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Trying a New Treatment that looks Promising!
Hello to our wonderful online community!
I usually stick to my professional role when posting on our forums; but, today I wanted to share some personal information regarding my ALS journey.
I’ve been volunteering for ALS research for over a decade. One of the studies I’ve been participating in is called pre-fALS which stands for pre familial ALS. As you know from my welcome message and previous posts, I have a SOD1 mutation and I’m the 16th person in my family to have ALS. Wowzer!!!
In December I was told by the research team that it was official, I have ALS. I was shocked to hear those words, yet at the same time I knew it was coming! They wanted to get me started on Tofersen which was expected to be reviewed in January. The FDA delayed the review of the drug, and my request for access through the compassionate care laws was also side tracked– I won’t go off on that tangent right now. But, let me tell you that I was more frustrated than I’ve ever been in my 56 years of life. It was finally reviewed in late April 2023 and it was conditionally approved by the FDA. It took several months to get the drug ready for commercial use and approved through health insurance. I ended up waiting from December 2, 2022 until my first treatment on July 12, 2023. It was a scary time because I was having a difficult time breathing until I finally got a non-invasive ventilator which I use mostly at night.
After months of waiting, and substantial declining, and struggling with the stress and with my mental health, I finally started Tofersen. It is now known as QALSody, and my first of three biweekly initial dosings was on July 12th! I still have one of the three initial biweekly dosings to go, on August 9th, and then I’ll get the treatments every 28 days.
The doctor on the research side thought QALSody would halt the progression, AND I may even gain a few things back! He said I would never be 100%, but that is to be expected. Now that is a scientific miracle in my book, although others might think that calling something a “scientific miracle” is more along the lines of an oxymoron. 🙂 I have just always thought, and said, “Sometimes miracles come in the form of science.” So, I’m hopeful and trying to maintain a positive attitude because I know mental health and perspective play a critical role in our physical health.
So the downside, yes, I dare say downside to all this ….. well, I suppose it is something along the lines of a type of survivor’s guilt perhaps. I don’t want to compare my experience with a combat veteran or anything inappropriate. A little over a year ago I lost my cousin, Bruce, to ALS. I was heartbroken. I have lost so many family members and friends to ALS. Several years back I lost my father — I wonder why couldn’t this have been available then? At times, I am reluctant to share too much information about my QALSody treatments on our forums because I know that I am in a small group (1 to 2 % of people with ALS/ around 300 people in the USA) that this treatment will help. It is still blowing my mind. I’m sure you understand—I am thankful that I am in that small group, but I wish that all of us had access to a treatment that is likely to stop the progression of ALS.
I am definitely grateful for this opportunity and this treatment. Please know that I get how fortunate I am, and my friends and family know that too! I will use this gift to continue fighting for pALS, cALS and advocating for more research and treatments.
If anyone is interested in how my treatments are going, what they are like, or if I am actually improving I would be happy to share as I go along. If anyone has questions about my ALS journey and the treatments, I’m an open book! And I am dedicated to helping anyone I can on my journey. I pray that the QALSody works they way the expect it too! If not, I am grateful for any additional time and or increased quality of life it affords me.
I know that research is ongoing, and one primary focus is trying to find a similar treatment for the C9orf mutation, along with others. The hope is that all of this information and research will lead to treatments for everyone with ALS.
I’m thinking about starting a blog to document my experience. Is that a good idea?
Warmly,
Amanda -
40 Replies
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Hi Amanda,
Halving the progression rate for the SOD1 mutation is a definite win for QALSody! Whether it be a scientific miracle, God’s miracle, or just the hunch of a researcher, be thankful, celebrate, drink wine, and dance in the streets! We all cheer you on because a win for you is a win for all pALS in the puzzle of ALS.
I think of myself as a small part of the 10,000-piece three-dimensional puzzle that it is. You were in the right place at the right time to try a promising treatment. Your data may provide a tangent of thought for researchers to pursue a cure for the myriad of variants! Each piece of the puzzle has profound hope as the beast takes form. Each piece is another bit of knowledge, and with knowledge comes a cure.
Please don’t go down the guilt trip path of destruction. It doesn’t help you, me, or any other pALS. It will distract you from advocating for the cure and dampen your positive attitude.
