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?
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