I have a serious case of drug trial FOMO (fear of missing out). I just completed my year-long Tirasemtiv drug trial. I don’t know whether I was on the placebo or active drug. However, I have been invited to join the open-label extension of the clinical trial. That would mean I definitely would have an active dose. Meanwhile, the company that created Tirasemtiv is applying for FDA approval at this very moment. I take all of this information to mean that the medication worked: it preserved strength in the diaphragm, preventing a decline in lung function. In that case, being in the open-label trial is a great opportunity because I will have the drug immediately and keep my lungs from deteriorating.
There is a complication, though. If I join the open-label extension, I will take Tirasemtiv for the rest of my life as a way to research long-term safety of the medication. The open-label extension also requires that I not participate in any other trials. There is a trial coming up in April that I have been excited about, but is it promising enough to give up Tirasemtiv? It would help if I knew how well Tirasemtiv works, but I don’t think that is clear yet, even to the research team. I do know that throughout the study, my lung function did not decline at all. So is Tirasemtiv the safe bet?
Reading the news, it seems that possible cures are being found more and more quickly. Tirasemtiv is a treatment, not a cure. Let’s hop back to the experiment I mentioned that will take place in April. That one might be a cure. How can I turn my back on that? It seems like if I play it safe, I could be excluding myself from something miraculous. On the other hand, if the drug trial in April fails, I will have given up lung protection for nothing.
The original plan was to protect my lungs at all costs no matter the collateral damage, and that way, when the cure comes, my vital functions will be strong enough for me to properly heal. And what is this collateral damage? It is pain. For the past year, I have chosen pain in order to be in this trial. Being on Tirasemtiv means I cannot safely take Zanaflex, the medication that completely erases my spasms and muscle cramps. Instead, I am on a cocktail of a narcotic (Vicodin), a controlled substance anxiety medication called Clonazepam, and the muscle relaxer Baclofen. Evan also massages Bengay all over my limbs when my cramps get bad. Plus, I have a sizeable stash of medical marijuana (60% CBD) that is also working to loosen my joints and muscles. That is the price of my involvement in the study, and I will continue to pay it if I join open-label.
Just like the structure of this narrative has spiraled into dizzying circles, my thoughts are a tornado. It hops throughout my imagination, stirring up awful and wonderful scenarios. It rips through my sleep, and there is no seller with Aunty Em waiting to make it all better. I’m Dorothy out in the storm dreaming of Oz with no idea how to get there.
How I Live Now: Life With ALS 
I came across your blog and i couldnt stop reading. I take care of my husbands grandmother who has ALS. Your hope is contagious. Your bravery is beautiful. Literally. You are beautiful. As long as you write, i will continue to read. Thank you so much. I needed this. Love, from Michigan.
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Thanks so much for the kind words! Let me know if you ever have any questions or topics you want me to address. I hope you know that the work you do as a caregiver is the most beautiful expression of selfless love.
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Wow…Thank you, Rachel. I am going to print, and frame these, and hang them up. I run a B&B, so hundreds of people will see them. This is a great thing, honest, clean, brave.
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Hello… you give me hope… it comes and goes. Your article has helped me today. Do you mind sharing the treatment you thought would be a cure in April? Have you researched Brainstorm – stem cell theraphy? Im following them and they seem to be making great strides.
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Hearing that I gave you hope has my heart lit up like a Christmas tree! Hope can be hard to hold onto, but I don’t think we can stay sane without it. I had my eye on NP001, but I haven’t heard anything about it in a while, so I am not sure how human testing is going. I’m no longer eligible for trial participation it ends up. I have heard that the stem cell therapy you mentioned is producing phenomenal results. I don’t think the cure is a question of “if” anymore, but rather “when” 🙂
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Oh you have made my day!!!! NP001 is also something I have really been interested in and I am going to duke university and seeing the top neurologist and he has mentioned in NP001 as well I stem cell. You make me feel like I can’t type fast enough! I too totally believe it is not a question of ‘if’ but ‘when’ a cure is coming. And Dr. Bedlack at Duke tells me he’s never been so excited. It’s so hard to not fall into the dark pit of being afraid. I yo-yo back-and-forth between that and keep telling myself to believe and keep my faith in God. I still feel like I’m going to this for reason to help others. To show them to not give up. This has been especially hard for me because I’m a fitness instructor !! I have been so active all my life and I love always having a lot on my plate to do. I did all of our landscaping even owning my own tractor and now my legs will not hold me up. But I’m determined and I can’t tell you how wonderful it is to speak with someone like you. Thank you thank you thank you !! I hope we can continue to encourage each other . I have to admit when I got the first email and went to confirm my address it wanted me to join some organization which I’m sure is OK. But I was just hesitant to put my name out there yet on another website that will send me emails . So to receive your email has just been thrilling that you contacted me anyway !!!
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Something tells me we are going to be friends. Keep in touch! Rachel.doboga@gmail.com
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