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    • #15947
      Steve Gillan
      Participant

      Hello, like all others I am looking and hoping to find anyway to slow this down.  My question is has anyone used off-label drugs? I was reading the article last week about Clinigens aldesleukin which is a cancer drug. I have also seen in the past about AMX-005 in a Phase 3 trial – I think.  Aldesleukin is FDA approved for cancer.  AMX-005 is a combination of two separate FDA approved drugs used for different treatments.  Since there is nothing else we can do for our love ones is the problem just getting a doctor to prescribe the drugs off-label? The afore mentioned drugs have been through their trials therefore we know the dosage, side effects etc….  I asked my wife doctor about prescribe the two AMX-005 drugs but she would not do it. It seems to me if a drug is showing promise in trials and we already know the side effects and ethnicity from their original FDA approval process why can it not be taken?  Is it just a matter of finding a doctor that will prescribe it?

      Sorry if this is stupid but it is driving me crazy not being able to do anything to help my wife from getting worse. I myself was a 911 first responder and ended up with leukemia.  I was the first in the country to be in a trial for a new approach to stem cell replacement.  So far it has worked for me – I need something to work for my wife.

    • #15979
      Danielle Uskovic
      Participant

      Hi Steve,

      I’m from Australia and here it is very difficult to get off-label drugs. We do have a special access scheme where doctors can apply for use of a drug not approved, but there is a bit of a process to it.

      I’ve read on some Facebook ALS forums that some doctors in the USA will prescribe off-label drugs but you have to spend a bit of time looking for the ones willing to do it.

      Good news for you is that iamALS dot org is campaigning congress to pass Two bills in the USA to grant faster access to new therapies. This will speed up getting the therapies showing promising results in trials.

      Not sure if you’ve checked out any of the current trials but iamALS has launched a new platform where you can easily find trials in your area. It’s a brilliant resource and worth checking out. You can find it here

    • #16158
      Simeon
      Participant

      Hi Danielle,

      I’m not sure that is correct in Australia. Any GP can prescribe off-label. The thing you can’t do is get the cheaper prices via the PBS.

      Thanks

      Simeon

    • #16163
      Steve Gillan
      Participant

      Thank you for the info Danielle,  I am familiar with the pending legislation in congress.  I joined the “Steven’s Nation” effort to try and push congress.

    • #16164
      Mike Minardi
      Participant

      My wife has been taking Sodium Phenylbutyrate and Tudca since January. While Tudca is available on Amazon, you do need a script for Sodium Phenylbutyrate. She currently takes 6g of SP per day (12 500 mg tablets) along with 2G (4 500 mg capsules of Tudca) per day. Her neurologist at Mass General / Healey Center prescribed the SP. Insurance will not cover it and it is expensive, but you can get a discount through GoodRX. you need to go through a specialty pharmacy such as CVS Specialty. We have seen no side effects and while Kathy’s ALS was already far along, she has remained stable. Find a neurologist who will prescribe it. In our case, Kathy is fortunate to have the Director of the Healey Center as her neurologist, Dr. Merit Cudkowicz.

      Good luck!

    • #16168
      Danielle Uskovic
      Participant

      Hi Simeon- thanks for the information. I know only certain drugs are available on PBS. My neurologist won’t prescribe some off label that I’m interested in. He made it sound difficult. That said, I may take your advice and see if my GP will do it instead. Cheers.

    • #16172
      Simeon
      Participant

      Hi Danielle – I provided the research papers to our GP. It also helped that they were pre-existing drugs with long history of safety and well understood, for example triumeq and tecfidera.

    • #16192
      Steve Gillan
      Participant

      Dear Mike,

      I read your response, thank you.  I am curious on the dosage – I guess your doctor at MG gave you the dosage for the SP.  How was the dosage for Tudca determined.

    • #16193
      Jane Kriskey
      Participant

      I found Tudca on Amazon and then did some research and to me is seems like no risk in taking it. My daughter goes beginning of Sept to her doctor at Duke and she is going to ask him about his thoughts on it. But I think she should take it as it is not harmful and why not? The Right to Try is really nothing because the drug companies DO NOT have to give it to you and they won’t because they will want to make millions on any drug so why would they give it out and if they did it would be so expensive you could not afford it. It is the drug companies that need to be reigned in. I wrote 8 drug companies, 16 hand written letters, to the co’s and the coo’s of the eight companies. Got no responses. Not even a form letter to acknowledge receiving my letter. Not enough money in ALS.

