SMA treatment access remains urgent, even if some patients choose to wait
I'm holding off on trying Isembyld, but it should still be made accessible quickly
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One of my favorite tees to wear is navy blue and says, “INSTALLING MUSCLES. PLEASE WAIT …” Underneath the text is a bright red loading bar about halfway done.
It was a gift from my mother, a token of her hope that I’d one day grow stronger instead of weaker. At the same time, it was an ironic inside joke for me and anyone who knew I live with SMA: That loading bar could never reach completion, as I could never install new muscles for real. I’m no cyborg, no self-bettering artificial intelligence. We would forever be waiting.
That is, until cutting-edge disease-modifying therapies became available to treat SMA.
First, we had Spinraza (nusinersen), Zolgensma (onasemnogene abeparvovec-xioi), Itvisma (onasemnogene abeparvovec-brve), and Evrysdi (risdiplam) to increase SMN protein levels in patients. This protein is necessary for the survival of motor neurons, the nerve cells that send movement signals from our brains to our muscles. Now, the U.S. Food and Drug Administration has approved the first muscle-targeting therapy, Isembyld (apitegromab-mstn), as an add-on to existing treatments.
I may not be able to install new muscles, but I can soon receive an infusion every four weeks that will allow my existing muscles to grow. Scholar Rock, the company behind Isembyld, also hopes to provide an injectable version of the drug in the future.
Who’s laughing now?
You would think my mum and I would be jumping up and down in glee. Her prayers came true! I will have muscles! No placing my brain inside a robot a la “Ghost in the Shell” necessary!
The funny (funnier?) thing is, as I talk to my friends and write about it, I’m finding I’m in no rush to try Isembyld. In fact, I may want to wait until the injectable version is available.
This revelation is shocking to me, too. From 2021 to 2024, I was the girl screaming into every megaphone I was given for Singapore’s Ministry of Health to get their butts in gear and subsidize Evrysdi stat. I was on a mission to access treatment, and no one could stop me for any reason at all. Time and time again throughout the lengthy administrative process to set up my crowdfund, I was asked if I wanted to risk my mental health by being in the public eye. I responded by asking who would if I didn’t.
The difference is, my condition isn’t actively worsening this time. Since starting Evrysdi two years ago, I haven’t had to fear the loss of strength and dexterity that naturally occurs with SMA. I’ve instead been able to celebrate small motor gains, such as improved swallowing and speech. My stamina lasts longer, too.
I don’t feel like I’m racing against a clock anymore. I can take my time to observe Isembyld’s results in real-world research before I make a decision about it. Which may be the wiser choice this time, as I have to discuss with my doctors if it’ll interact with the zoledronic acid infusion they’ve recommended to treat my low bone density. Both zoledronic acid and Isembyld’s current form are infusions that can ironically cause fractures as a side effect.
There’s also the issue of how fine my veins are.
My last blood draw took four nurses, a resident doctor, two of my hired caregivers, and one ultrasound machine — plus the 500 mL of water I drank to increase my blood pressure. We almost called my pain specialist (a trained anesthesiologist familiar with my veins) down to the endocrinology clinic before a single viable vein appeared on the ultrasound screen at the last second. As much as I enjoy gaming conversations with my pain specialist, I’m not sure I have enough veins we can stick with an IV every four weeks.
True strength
Of course, the ability to wait and see what a drug can do for patients is a luxury. Not every SMA patient can be treated with Spinraza, Zolgensma, Itvisma, or Evrysdi. Not every country’s healthcare system provides financial aid for treatment to every age group. It is therefore still important for policymakers to understand that a progressive disease like SMA requires all of its treatments to undergo urgent reviews, and for pharmaceutical companies to price those treatments as affordably as possible.
Content though I am now, my case shouldn’t be used as an argument against Isembyld quickly becoming accessible in Singapore or elsewhere. Any doctor or researcher worth their salt will tell you that one anecdote is not representative of the complete data set; there are sure to be patients for whom Isembyld is necessary for daily functioning.
We must remember that a country is only as strong as its most vulnerable. Let’s go get some muscles!
Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.
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