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    • #17024
      Dagmar Munn
      Keymaster

      Many members of this forum are tracking the progress of AMX0035 as it makes its way through the drug approval system. News and announcements are coming in from many sources. Let’s use this “topic discussion thread” to collect news on this proposed new ALS treatment, your questions about it, and any other thoughts related to AMX0035.

    • #17026
      Dagmar Munn
      Keymaster
      • #21997
        Stephen O’Neill
        Participant

        Hi Dagmar, hope you are well and still using your bubble machine?

        I have an LVR kit now too which helps you pump air in to your lungs to expand them and stretch the muscles around the lungs and its helped enormously with my lung capacity, i just thought i would mention it as its easy to use and very helpful.

        I am trying to find out if AMX0035 is on the market yet? there are so many articles ad so many things being said, its hard to find a definitive answer, do you know anything?

        Kinds Regards,

        Steve

        • #21999
          Dagmar Munn
          Keymaster

          Steve – – yup, I’m still using the bubble bottle. 🙂

          Here is what I found on the AMX0035 website:
          “Access to AMX0035 outside of clinical trials is available in the U.S. for certain adults with ALS who meet eligibility criteria for participation in the FDA-authorized Expanded Access Program (EAP) for AMX0035. To learn more about the currently open AMX0035 EAP, visit http://www.clinicaltrials.gov.”
          (https://www.amylyx.com/media/amylyx-pharmaceuticals-announces-launch-of-us-expanded-access-program-for-amx0035#:~:text=Access%20to%20AMX0035%20outside%20of,visit%20www.clinicaltrials.gov.)

        • #22688
          john hamilton
          Participant

          My ALS NP says that one of the EAP qualifying criteria is that you must have contracted ALS at least three years prior!

          Can anyone confirm or refute that?

        • #22555
          Sonsu
          Participant

          Hi Steve,

          What is LVR kit. Are you using NIV.  Is LVR kit in addition to NIV?  How does it help you.

        • #22556
          Stephen O’Neill
          Participant

          Hi Sonsu, hope you are doing the best you can in our circumstances.

          I use an NIV* at night, it took some getting used to and getting the pressures right, but i wouldn’t (couldn’t) go back now. Help with drooling issue, just a few tips i have found useful before i go on to the LVR Kit. I use a neoprine chin strap to keep my mouth closed, play around with the tightness, i use chapstick to help keep my lips sticking together, i made cotton pieces to match the rubber on the mask cushion to help seal it and ease skin irritation, and regularly change the head straps and mask cushion as the cushion changes shape and the head straps stretch making it difficult to get the mask sealing, hope that helps.

           

          The LVR*, is a small hand held pump, you put the hose in to your mouth and pump air in to your lungs to expand them to counter the effects of the MND (ALS). I helps keep lung capacity up which allows more oxygen in and more energy as a result, i have found it helpful for me, but like most things with this disease, you never really know how helpful it is as you only have anecdotal evidence and its hard to compare treatments. i try everything i can though, for obvious reasons.

          I don’t know what part of the world you are in, but if you email me, i will put you on to the respiratory specialist who gave it to me and you can see if you can arrange one.

           

          [email protected]

          Moderator comment: *NIV is Non-invasive ventilation is the use of breathing support administered through a face mask, nasal mask, or a helmet. Air, usually with added oxygen, is given through the mask under positive pressure; generally the amount of pressure is alternated depending on whether someone is breathing in or out.

          LVR is Lung Volume Recruitment. video here: https://www.youtube.com/watch?v=_S6Aux10iGE

        • #22566
          Sonsu
          Participant

          We are in US in New Jersey.  My husband is diagnosed with ALS for little less than year now.  He used NIV at night and during day when he naps.  Hence was interested if anything that can improve lung functioning.

        • #22573
          Esmeralda Trinidad
          Participant

          Stephen

          I forgot to mention that I live in North Carolina, USA.

          I have sent you an email from my personal email and I hope that you can help me obtain an LVR.

          Thank you for your support

        • #22638
          JohnW
          Participant

          I’ve learned the FDA advisory committee gave 7 to 2 approval recommendation on Sept 7. Final FDA ruling is Sept 29

    • #17029
      Kathy stitz
      Participant

      When will AMX0035 be available to ALS patients?

