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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.

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