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    • #14510
      Dagmar
      Keymaster

      Nurown, the promising stem cell treatment for ALS, is currently in a Phase 3 clinical trial, with results expected to be announced December, 2020. But, controversy between the company and ALS patients is dominating social media. For example: some patients are demanding early access, others are petitioning lawmakers and final results of this expensive treatment have yet to be confirmed on a large cohort.

      Here are a few of the key points, with discussion questions for our forum members:

      • The company’s Phase 1 and 2 trials had positive results with 2-3 participants sharing on social media how they experienced a reduction in symptoms. (Here is a news report on Mark Bedwell’s remarkable results) Question: Why don’t other trial participants come forward to share their experiences?

       

      • This led to other ALS patients demanding early access to the treatment under the new Right to Try law. But the company is resisting this early access, stating they want to wait until Phase 3 is finished, in order to preserve the integrity of their testing. (Here is a link to the company’s position) Question: Do you think they are correct in waiting? Should lawmakers force companies to provide ALS patients access to treatments not yet approved by the FDA?

       

      • Currently, the Nurown stem cell treatment is estimated to cost $300,000/patient. But it has not yet been established if the effects will eventually wear off, requiring repeat treatments. In addition, the company claims that stem cell treatment is neuroprotective; for existing motor neurons, but it has not yet been proven that it will also create new motor neurons to replace those that have already died off. Question: Should this hefty cost be covered by health insurers? Especially since Nurown only slows down symptoms, and is not a cure for ALS?

      What are your thoughts about Nurown as a treatment for ALS? Has this conversation topic sparked additional questions for you? Would you try Nurown; and what about its cost?

    • #14519

      I am not a doctor, but I think that Nurown is the fruit of a line of research which is valid: How to administrate neurotrophic factors where they could be useful.

      Nurown is certainly not the final cut in this line of research, I expect new therapies to be more effective and/or easier to administrate. Intrathecal administration is not fun at all, and probably Nurown will be needed every few months. On the long term it might be risky.

      What we need is a therapy which is easy to tolerate by the patient, that can not only stop the progression but also enable people to recuperate their previous health.

      – To stop the progression I only see genetic therapies with intrabodies targeting TDP-43 such as what the group of Jean-Pierre Julien in Canada or the group of Makoto Urushitani in Japan are studying. Therapies with neurotrophic factors are not designed to achieved that.

      – To recuperate pALS’s previous health is not something which can be done easily. People older than 70 yo that have not moved for 5 years (for example after a spine accident) cannot recuperate even with therapies that are helpful with young people that are treated just after the accident. Neurotrophic factors may help here.

    • #14522
      Dagmar
      Keymaster

      I agree with you Jean-Pierre:

      Nurown is an indication of “things to come” in the world of stem cell therapy, but at it’s present state, Nurown is not ideal for the general ALS population. This is due to (as you stated) a difficult delivery process, high cost and high possibility of needing repeat treatments.

      The current excitement and push for Nurown reminds me of the same when Radicava was emerging…. the spin in marketing implied it was “the answer” to ALS…but, over time, early adopters became disappointed and dropped out from using it. Radicava had the same negatives of high cost and difficult delivery system.

      Researchers need to focus on stopping progression of ALS. And yes, unless the patient has been able to maintain regular therapeutic movement during the course of their disease, they may not be able to regain movement once it is made available to them.

    • #14539
      Anonymous
      Inactive

      There is a method of subpial delivery which is safer than IT.  That method should be used for Nurown if possible since it will require repeated treatments, but from what I’ve read, the IT method is used which entails more risk. The phase 3 trial is being conducted at many locations with some of the best ALS researchers and neurologists involved.  They are deriving the cost from certain cancer treatments.  It may be as much as $400,000. or more.  I don’t anticipate any treatment to be inexpensive for ALS including oral ones.

      It will be interesting to see what materializes.  For some reason my mind revisits the Diaphragm Pacer (phrenic nerve stimulator)   and  GM604.

    • #14545

      I agree with you Kasper, particularly about the risk that no insurer will pay so much.

      This reminds me about Glybera, it was a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare inherited disorder which can cause severe pancreatitis.

      https://newsinteractives.cbc.ca/longform/glybera

      Its cost was $1 million in 2015, making it the most expensive medicine in the world at the time. In 2012, the European Commission approved Glybera, but only one person was treated with Glybera and at the end the biotech went bankrupt and 8 years later there is still no approved drugs for LPLD. Mostly the costs were due to bureaucracy.