      • #22636
        Laurie Orfe
        Participant

        I have been taking the meds for a few weeks as my neurologist was able to prescribe it offline. The liquid med is difficult to take, it burns going down so I take it in custard style yogurt and drink something warm after swallowing. I’m supposed to take it twice a day but find that difficult. I’m also taking herbs prescribed by a licensed acupuncturist which I find very helpful. Ask your doc if he would Peres rube offline until FDA approval. Hope this helps!

      • #22689
        john hamilton
        Participant

        I think the more critical question might be, When will insurance cover the AMX0035? Maybe a good six months after FDA approval? And now with Fed’s ability to negotiate drug prices, wonder how much longer that will delay insurance approval!

    • #17036
      Duane
      Participant

      Here is a link to a very meaningful article about AMX0035, including some of the history behind it.  I know one of these compounds has been used in Traditional Chinese Medicine for centuries.

      https://www.alzforum.org/therapeutics/amx0035

       

    • #17071
      Jens Buss
      Participant

      Hello everybody

      amx 0035 is availabel allready … you just have to buy TUDCA and you need a doctor for presciption of

      Ammonaps which is the trading name for Natriumphenylbutyrate.

      I am doing exactly this medication since about 4 weeks now and I still feel good, no perceptible change in progress of the disease and no side effects.

      • #22690
        john hamilton
        Participant

        IS “Ammonaps” covered by insurance in the states?

    • #17072
      Jim
      Participant

      Hi, I’m new to the forum, but have been reading a few articles from the forum over the last month, very informative! I’ve had ALS for two years now, it’s been difficult for all of us! I’m glad I can still walk abit (with walker) and have the use of my arms and using noninvasive ventilation at night now. Staying positive and not stressing out is so important! I wanna get to the point/topic. The past 2 months I’ve been taking an array of supplements, TransResveratrol, Curcumin, Glutathione and Co-Q10 etc. During my research/reading I came across some information about Amx0035 (Sodium Phenylbutyrate) and Tudca (Tauroursodeoxycholic acid). Tudca is inexpensive and overall easy to obtain, but the Sodium Phenylbutyrate is very expensive (1,000-5,000usd). I read an article which claims there is a possible substitute for Sodium Phenylbutyrate. I will put a link below! The article/study says Sodium Benzoate (inexpensive/10.00usd and easily found) can be a substitute or can be mixed with Sodium Phenylbutyrate of course lowering costs. If this is true, more people should know about it and of course I could be wrong or my theory is wrong, but wanted others to look into it. Please read the article and look into the two drugs used in Amx0035. Do you think Sodium Benzoate can replace Sodium Phenylbutyrate effectively? Wanted everyone to be aware of above info and please research it! Opinions are welcomed.. Wishing everyone all the best and stay strong!

    • #17073
      Jim
      Participant

      https://grantome.com/grant/NIH/R03-HD078447-01                                                                     Above is the link to a Study with Sodium Phenylbutyrate (used in Amx0035) and Sodium Benzoate. The results of the study are on the page toward the bottom, click. If the link doesn’t work, I’ll repost it! Keep in mind, many of these drugs are recycled and can treat several medical issues, as the above study dealt with hyperammonemia. Best Regards, Jim

      • #18462
        Isabelle
        Participant

        It says “Conclusion: Phenylbutyrate was more effective than benzoate at disposing of nitrogen. Increasing phenylbutyrate dose may not result in higher nitrogen excretion due to decreased conjugation efficiency at higher doses. Combinatorial therapy with phenylbutyrate and benzoate has the potential to significantly decrease treatment cost without compromising the nitrogen disposal efficacy”…I guess that is why we stay with SPB as a preferred choice …. the “may” has not been investigated nor tried as far as I saw …sadly !… I am wondering if TUDCA by itself is not as efficient as combined with SPB at slowing down the ALS symptoms progression…I am reluctant to take that SPB which was said to be neurotoxic …. any thoughts on this?

