATSM, This created particularly strong interest in Australia and the UK, but also throughout the rest of the World
                        The Alliance were contacted by many of our member organisations for more information and so the Alliance have sought the advice of it's scientific advisory council for a balanced and scientifically accurate response to these claims.

                        After studying the data the response from the Scientific advisory council is as follows:


                        An Australian company called Collaborative Medicinal Development were funded through FightMND to run a Phase 1 clinical trial of a compound called CuATSM that ended in 2018. The trial was designed only to determine if CuATSM is safe for humans and to determine what dose (if safe) would be ideal to test in a further clinical trial (Phase 2 or 3) that would be designed to determine if it has the ability to alter ALS disease progression.
                        CuATSM is a drug that has shown potential to treat ALS in laboratory animal models and was recently studied for human safety in a small Phase 1 clinical trial by the company Collaborative Medicinal Development in Australia. At the doses tested, using clinical grade CuATSM, it was considered safe, but a press release also states that the company has seen a substantial slowing of disease progression. This clinical trial was not designed to make such a public statement and a number of aspects in its design require this result to be taken with as much caution as possible. Essentially, there is no substantiated scientific evidence that CuATSM has any effect on ALS in humans. Furthermore, until there is a peer-reviewed publication of the data, it is not possible at this time to evaluate the safety beyond the claims of the release. A Phase 2 clinical trial is being setup to further test safety and to more rigorously determine if there is any effect on the course of disease. The field remains hopeful that CuATSM will work, but the proper trials need to be done before anything can be known.



                        We hope this information is useful to our membership. Within the Scientific Advisory Council there is a significant level of knowledge regarding the pre-clinical data, key investigators and and clinical trial nuances surrounding CuATSM. If you do have any other specific questions that are not answered by this response they can be directed to the Scientific Advisory Council via the Alliance. Please send these questions to [email protected]

                        Kind Regards
                        Amanda Bourne
                        Alliance Coordinator



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                        You are receiving this email because you are a member or a friend of the International Alliance of ALS/MND Associations.

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                        Last edited by Ellie; 8 February 2019, 16:13.
                        ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
                        Eye gaze user - No working limbs - No speech - Feeding tube - Overnight NIV.

                        Comment


                          #27
                          Hi Ellie,

                          Thanks for the update. Regardless of the caution advised it does sound promising.

                          Love,
                          Barry
                          I’m going to do this even if it kills me!

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