Goals of Therapy and Treatment Selection in Adult-Onset SMA


An insight into the goals of therapy and treatment selection between oral and intrathecal medications for adult-onset spinal muscular atrophy.

James Wymer, MD: With the 2 medications, you’ll see in the clinical trials that the kids lead to improvement. In adults there may be some modest improvement, but the most important thing is the stabilization we see in the disease. These have the potential in adults. For a disease that’s a gradually progressive neurodegenerative course, starting 1 of these medications can start to stabilize it. The goal of the therapy is stabilization of disease. With the other types of interventions, can they lead to improvement? 

We’ve got several very good trials that have come out. In the case of risdiplam, you have the SUNFISH trial, which was a 12-month trial that included adults. It showed clearly some benefits and some improvement, but most important, the big take-home I see is that a disease that’s gradually progressive and neurodegenerative in the adult level was showing stabilization so that we could enhance and improve recovery. As an oral therapy, it was very well tolerated. There were not many complications. A few you need to go over with your neurologist to make sure it’s the right medication for you. But it definitely was effective in the treatment, in the controlling of the disease, so you could introduce all the other therapies to try to enhance recovery.

In deciding the ideal medication for this patient, you’re going to discuss what’s going to work best for their lifestyle. You’re going to look at the options. Do you want the oral? Do you want an injection intrathecal therapy? Which is going to be quality? If the patient wants to take the oral therapy as this 1 did because of concern for the intrathecal, you’d then review with them the potential adverse effects that we can see. These include fever; GI [gastrointestinal] adverse effects, in terms of diarrhea; ear infections; and upper respiratory infections. We have to educate patients on what to follow. If these infections occur, they need to be addressed. They need to be identified. Certainly if they have respiratory involvement, as this subject does, then you must think about the concern. Are you going to develop pneumonia? Should you be getting antibiotics for treatment? Otherwise, addressing the adverse effects tended to be milder and easier to control. For the GI adverse effects, you can adjust the dosing back and gradually taper it to work toward that, just to come up with what’s ideal to get patients to tolerate. Most patients tolerated the medication well. Over time we’re able to stay on the medication and get the benefit from it.

Transcript Edited for Clarity

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