         

        • #22692
          john hamilton
          Participant

          Per https://www.alzforum.org/therapeutics/albrioza: “In a clinical study conducted 10 years ago in Milan, Italy, TUDCA alone reportedly had a treatment benefit in ALS. In a Phase 2 trial in 34 ALS patients, treatment with 2 g per day for one year slowed deterioration on the ALS”

          But I also read somewhere that while TUDCA showed relatively little benefit, the two compounds TUDCA and phenylbutyrate, which comprise AMX0035, have a synergistic effect, multiplying the (expensive) phenylbutyrate benefits by an order of magnitude.

    • #18469
      Jim
      Participant

      If there is any article/trial which states Sodium Benzoate can be neurotoxic, I’d definitely stay away from Sodium Benzoate! Looks like Tudca and many other supplements have shown ‘Promise with slowing progression! Best stick with the supplements that have some solid foundation within trials and light exercise (my opinion)! Thank you Isabella for your input! All the Best!

    • #18500
      Suzanne Gallo
      Participant

      My PALS neurologist at Penn ALS clinic gave him a script for Sodium Phenylbutyrate. It was over $1000 copay to fill at Walgreens. He has Medicare and AARP UH supplemental and AARP UH pharmacy benefits. I was advised to appeal our insurers decision not to cover Sodium Phenylbutyrate. Ask for a second opinion on the denial from a neurologist- making sure that neurologist knows how close AMX0035 is to being approved by the FDA……

      anyone have any luck appealing coverage of sodium phenylbutyrate?? We could sell our house, but, idk- where would we go?

    • #18522

      I think it’s interesting to note that two letters were sent to the NEJM after the AMX0035 results were published.
      https://www.nejm.org/doi/10.1056/NEJMc2030710

      The authors of the second letter to NEMJ affirm that only TUDCA is effective and therefore that the dual therapy proposed by Amylyx has little interest. The principal investigators’ response is that these are studies with different methodologies and therefore no conclusions can be drawn.

    • #18891
      Dagmar Munn
      Keymaster

      Webinar: An Update on the CENTAUR Trial: a Trial of AMX0035 in ALS

      Monday, May 17, 2021
      2:00 – 3:00 PM EDT
      To register: https://register.gotowebinar.com/register/7277752541137339663

      Presenters:
      Sabrina Paganoni, MD, PhD, Sean M. Healey & AMG Center for ALS at Mass General Hospital.
      Merit Cudkowicz, MD, MSc, Sean M. Healey & AMG Center for ALS at Mass General Hospital,
      Please these two presenters for a conversation about the results of this trial and the next steps for AMX0035.

    • #20244
      Tomasz Boski
      Participant

      hmmm, I am on TUDCA for 3 years……..not so many benefits

    • #21731
      Dagmar Munn
      Keymaster

      4/1/2022

      Trial Data Not Enough for AMX0035 Approval, FDA Committee Votes – – read article here: https://alsnewstoday.com/news-posts/2022/04/01/fda-committee-votes-against-recommending-als-therapy-amx0035/

      Message from the ALS Association:

      The Institute for Clinical and Economic Review (ICER) is reviewing Amylyx Pharmaceutical’s AMX0035 and Mitsubishi Tanabe Pharma America’s (MTPA) oral edaravone. Their review will make a “value assessment” on these new therapies which will impact decisions on cost and access made by private and public insurance such as Medicaid at the state level and Veteran’s Affairs at the federal level. ICER’s value assessment uses methodology which has been called discriminatory by the National Council on Disability, a trusted advisor of the President and Congress.
      Please read a blog here that provides more details. In the blog, Daniel Cramer, associate director of public policy initiatives, shares his thoughts on the ICER review through the lens of his family’s experience navigating ALS. Daniel shares what “value” meant to his mother and how value assessments—like the ones ICER uses—do not take patient and caregiver views of value into consideration.
      Sign the petition to tell insurers and policymakers to ensure people living with ALS have immediate, full coverage and affordable access to new therapies.

    • #21734

      It seems to me that “Target ALS” is one of the few ALS organizations that does not claim any intellectual property over the research it funds.

