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    • #13539
      Dagmar Munn
      Keymaster

      If there is any doubt that earnest ALS research is occurring, this list of current investigational ALS treatments and therapies proves that it is indeed happening. (read their descriptions here: https://alsnewstoday.com/experimental-treatments-als/)

      • BHV-0223
      • P.Acthar Gel
      • MN-166
      • GM6
      • AMX0035
      • Gilenya
      • IONIS-SOD1Rx
      • Arimoclomol
      • AT-1501
      • NP001
      • VM202
      • Reldesemtiv
      • Masitinib
      • NurOwn
      • NSI-566
      • CNS10-NPC-GDNF
      • Mexiletine

      Which ones are you most interested in learning more about? If you had to make a wager, which one do you think will be the first to receive approval for use in ALS?

       

    • #13645
      Diana Belland
      Participant

      Dagmar, thank you so much for posting this list of current investigational ALS treatments and therapies.   I’ve read about a few of them over the past six months and am planning to try to learn more about each of them.   I’m most aware of Ibudilast and NurOwn but I want to learn about all of them.

      My guess is that BHV-0223 may be the first to receive approval for use in ALS since it is a sub-lingual form of Riluzole and is nearing completion of Phase 3, I believe.   When I saw my neurologist at the Cleveland Clinic on July 10, 2019, he asked if I would like to participate in the Phase 3 trial.   I happily agreed, but was later contacted by the CC to say that the trial was no longer accepting new patients.

      I’m looking forward to reading what other forum members have to say.

       

    • #13691
      Diana Belland
      Participant

      An article which appears today, November 5, on the home page of ALSNewsToday has intriguing information about MN-166 (Ibudilast):  https://alsnewstoday.com/2019/11/05/patent-application-for-ibudilast-as-treatment-for-als-approved-in-canada/

      A Phase 2b/3 trial has recently been launched:  https://clinicaltrials.gov/ct2/show/NCT04057898.   I’m interested in the possibility of participating in this trial, based on the results that “ibudilast significantly improved patients’ functional activity, quality of life, and muscle strength, compared to a placebo. ”

      MN-166 has been granted fast track and orphan drug status by the FDA.  Perhaps this will be the first to receive approval for use in ALS?

       

    • #13704
      Dagmar Munn
      Keymaster

      This is an update on AMX0035:

      Podcast: “What to know about AMX0035”

      In this episode of the Endpoints, Dr. Steve Perrin, CSO at the ALS Therapy Development Institute, sits down to talk about AMX0035, a drug consisting of a combination of two small molecule compounds: sodium phenylbutyrate and tauroursodeoxycholic acid (TUDCA). Amylyx, the sponsor of AMX0035, recently completed enrollment of a 132 participant phase 2 trial. SIGN IN TO LISTEN 

      SUMMARY of PODCAST:

      “AMX0035 is a combination of two small molecule compounds: sodium phenylbutyrate and tauroursodeoxycholic acid (TUDCA). Sodium phenylbutyrate is a pan HDAC inhibitor and is approved by the FDA to treat urea cycle disorders and TUDCA is bile acid naturally produced by the liver and is not approved by the FDA for any marketing use. AMX0035 recently completely enrollment of a phase two trial in mid 2019.

      The trial, also known as the CENTAUR trial, was conducted by Amylyx and was a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of AMX0035. Even though the trial is currently fully enrolled, there is an ongoing open label extension arm, to assess the long term safety of the drug. Sodium phenylbutyrate has been described as a potential treatment for ALS for more than a decade based on preclinical results from rodent models of ALS. Unfortunately, other labs including ALS TDI have not been able to reproduce these results.

      Early clinical trials of the components have been done in Italy, South Korea and elsewhere by groups other than Amylyx. When tested separately in those clinical trials, the use of the compounds individually was found safe and well tolerated in people with ALS. However, no clinical trials examining these molecules to date have found them to show efficacy in people with ALS. This trial, is the first combination study of the two together in people with ALS. Amylyx decided to pursue AMX0035 following a high throughput screen and found this combination of sodium phenylbutyrate and TUDCA to be the most neuroprotective.”

    • #13708
      Bev
      Participant

      Hi Dagmar,

      You didn’t mention MRI focused ultra sound trial for ALS at sunnybrook hospital in Toronto On Canada. They have proved they can open the blood brain barrier temporarily to deliver stem cells or drugs to the exact area of the brain. I’m hoping brain storm Therapeutics will use that method in their trials someday. I was diagnosed with PLS so don’t qualify for any trials but I’m hoping to benefit from nurown some day.

      thanks for all you do for to inform us.

      Bev

    • #13706
      Owen
      Participant

      Just based on phase and study completion date, I’d put these five up at the top of the list for potential approval:

      In terms of the ones I’m most excited about, I’d say NurOwn and Ibudilast. There’s also a Phase III trial of ultra-high dose methylcobalamin (B12) in Japan that should be publishing results in the next six months: https://clinicaltrials.gov/ct2/show/NCT03548311

    • #13722

      Hi all,

      I wrote a book about the state of ALS research. It includes sections about the main achievements, the main drugs that are studied, a possible genetic therapy with current state of art and glimpses on future therapies:

      https://www.amazon.com/dp/1698147899

      My intention is to keep its content up to date as research progresses.

    • #13727
      Dagmar Munn
      Keymaster

      Congratulations Jean-Pierre! We haven’t heard from you for a while… now I know what kept you so busy!