By all means, start a blog right here on ALS TODAY. Possible names: “Amanda’s Journey with SOD1”, “Amanda’s Piece of the Puzzle,” or “The Joy, Hope and Grief of Treatment”! Whatever you call it, it will provide perspective and comfort to all.
We thank you for all you do!
Len
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Thank you!! I love those name suggestions!
Amanda
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Hi I was the first person in the U k possibly Europe to be put on the Toferson trial now a treatment I was diagnosed in Aug 2012 ai the Hallamshire hospital Sheffield England.I have had filming done of me and it has been on several News bulletins including the BBC I had my treatment today it went well though to honest it usually does.to begin with once put on the full dose I felt an almost immediate effect and a massive improvement but after six years and nine months,I’m not so good .talk to me I am LES WOOD honest trustful replies will be given,you can check with any doctor at the mnd research team at the hospital
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Les,
What isn’t going good now? How long have you been on Tofersen? Can you share any of the media links? I would love to watch.
I appreciate your willingness to honestly share the good, bad and ugly.
Amanda
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I did find this article on Les. https://www.bbc.com/news/health-62851186
It’s interesting, and positive, although it reminds me that QALSody is a treatment, not a cure.
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I did find this article on Les. https://www.bbc.com/news/health-62851186
It’s interesting, and positive, although it reminds me that QALSody is a treatment, not a cure.
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Amanda, I excited for you with this new treatment and would love to hear all about your journey and hopeful good news with this drug. Sending all my best for you (and everyone on this thread). May your progression halt and may regain function.
Best,Christine
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Amanda, I am so happy you are able to start this treatment!
I think it would be helpful for you to add weekly comments to this discussion topic updating about your experiences and thoughts. This would be the perfect “blog” spot for you.
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Please start a blog! My husband is SOD1 and debating about taking Qalsody.
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Sandy,
I will!
Out of curiosity, what are the pros and cons for your husband taking QALSody? For me, It sounded so promising and I felt I was more likely to have a future. I know with new treatments there is always so many unknowns.
Amanda
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Amanda,
He doesn’t like the idea of the spinal injection. Does want to try it though and is in process of getting insurance override and approval.
Sandy
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Sandy,
The spinal tap aka lumbar puncture is not bad at all!! I’m sure your husband is rolling his eyes to that comment, I did when someone on here said that to me :).
The only thing I really feel is the two shots of lidocaine they use to numb the area. I’ve had 5 so far, two for research and 3 for treatments. There is very little down time too! My back is a little tender at the injection site but nothing too serious. Sometimes I get achy muscles a few days after and I assume it’s related. To be honest, I think the emotional toll played a harder time on me these first 3 treatments. I waited so long to get this approved and started I was just wore out.
Sandy, if you want to talk, or if your husband wants to talk about Tofersen I would be more than happy to share what I know and my experience. I know that each person is different and has different needs. I don’t know if this treatment is a good choice for your husband but I’m here if I can help. No pressure. I’ll send you a private message.
Amanda
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Sandy,
The spinal tap aka lumbar puncture is not bad at all!! I’m sure your husband is rolling his eyes to that comment, I did when someone on here said that to me :).
The only thing I really feel is the two shots of lidocaine they use to numb the area. I’ve had 5 so far, two for research and 3 for treatments. There is very little down time too! My back is a little tender at the injection site but nothing too serious. Sometimes I get achy muscles a few days after and I assume it’s related. To be honest, I think the emotional toll played a harder time on me these first 3 treatments. I waited so long to get this approved and started I was just wore out.
Sandy, if you want to talk, or if your husband wants to talk about Tofersen I would be more than happy to share what I know and my experience. I know that each person is different and has different needs. I don’t know if this treatment is a good choice for your husband but I’m here if I can help. No pressure. I’ll send you a private message.
Amanda
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Definitely start a blog. You have been a voice of reason here and more of that is needed.
One word of caution, when I started having breathing issues, it wasn’t long before I was prescribed the drugs that dry up the saliva to reduce aspiration risk.
Immediately I felt worse in every way but I attributed it to ALS. Walking declined, couldn’t hold my head up and extremely tired daily.
Fast forward to today. I had a laryngectomy to elimination aspiration risk and am off the drugs. I went off the drying drugs a few weeks before surgery and felt like a new person. All the issues reversed and then I realized they were side effects only.
just a heads up in case it helps.