    • #21736
      Duane
      Participant

      We all want something to address this disease, but I fail to understand what all of the commotion is about. Perhaps it MAY extend life by 6 months but if you are in the advanced stage of ALS you may not want to live longer. It may slow the progression which is good but apparently 6 no’s is meaningful,  at least for me, binding or not.

      There is a Phase 3 trial that I think has merit.
      Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS
      https://clinicaltrials.gov/ct2/show/NCT03800524

       

      • #21739
        Amanda
        Keymaster

        Duane,

        I understand your opinion and your post response. My father felt similarly regarding ALS treatments. He did not want to live longer because of his quality of life being poor in his opinion. He did not seek medical attention or take anything to address ALS or any of the symptoms. The only time he accepted medical assistance was when he wanted Hospice in the picture. That was in 2013.  He had taken care of his father, and helped with both of his sisters and my cousin (all who had ALS).  He clearly knew what to expect and made his choices being well informed at the time.

        I’m not suggesting this is the right path for every pALS, or the only path by any means. I know that each pALS is different and their needs are different.  Being that I am in a pre-fALS study, I tend to look towards and focus on the research that could possibly help to identify ALS earlier using biomarkers and target preventing the onset and/or stop the progression of the disease regardless of the stage.  Those are some of the areas that the pre-fALS study is targeting.  All of the research is important and like all of us in our community, I want everything that will help all pALS at every stage easily accessible and affordable.

        Amanda

    • #21740
      Duane
      Participant

      Yes Amanda, anything that will slow it and of course anything that will stop it entirely. So long as the cure is not worse than the disease. I think it is initially caused by a virus that alters the genes that causes one to be susceptible to the cascade of neuron events that lead to this.

      • #21741
        Nina
        Participant

        I have to agree with you, Duane. For those of us who are in the late stages of ALS it’s hard to cheer for any treatment that will prolong our lives. I hope that those of you who are in earlier stages and are doing the research will find the right path for you and help future pals live longer and better lives.

        • This reply was modified 7 months, 3 weeks ago by Nina.
    • #22001
      Sandra mcleod
      Participant

      I would like to know if people saw results after taking amx0035 and how long after taking it did they see any improvement

    • #22007
      Bill
      Participant

      I think you just have to believe the study results forAMX 0035. The improvement is small enough you need the study., I don’t believe it to be enough that people could tell the slow progression change. I would not trust opinions positive or negative. Hopefully phase 3 willl bring stronger results. I’ve read so many reviews of Radicava and Riluzole where people said that couldn’t tell if they helped but studies report they do.

    • #22008
      Sandra Isaacsoln
      Participant

      My physician contacted the company administering the EAP program. This is the first paragraph of their response. ” Thank you for your patience. Amylyx are only able to support a limited number of sites for this Expanded Access Program and so we recommend referring your patients(s) to one of activated sites that are published on ClinicalTrials.gov [clinicaltrials.gov] for treatment. Please also note there are a limited number of patient slots available through the Amylyx EAP prior to potential commercial launch availability as well.”

      Not very encouraging. I also scanned the FDA Advisory Committee’s 50 page report. There is a short summary in the April 1, 2022 edition of GlobalData Healthcare. The bottom line seems to be that the study results to date are not statistically significant. I’m also a bit concerned that the articles in the healthcare literature note that the studies reported on are funded by Amylyx.  For 20 years I worked in Federal research organizations. We always thought that research studies funded by the organization with a vested interest were not necessarily the best studies. Hopefully, that is not the case here. I have been very hopeful that AMX3500 does slow down progression.  I have been taking 1000 grams of TUDCA twice a day. I have know way to tell if it is working. But, I have no side effects because I use it, so I take it and hope.

    • #22014
      Vance
      Participant

      I have been a participant in the clinical trail  of AMX 0035 since 2018.  Below is the public testimony I gave to the FDA at the hearing.  My response to AMX0035 may not be typical, however I believe there were other case similar to mine.  

      I was diagnosed with ALS in December 2017. I have been a participant in the Centar Clinical Trial since March 2018.