      What is strange is that a few scientists claimed that the cost of production of this drug were actually only a tiny fraction of 1 million. Last year they made a proof of concept with only $7000.

      Scihouse goes to BioHack The Planet 2019

      But the scientists that created Glybera told that this low cost is unrealistic as it does not take in account the cost of training medical staff around the world nor distributing the therapy through secure channels that themself are very costly.

      In turn I find this critic not very realistic, as the disease is even more rare than ALS, they could have sent the patient (who often were not disabled) to one clinic, instead of trying  to send to drug close to the patient. Even in cancer there is something similar. A new therapy is CAR-T, if a patient in Europe is treated with this therapy, cells are extracted from the patient, sent over the Atlantic, processed in US and sent back in Europe. Does this serves the patient (and insurer) interests?

      I suspect that the new trend high cost of drugs in general is similarly not due to real costs, but is set to the highest price that insurers would pay. It was recounted that this was how Zolgensma was priced.

    • #14568
      Anonymous
      Inactive

      I have an ‘attitude’ about all of this.  So far as Mark Bedwell is concerned, his treatment tells me nothing.  He was new to the disease, and he claims to have improved.  I don’t know how he was before the treatment.  Time will tell.  Let’s see how he’s doing in a year or two.  And if Nurown requires multiple treatments as I have read, who is going to pay for it?  Insurers?  Maybe, maybe not.  300k or 400k is a bitter pill to swallow for anyone, especially multiple times.  I recall the famous Rabbi and the video of almost a miraculous turn of events for him.  I have not read anything since about him and I’m curious about how he’s doing.

      The bottom line is that if it requires ongoing treatments, do you want to take the risk of numerous lumbar punctures  even if it were free?

      I believe the Phase 3 trial should continue until properly finished and evaluated before the FDA (or others) grants approval. I, for one, am waiting to see what happens with it.

      There are a few oral meds on the horizon which seem to have some merit, AMX 0035  being one.

    • #14575
      Dagmar
      Keymaster

      I agree with you Kasper. We need to keep asking questions, and not take at face value “breathless” announcements and viral videos.

      Where are the Phase 1 patients, and how are they doing now? Same goes for Phase 2.

      This happened with Radicava – – a rush to market, only to find out the test cohort was on pALS in 1-2 year of ALS and with low symptoms. Only 140 subjects! But everyone wanted to try Radicava…come to find out it was expensive, required an injection portal and, had a strenuous injection schedule. Many dropped out citing poor results.

      I would rather wait until a product is fully vetted. Risking unnecessary surgery, exposure to infection, side effects and stress from fighting insurers is just not worth it. So far, riluzole, exercise, mindfulness and focusing on meaningful projects have helped slow my symptoms down: https://alsnewstoday.com/2019/11/19/formula-wellness-simple/

    • #14577
      Anonymous
      Inactive

      Exactly!  No one divulges information about the phase 1 and 2 patients.  Perhaps the Radicava info you mentioned (above) will say to Pals WHOA, wait a minute.  I think I can somewhat relate to Pals sense of urgency for something, anything that may provide them with a chance. The whole Radicava thing to my mind is suspect. I didn’t know many people discontinued it, but I don’t follow news of it since I’m not interested in Radicava at present.  Mitsubishi and Treeway supposedly have an oral form that has greater bio-availability. I haven’t heard about those in quite awhile.  I can walk a limited amount with my afo’s now. Radicava’s info states it may cause gait disturbance.  I asked my Neuro about that and all he said is “people with ALS have gait disturbance”.  That tells me nothing. I was questioning whether using it would lessen my ability to walk.  I thought about Radicava in all of it’s aspects and decided it would do more harm than good, since they say you won’t notice any difference. Who knows, maybe it does nothing worthwhile at all. I was not willing to mentally and physically put myself and my loved ones through that regimen of infusions and surgery to have a port implanted.  I seriously considered the DPS also, the surgery, the benefits and risks.  That was another that was not worth doing.  Infections and stress of any type need to be avoided.

       

    • #14579
      Michael Stedman
      Participant

      First of all, I am grateful for the education and perspectives provided within this forum.  Many years ago, a casual friend of mine became afflicted with ALS and I saw him once or twice in the months preceding his demise and death.  A year ago, I was reintroduced to ALS when my wife was diagnosed with it.  Having a spouse with ALS is certainly a life changer and I have sought to educate and inform myself so as to be of real assistance to my wife.  I follow multiple forums, which I would presume many pALS and caregivers do as well.  Some may be characterized as more militant, but to my mind all are working toward the day ALS has been drastically altered in its symptoms or even cured!