    • #16194
      Steve Gillan
      Participant

      Hi Jane;  Mikes comments ”

      My wife has been taking Sodium Phenylbutyrate and Tudca since January. While Tudca is available on Amazon, you do need a script for Sodium Phenylbutyrate. She currently takes 6g of SP per day (12 500 mg tablets) along with 2G (4 500 mg capsules of Tudca) per day. Her neurologist at Mass General / Healey Center prescribed the SP. Insurance will not cover it and it is expensive, but you can get a discount through GoodRX. you need to go through a specialty pharmacy such as CVS Specialty. We have seen no side effects and while Kathy’s ALS was already far along, she has remained stable. Find a neurologist who will prescribe it. In our case, Kathy is fortunate to have the Director of the Healey Center as her neurologist, Dr. Merit Cudkowicz.

      Good luck!

    • #16196
      Marianne Opilla
      Participant

      Google the TUDCA and ALS and the AMX0035 studies.  The dosages and results are in several articles. My neurologist says the data is great on this drug. It is held up somewhere for now.

       

    • #16197
      Debra Kaufman
      Participant

      Hi Steve: Right after my cousin was diagnosed with ALS, I started researching treatments and found the AMX-005 possibility. I was put in touch with someone who has been taking this off-label for his ALS. He got the Tudca online and the other drug via the European Union (long story and his to tell).  Although he knew someone who reportedly had really good results from this, he said that he had not had any noticeable results.

      I became kind of obsessed with the status of AXM-005 since it had been through Phase 3 clinical trials, apparently successfully. I emailed the company and had no reply. I even enlisted the help of a friend who is a doctor working in the pharmaceutical industry. He found some of the research and said that the lack of forward movement after the trials could indicate bad side effects or many other possibilities. So it remains a mystery.

      Meanwhile, my cousin hasn’t shown a strong interest in trying this off-label, so I’ve let it rest. Good luck. I know other people are pursuing this off-label. If you do, please share your wife’s experiences.

    • #16208
      Paul Tavano
      Participant

      Find a doctor familiar with treating ALS. They tend to understand the desperation of patients and caregivers. They also stay up to date on trials so will be more familiar with the drugs involved and more open to helping. At least that has been my experience here in TX.

    • #16218
      Mike Minardi
      Participant

      Hi Steve, just saw your question on dosage. Yes, the dosage was ordered by Dr. Merit Cudkowicz at Mass General. We mix 6 500 mg SP tablets with 2 capsules of 500mg Tudca twice a day, morning and evening. She takes quite a few different scripts (all ordered by Merit Cudkowicz), each one backed up by a research study. All with the hopes of slowing this down. My wife actually has the C9 mutation and we are hoping some of the therapies in the pipeline will arrive sooner rather than later. If you would like to discuss, please feel free to give me a call, I am in Illinois and my mobile is 630.768.8616

    • #16234
      Jerry Manning
      Participant

      Good morning. Mike, would you be willing to share the off label meds your wife is using. We are still hoping for the Healey trial but also want to have alternatives if this doesn’t happen. Currently I am only taking basis and then going to try nuedesta off label to see if it helps swallowing. We try to keep up on the trials and research but it’s difficult.  Our neurologist doesn’t really suggest other things to us so this would provide a starting point for discussion. Thanks so much!

    • #16281
      David Crellin
      Participant

      I was diagnosed 3/3/20 with C9ALS. I have friends in genetic therapy research,several are medical consultants, and I have siblings in medicine. So lots of contacts to come to my aid. I gave up trying to persuade my consultant to prescribe the treatments I had researched: Metformin, TUDCA, Ibudilast. He admitted they probably had more evidence than Riluzole, but he wouldn’t prescribe even though the cost of Metformin and TUDCA is negligible.
      So, TUDCA available on Amazon -kershing £££- and I put the word out. A mysterious package from Singapore arrived with 360 Metformin tablets. Who sent it? No idea. But a mate who is a thoracic consultant and medical director now knows I want Ibudilast/MN166. I await developments.
      It seems it’s both what one knows and who one knows that counts.