Definitely start a blog. No ALS patient is going to fault anyone that benefits even if we don’t. We all know the villain here is ALS, not fellow PALS.
Doug
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Hi Amanda. Thanks for sharing. God Bless you!
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Amanda:
I’d love to hear how your treatments are going! My husband has sporadic ALS, so Toferson doesn’t apply to his disease, but I’m always ready to sit in someone’s corner and root for them. Any success in treating ALS is a victory for all of us and the tireless researchers trying to find more effective treatments.
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So treatment # 3, the final loading dose is complete!! I’m so excited! I’m still working, walking (slowly) and fully functional. I guess I’m fully functional :). There are some things I can’t do but these are minor compared to what I could face later. I can’t take care of my yard. Things like pull weeds, plant, basically anything that requires me to bend over or carry anything over about 5-10 pounds. When I do, my muscles lock up and spaz. The pain is excoriating. Does anyone else experience that? Afterwards, those muscles are sore as if they are bruised for days. The doctor doesn’t want to give me anything for the cramps and spasms because I/he wouldn’t know if the treatments were helping those or not. Ugh!! I get it, but it’s painful.
I also had a brief neuro exam right before my treatment. I scored -4-5 on everything. 5 is perfect…My scores were mostly -4 to a 4+. The weakest areas are my hip flexors and deltoids. They did not do breathing tests this time and that’s my biggest problem area. I can’t walk and talk at the same time. I get out of breath walking from the car to the house. I’m not complaining, just documenting! I’m thrilled I can even walk 8 months in!
This should be the month that I hopefully see progression slow down or halt! Overtime I will know if i gain anything back. Once interesting note, I can step up and down a curb a little easier this week. I was having to turn to the side and step down, but yesterday I noticed I just easily stepped down a couple of times. After that, I had to walk to the ramp area :). I have stairs at work and my office is on the second floor. I take the elevator. I can go up the stairs once, using my hands to pull a fair amount of my body weight. After one flight, I’m zapped and need my non-invasive ventilator.
They want me to bring my ventilator to my next treatment. I cannot lie on my back and breath. After the treatment they want me to lay flat on my back for an hour. This supposedly helps the treatment. If I have my ventilator on I can lay on my back. The darn thing is heavy though!! I’ll figure it out 🙂
This time my injection site has been slightly tender for about 3 days. I always feel a little achy about 2-3 days afterwards, but nothing bad. It’s that feeling, “Ah, am I catching a cold?”
I choose to continue seeing the doctors in Miami. Its about a 2 and half hour drive. I stay the night in a hotel the day of my treatment. I usually go back and sleep for several hours. This is getting expensive though. Does anyone know of grants that might help with travel and medical expenses for treatments?
If you have questions, just ask!
Amanda
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Amanda
Thankyou for your bravery and inspiring story.I beleive those who have had the luxury of accumulating free hotel nights/ airline miles may donate them to you
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@Community,
Thank you so much for the well wishes and kind words. I feel so fortunate that I am a candidate for QALSody. Know that with every treatment and every appointment I continue to pray for all of us to have this opportunity. I continue to advocate and reach out as much as I can to bring awareness to our cause and the need for treatments for all of us.
Yesterday I was interviewed by a Channel 10 in Miami. On Sept 8, WINK news here in SWFL will be joining me at my next treatment and interviewing the research team. I think there is one more Miami station on the list to interview with soon.
These interviews started when I reached out to local news stations asking if they would get the press release from the FDA when it made its decision on QALSody/Tofersen. I reached out to everyone I could think of because I wanted to hear immediately.
I’ll keep you posted and share the links to the next stories aired.
Amanda
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Hi Amanda thank you for sharing this! I’m just south of Fort Lauderdale and have recently been struggling a lot. I’m wondering what treatments/meds you’ve been doing since diagnosis before this new one and if you found anything helpful?
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Eric,
I wasn’t on anything and I’ve never taken supplements that are supposed to help ALS. I started taking Riluzole in May. I’m still taking that even though I started QALSody. I really don’t know if the Riluzole is making a difference or not. I’m hoping that over the next month or two that I will have some medical test that will measure the neurofilament light chain levels, and anything that will be helpful to understand if either medications are working.
I will keep you informed.