      Not intending to be offensive in anyway, but it seems relatively obvious that NurOwn is not anything remotely related to a final cut in research.  However, in terms of the “hope” factor, NurOwn has stimulated such hope.  Based upon review, there is not just one person who has benefitted from NurOwn, there reportedly are several if not many people who have experienced positive results.  (See prior Nightline reporting, reporting from ALS advocates No More Excuses, etc.  It was expressed in this stream that researchers should focus on ‘stopping progression’ – it appears NurOwn has done just that and more.  Not only stopped progression, but allowed some to experience improvement from their prior physical limitations (e.g. standing, pulling in a clutch on a motorcycle and actually riding a motorcycle).

      With that in mind, there was a recent meeting between the FDA and Brainstorm Cell Therapeutics (NurOwn) regarding the possible fast tracking of NurOwn for approval by the FDA going back and seeing what can discerned up to this point in time regarding NurOwn efficacy.  Hurrah!  Quite frankly, when I hear people express that is might be risky or dangerous, an ALS patient such as my wife could care less!  How many ALS patients will die in the next year waiting for a possible effective treatment?

      Regrettably, the almighty dollar factors into the equation as one could anticipate and expect. That is little comfort to the thousands of ALS patients fighting this horrific disease.  Yes, there is a balance to be achieved knowing there are not unlimited resources.  However, we all can appreciate that veterans suffer a disproportionate percentage of ALS patients in this country (about double as I understand it).  I was glad to see DOD is attempting to earmark more research dollars for ALS for its impact on the veteran community.

      I was not part of the Radicava inception process and was unaware of the hope it generated in the ALS  community.  As I understand it, in all honesty, Radicava and Riluzole likely slow the progression some, but at varying rates depending upon the individual.  That’s not exactly what could be called real hope!

      Again, grateful to be part of the dialog and of course my thoughts are my own and I am certainly desire to hear the opinions and thoughts of others.

    • #14580
      Anonymous
      Inactive

      I’m a veteran and have been living with ALS for 13-14 years.  Nurown may make a difference but if numerous people have truly benefited, why isn’t that played up more than it has been?  And who has it had as patients—newcomers to the disease process, usually 18 months or less.  While I wish them well, where is the efficacy for the rest of us?

      It’s legitimate to question both the safety and efficacy of Nurown or any other treatment.  The FDA needs to make that decision ASAP.  Nurown has been in the works for 10 to 11 years.  One would think that by now that a conclusion could be reached about it. The famous Rabbi was a few years ago.  I’m still curious.

      No one wants to stick the neck out for fear of the numerous costly lawsuits that may ensue.  While it is fine to say that an individual with ALS couldn’t care less about the safety of a treatment, other parties to the treatment need to do so.

      If and when it becomes available I will give it a try and hope for the best.

    • #14581
      Michael Stedman
      Participant

      Kasper, first thank you for your service to this country.  I appreciate your weighing in and making your perspective known.  I am in no way going to characterize you as a lucky one, but the fact you have lived with ALS for 13-14 years makes you an outlier in the world of ALS patients. Based upon some commentary, it may be likely NurOwn helps some significantly and others not so much.  That may be the dilemma with respect to why it’s not being played up more (some speculation freely admitted here, based upon inference).  Also, I am inclined to be optimistic given what the disease is doing to my wife and the thousands of other brave ALS patients fighting the good fight under difficult at best circumstances.  My wife and I pray regularly for her situation and others across the globe who too are suffering from ALS.  Kasper, may God bless you and all those afflicted with this awful disease.

    • #14582
      Anonymous
      Inactive

      Michael, thank you for your kind words.

      If it were to improve my condition by 20% and slow or stop it I would be grateful.  I plan for another ten years at least, and that would make the remaining time easier for me.

    • #14596
      Amanda
      Keymaster

      Kasper and Micheal, thank you so much for sharing your stories and information. The information you share will benefit so many others on this forum.  Please keep us posted on how you are doing. Also, we welcome our members sharing the information (and their experiences) they find and what they think about it.

    • #14633
      Bev
      Participant

      Hi all, intetesting topic. Im hopeful for approval by the fda for nurown. Due to the cost I doubt the insurers will cover. Thats too bad for all of us. As far as administration of the cells I hope they Brainstorm tests the delivery to the exact part of the brain using mri focused ultra sound. That may be more effective than injecting to the spinal chord and may be safer too. Im a canadian citizen so if the fda approves it we will soon follow hopefully.

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