      I’m sure your new book will do well!

    • #13736

      Many thanks Dagmar!

    • #15686
      DAVID
      Participant

      Hi Jean-Pierre

      Thank you for your work ! Do you publish a french version ?

       

    • #15687

      Thanks David, I really appreciate.

      There is a French version, it is also available as an ebook :

      Initially I planned to publish an updated version in October. But so far 2020 had not been very prolific on the ALS research front. I look forward at the results from the clinical studies of Arimoclomol and NurOwn, so maybe the updated versions will be available in February 2021, hopefully with good news.
      I am also happy that in 2020 ProMIS neurosciences designed intrabodies against TDP-43 granules. I am still waiting for biotech to propose a genetic therapy against TDP-43 cytosol granules. This is not a scientific or technical challenge, it is essentially a funding problem.

    • #15762
      Sara
      Participant

      Is anyone taking oral methylcobalamin? It seems that the research is really only around IM delivery. Is there any utility to oral methylcobalamin? And if so, what dose?

       

      Also, as to TUDCA, which seems quite interesting, can anyone recommend a brand? I feel quite in the wilderness when it comes to supplements and I do worry about the quality/contaminations.

       

      THANK YOU!

    • #15764
      Dagmar Munn
      Keymaster

      For the sake of our forum members who are not familiar with chemical names:

      Methylcobalamin is B12 – – most commonly used to treat anemia.

      TUDCA is Tauroursodeoxycholic acid (TUDCA) and is a bile acid derivative that has been used in Europe to treat cholelithiasis and cholestatic liver disease.

      Sara – – did you know that this is what they are used for? Do you feel they have any impact on the condition of ALS?

    • #15766
      DAVID
      Participant

      Thank you Jean-Pierre !

    • #15775
      Sara
      Participant

      Hi Dagmar, thank you for your help in framing my questions.

      1. TUDCA: yes, there is some data that PALS lose function more slowly with this supplement vs. PALS taking placebo. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5024041/  It is available OTC. I would suspect that some PALS on this forum are taking this, and I’m hoping to get a recommendation on brand.

      1. Methylcobalamin is a type of B12 (most OTC is cyanocobalamin). In my (quick) perusing, it seems that IM is the only studied route. https://jnnp.bmj.com/content/90/4/451

      I am curious is anyone is aware of any data supporting oral administration. I can’t find any, so it seems one would not want to take oral B12 supplements for ALS (of course there are other reasons to take B12)

      Thank you very much!

       

       

    • #15777
      Dagmar Munn
      Keymaster

      Some more information on TUDCA: https://alsnewstoday.com/tudca-tauroursodeoxycholic-acid/

      Regarding B12 – – I’ve always heard that oral supplements are not the best way to go (and a waste of money) – – so intra-muscular (IM) injections are the preferred delivery method in order to benefit from the dose.

    • #15783
      Anonymous
      Inactive

      I took very high doses of both forms orally of B12 for about 6 months.

      Here is a good article (2020):  https://ods.od.nih.gov/factsheets/VitaminB12-HealthProfessional/

    • #15795
      Lisa Bonahoom
      Participant

      Dagmar, what have you heard about BIIB078 from Biogen for genetic ALS?  C9orf72.  I start the open label next Friday.  I have high hopes!   Lisa

    • #15796
      Dagmar Munn
      Keymaster

      Lisa, I am not familiar with that one (I don’t have familial ALS)… perhaps Amanda knows about it. Either way, good luck! 🙂 Be sure to check back and share with us how you are doing.

    • #15800
      Lisa Bonahoom
      Participant

      I was chosen to be one of 12 people worldwide to be in the third cohort last year. I only had a 50% chance of getting the drug, but I really think I did.  I gradually grew stronger, spoke more clearly; friends and family noticed.  When the dosing stopped last November I slowly started getting worse again.  I will receive a higher dose, with no chance of a placebo next Friday!  The dosing is intrathecal, not fun, but I really hope for evidence of slowing or stopping progression, or even reversing the path a little.  I will let you know!

    • #15801
      DAVID
      Participant

      Hi Walter

      Do you believe high doses B12 had an impact ?
      My Dad had injections last year. Unsure if it/how much it helped

    • #15802
      Anonymous
      Inactive

      Hi David,

      It’s a gamble.  No way to tell for sure, but I gave it and algal DHA a try. Costs a little but what the heck!

    • #15804
      Anonymous
      Inactive

      Lisa,

      I hope it works for you.

    • #15805
      Debra Kaufman
      Participant

      Regarding AMX0035, I have been trying to find out where the Stage 3 trials are at. There has been radio silence since the end of Stage 2 trials in December last year. I wrote Amylyx directly but received no reply – there is probably some embargo. I’ve heard that Stage 2 trials were very positive and the company was asking for compassionate use release.  I have an MD/Ph.D. friend who works for a pharmaceutical company – he has done work in ALS before and is interested, so he’s trying to do some research for me on what’s going on with AMX0035 – if he does, I will post it here. If anybody else has any news, I’d appreciate hearing about it!  Many thanks!

    • #15810
      abraham
      Participant

      A few hospitals in Canada are starting a clinical trial with the oral administration of Radicava (edaravone). Apparently the side effects associated with intravenous/IV administration of the drug is eliminated.

      I am curious to see if anyone in this group has enrolled for this study in USA and how they are feeling after starting the trial? Thanks

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