• Amanda

    Member
    December 9, 2022 at 6:31 am

    Updated:

    I was recently diagnosed, this past Friday, and this treatment was discussed because it targets the specific mutation that runs in my family. We’ve lost 15 family members to ALS, the last being this past February. I also have the mutation.

    I’ve been volunteering for medical research since 2010; however when the clinical trials started for Toferson, I did not meet the criteria for the trial. I was asking so that I could gain insight and information to help me make decisions about my future health care.

    Toferson was supposed to be reviewed by the FDA in January; however, Biogen submitted additional information to the FDA. The company put out a press release saying the the review date has been extended until the end of April.

    I will have to apply for extended access in order to be considered for the treatment. I was hoping that some of our members could provide me with information. I have learned since positing that many times when people participate in clinical trials they are legally obligated not to share or discuss information until the drug has gone through the FDA process.

    Amanda

    • john-ceriotti

      Member
      December 28, 2022 at 4:37 pm

      Amanda

      I started as one of the last participants in the “closed” portion of the study and then was allowed on the extended access or open access portion of the study.  I just received my 20th dose on Dec. 13th.  Myself and my wife (an RN) are very happy with the results of the Tofersen drug.  20 doses ago (one every 4 weeks via Lumbar Puncture) I was unable to lift my right arm above my head and continued to lose strength in my lower extremities.  I am very pleased with the results to date and actually started physical therapy at the Washington University Orthwein Center Sept 26th.  My results have been good to very good with regaining strength and the ability to perform many more physical things without having to tremendously focus as before the doses.  I do things physically without thought just as I did prior to my diagnosis.  I am still not walking but continue to gain more and more strength across all body parts.  In fact, my breathing capacity test increased to 48% from 38% over a 3 month period and tested on Nov. 13th or so.  my 27 year old son who lives out of town is just in awe and what I can do …like transfer from my chair to a PT mat, or from my chair to a toilet/shower chair or even at night when I transfer myself from my chair to my recliner without any assistance from others.  I feel stronger than I did 2 years ago.  Really I do! I am very pleased with the results and will continue to go to PT 2X per week looking for some more tangible results in the next 3 months…targeting the 6 month mark or so to be able to walk on my own power using a walker to start!  Get the drug , I believe it is worth the monthly LP until they come up with a preferred delivery method.  Email me with any other thoughts or questions…[email protected]

  • les-wood

    Member
    December 13, 2022 at 4:32 pm

    Hi my name is Les Wood I was diagnosed in August 2012.i am on the Toferson treatment I am just starting my seventh year on it.I am the first person in the UK to be on the trial,it is now classed as a treatment,I feel the drug has helped me a great deal,I have endured around 75 lumber punctures during this time as of course that is how the drug is administered.there are I believe ten of us on the treatment which is at Sheffield Hallamshire Hospital.the staff there are brilliant and very professional.If any one wants any imformation on tis subject I am only to happy to help all I can

     

    • Amanda

      Member
      December 14, 2022 at 11:24 am

      Les,
      Thank you so much for the information. Has it been approved as a treatment there? Tofersen was supposed to be reviewed in January byt the FDA, here in the United States. Biogen provided additional information so now it is expected to be reviewed in late April. my doctor’s have applied for early access to the treatment on my behalf. They are hopeful that will be approved and I could expect to start the treatments in Jan/February.

      How do you feel the following couple of days after the treatments? Are you getting them every 4 weeks or what is the schedule.

      I’m so happy for you and that you feel the treatments are helping you. I had no idea it had even been available anywhere for 7 years!! Any breakthroughs will help the medical professionals figure out how to help all pALS!

      Amanda

      • tina-johnson

        Member
        December 15, 2022 at 7:43 pm

        Hi Amanda,

        I’m also interested in the tofersen treatment. Can I ask you what state you live in? Also where are you being treated at?