      At the time of my diagnosis in the 2017 I was experiencing great weakness in my arms and with my hand grip strength. I was having hand tremors that required 2 hands to hold a cup of coffee.  Dressing was extremely difficult, I needed assistance to pull up my pants, buttoning and zipping them required help or adaptive tools.   I could not get my arms up high enough to put on T-shirts by myself.  I was also experiencing muscle twitches in my lower back upper arms left front neck and occasional drumming in my ears.

      Walking had become extremely difficult I required a wheelchair to get to my appointments throughout the Mayo clinic during my diagnosis in December. I was fitted with a brace at that time to help address the extreme case of foot drop.

      Upon my return home I was put on prescriptions of Riluzole and  Radicava.  I was asked by my neurologist if I would be interested in participating in a clinical trial for a new drug. I agreed and began the trial in March 2018. My first strength  assessment found my grip strength in my right and hand of 18 pounds my arm and leg strength was extremely low 32 pounds arm flexing was extremely weak.

      I had to stop working in my store as I I no longer have the strength or stamina to stock shelves or help customers.

      I had starting to notice an improvement in my strength and mobility by June 2018. In August I built a 100 square-foot deck digging post holes pouring concrete cutting lumber and attaching the deck. My wife and I began to travel again and I no longer needed a wheelchair to get around airports although I  was still using the brace at that time.  And by the end of the year in 2018 hours again able to work overseeing the daily operations of my business.

      This drug has greatly improved my quality of life and that of my wife, children and grandchildren. Prior to the outbreak of the pandemic we were regularly traveling, we have walked many miles touring Europe,  China and Tibet.  Two years before I would’ve never thought this to be possible.

      My health and strength seem to stabilize although I’m not back to 100%. My hand grip strength is now back to normal for a man of my age. I am fully independent and capable living a quality life. AMX 35, for me has been a life-changing drug.

       

      • #22015
        Mike
        Participant

        Vance, do you have any video footage of the way you were before and after taking AMX?

    • #22016
      Dagmar Munn
      Keymaster

      Vance, thank you for sharing your experiences and your FDA hearing testimony with us. I wonder if these questions were ever posed to you:

      – You began taking Radicava and Riluzole shortly after being diagnosed in December 2017.
      – A year later, in March 2018 you began AMX0035, along with continuing Riluzole and Radicava.
      – You began noticing strength improvement 3 months later… and built a deck for your home 2 months later. (5 months since starting AMX0035).

      How can you attribute the positive changes to only AMX0035? Could it be possible that the combination of the 3 drugs worked synergistically? In only 3 years you went from weakness and needing a wheelchair to walking independently, traveling and trekking China and Tibet – – remarkable, indeed.

    • #22552
      Stephanie
      Participant

      Hello! I just joined and am finding these forums very helpful. My husband was diagnosed in July 2022. My question is with all the clinical trials has anyone heard anything on the Treggs trials?

      • #22642
        Lisa Brown
        Participant

        Me too!  Dagmar – can we have a separate thread for Treg therapy please?

      • #22671
        Amanda
        Keymaster

        Hi Stephanie,

        I’ve read a little about the Tregs clinical trials online. I believe it is looking at cells in an pALS’s body that don’t perform properly (removing blood and separating) and disrupt the immune system, specifically causing (?) or not preventing inflammation in the body.  I can’t remember the exact details.  Did you have specific questions?  I can try and find the articles and post the links.  I’m sure one was on the government’s clinical trial web page.

        Amanda

      • #22672
        Amanda
        Keymaster

        Hi Stephanie,

        I’ve read a little about the Tregs clinical trials online. I believe it is looking at cells in an pALS’s body that don’t perform properly (removing blood and separating) and disrupt the immune system, specifically causing (?) or not preventing inflammation in the body.  I can’t remember the exact details.  Did you have specific questions?  I can try and find the articles and post the links.  I’m sure one was on the government’s clinical trial web page.

        Amanda

      • #22673
        Amanda
        Keymaster

        Hi Stephanie,

        I’ve read a little about the Tregs clinical trials online. I believe it is looking at cells in an pALS’s body that don’t perform properly (removing blood and separating) and disrupt the immune system, specifically causing (?) or not preventing inflammation in the body.  I can’t remember the exact details.  Did you have specific questions?  I can try and find the articles and post the links.  I’m sure one was on the government’s clinical trial web page.

