Even with Isembyld’s OK, I’m still getting used to SMA therapy approvals
My late friend and colleague Michael Morale liked to utter a phrase to me whenever I had an aquatic therapy session: “Get strong.” Michael spoke with a thick Texan accent and the utmost sincerity as he emphasized those words. He fully believed that all of us with SMA could reach for the stars in terms of our physical and treatment goals.
Though I appreciated his gesture of faith, I considered my level of optimism more restrained than Michael’s. I went to aqua therapy and received my Spinraza injections with the intent of slowing my disease progression. The notion of building back muscle and regaining lost strength sounded appealing, but I managed my expectations.
Michael, on the other hand, had a relentless enthusiasm for SMA research updates and documented his thoughts in numerous YouTube videos. He was particularly interested in an experimental SMA therapy from Scholar Rock, and declared multiple times that its approval was just a matter of time.
A breakthrough
Well, my friend, you were right. Go ahead and smirk.
On Sept. 11, the FDA approved Isembyld (apitegromab-mstn) for children and adults with SMA. This muscle-targeted therapy marks another breakthrough for the SMA community, and I’m sure that the next few months will be busy for neurologists and SMA care centers.
I expected this news, given that I work in the rare disease space and have been following updates from Scholar Rock. Still, I can barely keep up with how the world of SMA is changing. Our community now has five FDA-approved treatments, and these approvals happened over the course of 10 years. Back when I was a kid, the idea of any type of therapy was just a dream. We thought there might be something for future generations, but certainly not anything in my lifetime.
As someone who doesn’t often dwell on sentimentality or even write much about treatment and research topics in my column, I’m switching things up this time. This moment calls for reflection and celebration.
I learned about the approval while scrolling through my Instagram feed on a Friday night. I was outside on our family’s porch, and I texted my mom and some SMA friends. My phone continued to buzz with more notifications, and I saw multiple friends share the news on social media.
I didn’t cry or scream with joy, but I expressed my emotions in different ways. As I went for a stroll that evening, I thought about Michael and others who I wish were here to see this moment. If Michael were here, he would have uploaded a video before our news team even had time to cover the story.
Then I thought about whether I would pursue this treatment for myself. The potential benefits are readily apparent. If I can get just a small amount of muscle strength back that I’ve previously lost, I would be happy.
Plus, I don’t have to worry about switching from the therapy that I’m already on. Since Spinraza (nusinersen) still works great for me, I’m happy with it, but I’m also interested in looking at additional therapies that would work in conjunction with it. Regardless of what I end up doing, the fact that I have choices is nothing short of spectacular.
I used to think Michael had a little too much optimism. He and I worked together during the COVID-19 pandemic and during times of political and social upheaval in our nation and world. His lack of cynicism frustrated me at times, but now I realize it’s exactly the kind of spirit we need. Despite the proliferation of negative news, we have reasons to hold onto hope. We are living in an unprecedented time for the SMA community, and I can only imagine what kind of advancements are still to come.
One thing I do know: Michael is smiling.
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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