        Tina

      • john-ceriotti

        Member
        December 27, 2022 at 4:51 pm

        Tina

        Just to assist although the question was to Les…I am in StLouis and being treated at Washington University Medical Center where the creator of the drug Dr. Timothy Miller is the Chair of the department with his PI Dr Robert Bucelli who is Vice Chair!!

      • tina-johnson

        Member
        December 29, 2022 at 12:01 pm

        Thank You, John

        Sounds awesome what it has done for you.

        I am 40 and walking with a cane, I can’t lift up my left arm very high anymore and now it’s starting in my right. I am also doing PT and exercising at home as much as possible.

        I talked to my Neurologist and they put me on top of the list for Tofersen. I am just praying I am able to get it.

        Thank you for posting!

        Tina Johnson

         

      • john-ceriotti

        Member
        December 31, 2022 at 11:49 am

        Tina

        Any supplements as I do take (7) to help with body health!

        My wife who is an RN-BSN did a bunch of research 2 1/2 years ago when I was diagnosed and found a good amount of info concerning what supplements showed any type of positive or non-harmful results.

      • tina-johnson

        Member
        January 4, 2023 at 11:03 am

        Hi John,

        Would you be able to let me know what the supplements you take? Either on here or email?

        My email is [email protected]

        Thank you!

  • suzanne-gallo

    Member
    December 16, 2022 at 9:01 am

    Amanda

    i am so very very sorry that you have been diagnosed with ALS. Please know that I am extremely sad. I have been following your column sincd my husband was diagnosed about 3 years ago.
    my father passed from ALS 6 years ago. Not familiar, but super lousy, just the same.
    I suggest you contact Ron Hoffman, at CCALS.org. Please do this, to broaden awareness of patient, family, and caregiver care.
    you must do as you wish, clinically, but as far as genuine care for you- and, your caregivers, please reach out to CCALS.
    They have been the world to my PALS and I.
    suzanne

  • les-wood

    Member
    February 8, 2024 at 4:05 pm

    Hi fellow Toferson friends this is just an update or refresher if you like my name is John Wood though I am known as Les after my middle name Leslie also my father Les .Any way as I said earlier I was diagnosed in August 2012,though I I’m sure I had mild symptoms a few years before that.I am in the U Kand have my treatment still at Sheffield Royal Hallamshire Hospital every 28 days I am obviously the longest Toferson survivor in Britain and quite possibly in Europe due to the fact thr I was the first person in Britain to receive the drug I must have been on Toferson for around 8 years or so now.Like I said some time ago to begin with well certainly when I progressed to the maximum dose of 100 milligrams my health improved quite dramatically Evan appearing on the BBC national news.im now aged 70 and sorry to say that I believe the illness has overtaken the treatment.I can no longer walk,or even stand or weight bare without a wheelchair I am completely bed bound my only outing is my treatment day at the hospital and a occasional day at the horse racing to see one of my horses ( share of )race my wife drives me there and I get transferred to my wheelchair.im sorry to post such a negative review but I can’t see any point in glossing things over.Also no two persons are the same so some may benefit more than others If any of you want more detailed imformation on the Toferson treatment just reply to this message and I will endeavour to get back to you

    • Amanda

      Member
      February 14, 2024 at 8:11 am

      Les-Wood,

      Thank you for your update, directness and honesty. I have heard there are some situations where a person’s body builds up a resistance to the medication and it no longer has the same impact. I’ve also heard an instance where someone had meningitis and had to stop for 3 months, and then start back up with half a dose to see how their body responded. As with everything, there are major risks. It is great to hear from someone with extended experience on Tofersen. Sharing your information is very helpful, so please continue to do so.

      My doctors have always stressed it is a treatment, not a cure. They also stated that people will respond differently based on the SOD1 variant (rapid or slow) and how much damage has already occured. I know here in the US, it is still in clinical trials and was conditionally approved by the FDA. Hopefully they will continue to make strides in the area research for all kinds of ALS.

      Les, thank you again for sharing. You really are a wonderful source of information.

      Warmly,

      Amanda

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