I’m aware that these treatments have only been used a couple of times so far, but I’m wondering if there are many predictable side effects or risks? Our son, might be on the milder side of symptoms, but he’s also in a critical developmental stage. I’m wondering if there’s some downside that we’re not aware of that might deter us from wanting to try something like an ASO therapy?
Hi Cory,
I have to start with a caveat: Susannah and Sloane’s ASO experiences are still experimental and being optimized, so none of my answers here are the end-all-be-all. We’re still learning. The good news is that nlorem, Dr. Chung, and Dr. Bain have done an incredible job of profiling safety while working with Susannah. Any time Susannah has any kind of medical event she’s brought in for observation to assess potential association with treatment.
To date there has been one adverse event associated with administration; this was Susannah’s first dose of the ASO, and there haven’t been similar events to date. Here’s a description from the ASO paper:
"The first intrathecal (IT) administration was complicated by an epidural cerebrospinal fluid (CSF) collection at the injection site causing back pain that necessitated an external 4% lidocaine patch, acetaminophen and increased doses of gabapentin for 10 days, and leading to inability to walk for 7 days. The patient fully recovered 11 days after ASO administration once the CSF collection resorbed, which was confirmed by repeat magnetic resonance imaging. Because it was unclear if or how much of this first dose went into the epidural space, the 20-mg dose was repeated 1 month later.
“During the 9-month study, no other adverse events were observed. Clinical signs of increased intracranial pressure were specifically monitored and none were seen during treatment. Laboratory tests for safety assessments remained within normal during the study. The patient is still being treated.”
At their 2024 conference nlorem shared data regarding their broader program and had a really impressive safety profile across disorders, without other adverse events; they’ve done a great job of prioritizing safety and robust data throughout the ASO development process and I think that shows in how well the treatment is tolerated.
Another consideration is logistics: Because the ASO is still being studied, engaging as a patient family requires rigorous guidelines. Each intrathecal treatment is followed by a night of observation, and as I said any time the patient experiences a medical/health event, including things like colds, they have to be brought in for additional observation. Having a clear understanding of the process is crucial for families looking to engage in this process.
Dylan
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