    • #22355
      Bruce Black
      Participant

      My wife has also been taking sodium phenylbutyrate plus TUDCA since January.  The phenylbutyrate is prescribed by her MGH neurologist, Dr. Ho, and comes from CVS Specialty Pharmacy, and we get the TUDCA from Amazon.  The doses are the same as what were used in the AMX-0035 trials, 6 grams of sodium phenylbutyrate and 2 grams of TUDCA daily, taken in divided doses AM and PM (6 tablets of sodium phenylbutyrate 500 mg and two capsules of TUDCA 500 mg in the AM and the same in the PM).  The phenylbutyrate is expensive but fortunately my wife’s insurance, Massachusetts BCBS Medicare Advantage PPO, is covering it, despite it being an off-label indication, and did not require a prior authorization.  (I’m guessing that many insurance plans may not cover it for an off-label indication, and I was suprised that it was so easy for us to get it covered.)  She is also taking riluzone and recently switched from Radicava IV infusion to the new oral suspension.  We have not noticed any adverse effects of any of these medications.  Under her insurance plan, these four drugs (sodium phenylbutyrate + TUDCA + riluzole + oral Radicava) are costing us about $22,000 per year, and would cost $280,000 without insurance coverage.   The insurance was covering the Radicava home infusions 100%, including the drug, all supplies, and any required nursing visits, with $0 copay.  Unfortunately, the oral form is covered under the part D benefit.  Since we have already passed the annual “catastrophic” cut-off, we are now paying $653 every 4 weeks (5% of $12,700) for the oral Radicava.  The TUDCA costs us $1,500 per year and the insurance does not cover it at all.  She also takes Tru-Niagen 300 mg twice daily, which has not been studied for ALS but was recommended by another friend who is an aging-brain research scientist.  This costs us another $800 per year.  (I am also taking TUDCA and Tru-Niagen, even though my 68 year-old brain seems to be entirely healthy as far as I can tell.)

      As a “supplement”, TUCA is not FDA regulated, and the purity and dosing accuracy of supplements are notoriously unreliable.  (A laboratory study of 25 different over-the-counter preparations of St. John’s Wort some years ago showed that some contained no St John’s Wort at all and some had many times the stated dose.)  There are quite a few different TUDCA products available on the internet.  The Nutricost brand was recommended to us by a friend of a friend who is a leading aging-brain scientist at MGH/Harvard and takes it himself for brain health.  There are cheaper brands out there, but I think it’s worth a little extra money to have more confidence that we’re probably getting what we think we’re getting.

      I see many testimonies in these forums that “this drug is helping me (or my loved one)” or “that drug is not helping me”.  Although that may be easy to determine with respect to medications for specific symptoms, like hyper-salivation, muscle spasms, or pain, it is probably nearly impossible to determine if any specific treatment is helping any specific individual with respect to the progression of ALS disease and disability.  The progression of the disease is too highly variable from one individual to another and even over time, the rate of progression is too subjective and difficult to measure, and there are no treatments yet that seem to have dramatic (i.e., very large) effects on the rate of progression or to halt or reverse the progression.  All we have to go one is that randomized, double-blind placebo-controlled trials of riluzole, Radicava, and the AMX-0035 combination seem to slow progression and prolong survival for individuals taking the active medication compared to placebo.  (My wife and I are both retired physicians.  We are not ALS specialists but we do understand how medical science works.)  The evidence seems to me to be stronger for some drugs than others.  My impression is that the evidence for AMX-0035 is much stronger than for either riluzole or Radicava.  We’re guessing that taking these three treatments may prolong my wife’s survival by 12 to 18 months.  On the other hand, given that ALS is probably many different diseases with a common symptom manifestation, and that all of the drug studies are too small to provide statistically meaningful breakdowns even between known disease subgroups (much less between different underlying pathophysiologies that we don’t even know about), it is quite possible that some PALS get no benefit from any of these treatments while others may get much more benefit than the study results seem to demonstrate.  (For example, if 50 PALS taking placebo in a given study survive an average of 12 months, while 50 PALS in a one subgroup take the study drug and survive an average of 24 months and 50 PALS in another subgroup take the study drug but receive no benefit and survive an average of 12 months, and we don’t have any way of identifying these subgroups, the study results will show that PALS taking the study drug survive an average of 6 months longer that the PALS taking placebo, and there will be no way of knowing that half the PALS got an extra 12 months and the other half received no benefit.)

      • This reply was modified 4 months, 1 week ago by Bruce Black.
      • This reply was modified 4 months, 1 week ago by Bruce Black.
    • #22367
      Sherri Morehouse
      Participant

      Not seeing this information posted yet in this thread, so thought I’d share.  For those of you who want to try “AMX-0035” (homemade), TUDCA is straight forward to source (although not the pharmaceutical grade used in the Amylx clinical studies), but the Sodium Phenyl Butyrate is not.  We were unable to get our insurance to cover it, and luckily heard of a distributor in Lithuania where cost is reasonable ($580/40 day supply), and no prescription is required.  They are called CanChema where we’ve analyzed the compound against one obtained via a US prescription with a perfect match.   Many other PALS have also verified.  Discount code “pba20off” gets you down to the $580 for a 250g purchase.  My husband has been using this with TUDCA for 10 months along with many other things.  He’s not progressed much at all, just slight slurring.

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