Cheers,
Amanda
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Thank you Amanda! Looking forward to hearing about your progress.
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Latest Interview — I love that the reporter is focusing on the research!!
https://www.local10.com/health/2023/08/25/uhealth-research-may-expand-benefit-of-als-drug/
Cheers,
Amanda
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This is great, Amanda!
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Hi Amanda,
i too have the familial gene that gave me ALS, although it is C9 gene. I’m the first one in my family to get ALS. Fortunately I’m a veteran and VA is taking good care of me.
Daniel
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I too have the SOD1 gene and have been taking Tofersen/Qualsody now for 2 1/2 years. The progression of my symptoms has virtually stopped. I am hoping, Amanda, that the same thing will be true for you. Of course every decision to take Qualsody is a personal one, but I agree with Amanda in response to Sandy that the Lumbar punctures have not been too bad. I have had over 30 of them and plan to continue as long as I continue to see the results I have experienced so far.
I also would love to hear more from Les Wood again about his comment that “after six years and nine months, I’m not so good”.
I am hoping that the science behind Tofersen/Qualsody will just be the start of understanding ALS for all pALS and not just those of us with the SOD1 gene.
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Amanda, we are all pulling for you! Please keep us posted!! Eric
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AKV9/NU-9 has been approved for ALS clinical trials. This is the most promising one I hope. Prayers!
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Mel,
I missed this post!! I have to read up about it!! Anything new to share??
Amanda
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I had my 5th dosing of QALSody this week, and a neuro exam right before the treatment. I was told I had ALS on Dec 2nd through research, had the first official diagnosis and ALS clinic visit May 19th, 2023 and this was my 5th dozing. My diaphragm is the initial site of weakness. (Respiratory onset sucks! All ALS sucks!!) Only, we didn’t relate this to ALS until this past few years. It’s been part of the discussion but it’s much harder to determine, or at least it seemed that way to me. I’ve been getting short of breath for years, and years. Between December and July my Forced vital capacity dropped from 80% to 35/40%. My legs got very heavy and I was exhausted all the time. I came home from work and took a nap every single day — a very long nap.
This week all of the muscle strength test came back the same or better than the previous results from July. Yes, you read that correctly, Some better!!!!! I’m still walking on clouds over this news. My FVC stayed the same!! No decline…. (I was really hoping for improvement but I didn’t feel any difference so I got the results I was anticipating.) The doctor was excited and I could see him smiling as he performed each test. Then when we were able to discuss the results, I was…well, I have no words to describe how I was feeling. Excited, overwhelmed, blessed, relieved, strong…nothing seems to capture that feeling.
I know that QALSody is not a cure. It’s a treatment that looks promising for me, and my other SOD pALS community members. I feel a little stronger and I notice it in things such as when I walk up a flight of stairs without having to use the handrail to pull my weight. I used it for stability :). I don’t have to sit down when I shower. My breathing is the same which can be frustrating at times, but I remind myself that I’m almost a year in and I’m still taking care of myself. I’m still working. I’m still eating. I’m still enjoying my life. I’m still “Me” and I always will be regardless of ALS and what it may take in the future.
I know that many of our community members have progressed much further and faster than I have. I wish I could change that for each of you. I hope that this treatment leads to even more advances to help all of us.
My next appointment is November 1st in Miami.
With respect and gratitude,
Amanda
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Amanda,
That is such good news! A win is a win, no matter how small, and ALS teaches us to celebrate the small stuff!
Len
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Hi Amanda,
We live in Edison, New Jersey, my wife is diagnosed ALS in June 2022 after 2 years of investigation and since then we are struggling to find best care center. We are being treated at RWJ, NJ but her condition continues to decline. Today she cannot walk, talk and is on spoon feeding (semi liquids). We are on Radicava only.
We are at cross roads and unable to understand how to proceed.
We are open for any treatment and I look forward for your advise.
