Without starting this piece off on a completely morose note, I’m going to say probably one of the most revealing things that I’ve admitted on the written page before: I’ve had to learn — from an early age — how not to get my hopes up. That doesn’t mean what you think it does though because as many of my longtime readers are aware, I’m actually a pretty optimistic person. I just have to practice it differently: by being cautiously optimistic, as an act of self-preservation.
It’s a sentiment that many people with chronic illnesses can relate to, but it’s one that is not really discussed openly. And as usual, I make it my own responsibility to ram the door down on topics that I don’t feel are talked about enough.
To set the scene, cautious optimism is something that I’ve had to practice almost daily in the past two months.
The third week in September, I received two pieces of disappointing news in the span of two days. The FDA rejected two medications for my condition. The first medication, which is called Apitegromab, was one that I’ve talked about before. It would be administered via IV monthly. Since it’s an infusion, it wouldn’t be terribly invasive, but it would have to be given in conjunction with Spinraza. To attempt to put these medications’ purposes in layman’s terms, Spinraza has to be delivered past the blood-brain barrier in order to work on the missing genetic component at the cellular level throughout my whole body, whereas the new medication would work solely at the muscular level.
The second medication that was up for approval was a higher dose of the Spinraza medication that I’m currently getting. There is only one dose of the medication that everyone can receive, which is 12 mg. The new dosage that was supposed to be approved was for 28 mg, over more than twice the amount that I’m currently receiving.
The good news is both of the medications were encouraged to resubmit once they fix the initial issues identified by the FDA. However, that resubmission process could likely take months, not even taking into consideration the amount of time it will take the FDA to actually review the medications again.
It’s ironic, in a way, because since my mom and I go to Boston for all of my appointments, we are very aware of the cutting-edge medications on the horizon, given that Boston Children’s is a very prominent research hospital for these medications. My doctors had begun talking about the Apitegromab medication over six years ago, and they were incredibly optimistic about it. Even during my last appointment with my team, more than a year ago, they were absolutely certain that the FDA would approve the medication because the results were overwhelmingly positive. One of my doctors was more leery about the increased dose of Spinraza because of the complications that could likely arise from it.
Personally, I was more excited about Apitegromab because of the amount of motor strength that I have lost over the last 10 years. It is one of my deepest desires to be given the chance to regain some of the strength and independence that I have lost.
And I was hoping that I would be able to work towards achieving that wish sooner rather than later.
Both of these medications had the potential to help me tremendously because while the current dosage of Spinraza is helping, somewhat, to sustain my current level of health, it isn’t working like it was once was. Point being, my health since my last dose, at the beginning of August, has been on a continuous rollercoaster, with me doing better, then randomly declining.
Between medication changes and ongoing issues with my equipment, it has been a struggle to breathe clearly most days without feeling like I was drowning in the rivulets of saliva running constantly down the back of my throat, which my weak throat muscles cannot manage.
This being said, I have been trying, in vain, to catch my footing on increasingly unsolid ground for the last two months. And I know my body needs something else, medication-wise, so part of me really needed the reassurance that there was something on the horizon to help me. There is a part of me that was absolutely devastated upon hearing this news. But the more rational part chooses to be cautiously optimistic that these instances were just delays, not immediate no’s.
My hopes were not crushed by this news. Because I know, deep in my soul, that eventually a new medication will be approved. I will just have to bide my time until it is.
Gazette columnist Joanna Buoniconti is a freelance writer and editor. She is currently pursuing her master’s at Emerson College. She can be reached at columnist@gazettenet.com.