        Amanda

    • #22588
      Maureen Williams
      Participant

      I’ll be starting Albrioza problem this week! We shall see how it goes.

      • #22600
        Maureen Williams
        Participant

        That should read probably, not problem, and I’ll be starting to take it next Tuesday.

    • #22635
      Dagmar Munn
      Keymaster

      LATEST NEWS!!!

      “Amylyx Pharmaceuticals Announces FDA Advisory Committee Supports Approval of AMX0035 for the Treatment of ALS”
      https://www.amylyx.com/media/amylyx-pharmaceuticals-announces-fda-advisory-committee-supports-approval-of-amx0035-for-the-treatment-of-als

    • #22650
      Dagmar Munn
      Keymaster

      And for the other side’s opinion… here is an interesting article:

      “The FDA Is Rushing a New and Unproven ALS Drug”

      FDA and its advisors are skeptical the drug works as well as the company purports, and clear evidence of its efficacy won’t be available until 2024 at the earliest. Still, the agency is widely expected to formally approve the drug as soon as later this month. That sets up a bad precedent not only for future treatments for ALS, but for other diseases.

      Read more here: https://www.bloomberg.com/opinion/articles/2022-09-08/the-fda-shouldn-t-rush-an-unproven-als-drug?leadSource=uverify%20wall

      Do you agree or disagree with this opinion?

    • #22658
      Bill Osullivan
      Participant

      I found the article you shared about FDA rushing approval of AMX0035 very interesting.  I Read the comments that Bryan Trayor of NIH voted to approve and was interested in the company talking about removing from market is history does not support slowing down progression.

      Dr Trayor and his team were interested in our family gene mutation and they are working with us collecting biopsy and blood from my children and siblings who tested positive for our family defective gene. I spent an entire day with him and was impressed with his confidence in future treatments to slow ALS.  I am taking riluzole and Radicava ORS with no side effects, I have no hesitation adding AMX0035. All medications that have potential to slow progression are welcome.

    • #22664
      Jerry Fabrizio
      Participant

      Bill,

       

      Which genetic are you?  I am C9orf72.  My mother and cousin died of this disease.  Time for us to eradicate it!

      Jerry

    • #22674
      Bill Osullivan
      Participant

      Hello Jerry, Our family has the CHCHD10 variant c.44G>T (p.Arg15Leu).  We lost our mother and 4 of her brothers and sisters to ALS.  I have 8 brothers and sisters some have tested for the defective gene and are positive including my son & daughter.  I am the oldest at 65 and the only one currently with ALS degradation. Agree it is time to find a cure or slow down the progression for the next generation.

    • #22802
      Dagmar Munn
      Keymaster

      Relyvrio (AMX0035) Approved in US for ALS Treatment

      Relyvrio (AMX0035) Approved in US for ALS Treatment

      Looks like AMX0035 has been approved by the FDA.

      What do you think of this news?
      Is this a treatment you would want to try?

      • #22805
        Dagmar Munn
        Keymaster

        Although it’s great to have another ALS treatment join Riluzole and Radicava… I agree with Carolyn that at the price of $158,000 per year, AMX0035 (oops, Relyvrio) is unbelievably expensive.

        All three medications slow symptom progression – – so why not choose the cheapest one? Less hassle with insurance coverage and availability.

    • #22803
      Carolyn Barry
      Participant

      I would try it if it were affordable. I highly doubt that it would be, just like how oral Radacava turned out, once it was approved.  It is so wrong for these drug companies to not help ALS patients with the super high cost of their drugs.

    • #22818
      Mam Sadek
      Participant

      I am newly diagnosed with ALS and my Central FL ALS Clinic put me on AMX0035 compound 5 capsules twice per day .
      after one month, lost my right arm by muscles atrophy. Weakness now is all over my body.
      I don’t believe Amx hasn’t revealed all components intentionally as a fixed-dose combination of two compounds — tauroursodeoxycholic acid and sodium phenylbutyrate —

      Mam Sadek

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