Regards
Ramesh Kodali
Husband of Vijaya Kodali
my email id: [email protected]
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Although I had a very positive experience at my last ALS Clinic visit, they still are recommending that I get a feeding tube soon, next week. It is not because I can’t swallow or anything of that nature. I have no bulbar symptoms. My diaphragm is the most impacted by ALS. My FVC dropped from 80ish (normal) in January to between 30 and 40 by May. It probably dropped that low y the end of February I just didn’t go to the Clinic until May. It really did feel like it dropped significantly in just a week or so. If it drops below 20, I would not be eligible to go under anesthesia at all and the feeding tube may not be an option if and when I need it. They are also concerned that I could take another big drop quickly. The doctors, and I am hoping that QALSody/Toffersen also slows the diaphragm weakness progression and I make some improvements! However, there is very little, if any, information on this from the clinical trials. This being Rare is getting old quickly! 🙂
I have to admit that this step has hit me hard. For my family, not eating was the last and final step in the ALS journey. I associate this with end of life. Intellectually I understand and know that this is not always the case. Each ALS case is different and has to be treated according to each individual’s needs. I know that for many with Bulbar onset, this is just a step and the quality of life is still very good.
It took me some time to wrap my head around this step. I do believe that it is in my best interest to have the procedure. I am still living a good quality life and want to maintain that as long as possible. I cried a little out of fear. However, after a few conversations and time to let all the information sink in, I’m feel like I’m back to my happy and hopeful self.
Has anyone else made decisions like this? What was your process like? What information weighed in the most for you?
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Amanda, this is a big step – – and you are facing it with reality and strength – – however, I read “in between the lines” that you assume having a PEG means no more eating – – quite the contrary. Your doctors are being preventive, and correct that since your FVC has dropped you may not be able to have surgery later on. But, but… even if you have a PEG, you can continue eating… it is just “there.” Many pALS use it for taking a daily liquid, but continue to eat three meals a day by mouth. It’s an adjustment for sure. And it is making you confront your assumptions about ALS and what each change “means.” You are YOU. And I have confidence in your ability to adjust…and move forward with your life. Be well, my friend.
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Hi Amanda,
I have not needed to decide whether I will get a feeding tube. I thought I would have to make that decision soon, as at my neuro visit, using the quick breathing test in the office with the small portable machine hooked up to the laptop, I scored 41%. I was not having shortness of breath but got tired and had to rest during the day. My doctor scheduled a complete workup with the pulmonary breathing test and an artery blood gas test for oxygen and CO2 levels. On the breathing test, I scored 67%, and on the blood test, my oxygen was a little low, but CO2 was in the normal range.
In my case, the quick test was wrong, as it can be for several reasons, so I explored further. You may have had an accurate test and have the correlating symptoms. I did not.
Given your intense family association with ALS, your internal conflict brought to mind an analogy that may help put your mind at ease. I assume you have a car and have insurance coverage to protect that car against the possibility of damage in case of an accident. Most people do not have many accidents over their lifetime but have insurance for peace of mind in case you do have one. Think of it as protection for your car for the future – It is there when you need it, and hopefully you won’t.
The feeding tube is your insurance policy; your health insurance should cover it, so it is free! It is there if you need it. Hopefully, you won’t, but it gives you peace of mind and options for the future!
I agree with Dagmar; you are confronting your assumptions about ALS, which is scary. You will make the right decision for you, and your mind will be at peace.
On a lighter note, If you get the feeding tube, “You will be able to get your cake and eat it too!”
I hope this helps.
Len
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Hi Amanda
Any update about your treatments! How are you doing?
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I’m so happy!! Wednesday I had my 6th QALSody treatment in Miami. They think my ALS progression has plateaued!! It’s on PAUSE!! This is the 4th month of treatment. The doctor said that they tend to see this step around month 6th. I’ve also gained a few small things back. I walked up two flights of stairs last week. I even alternated feet for each step. I didn’t even use the handrail to pull my bodyweight up, just for balance. I was a little winded at the top, but in July I couldn’t step up 2 steps on a bad day. There are a few other things too! I’m so excited. I have to remind myself that this is a treatment and not a cure. However, for now my quality of life has improved!
I keep calling this a scientific miracle 🙂
The doctor did say that this should pave the way to find treatments for both genetic and sporadic ALS! I sure wish I had a timeline to share, but I don’t.
I don’t want to write a book on here, but I did want to share the good news!
Amanda
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Hi Amanda,
That is excellent news! Celebrate the win!
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Hi Amanda,
I am having the worst time navigating the discussion thread. I see your posts and share them with my husband. Thanks !!! Sandy
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Sandy, describe your problem seeing the discussion, perhaps we can help.
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