Evolving Landscape of SMA Treatment
In their final thoughts, Julie Parsons, MD, and John Brandsema, MD, share insights on the evolving and future SMA treatment landscape.
Julie Parsons, MD: We’re thrilled to have 3 disease-modifying therapies, but each agent can be improved on. We can do the 1.0 and 2.0 drug. We still have room for improvement, and we have new drugs being developed that aren’t necessarily SMN-dependent drugs. Maybe we’ll start working on the muscles. Can we increase the force of the muscle and increase strength? Can we do even better for patients? Adult and young adult patients with SMA [spinal muscular atrophy] complain significantly of fatigue as being a problem, and it gets in the way of their life. Is there another agent that we could use to get rid of the fatigue for these patients so that they have the strength and energy to continue to have active, productive lives? It‘s an incredibly exciting time. This is only the beginning. We’ve made amazing changes and have benefited the patients, but there’s still much more to come. We aren’t done.
John Brandsema, MD: Another very important message that we say to any person living with SMA, their caregivers, the payer system, or other colleagues related to this disease is once we know you have SMA, we need you to be established with an SMA-focused care team for your care for life. This is to optimize the standard of care and because these therapies are new. We need to be vigilant to ensure that we’re assessing the short- and long-term aspects of how these therapies impact the disease and their potential complications in a way that gives the best possible outcome for the person living with the disease. This is an important message that needs to be clear in any care plan and any endorsed approach to this disease. We want you to be connected with people who know this disease and know how to best help you.
Transcript edited for clarity.
In a response to a survey, caregivers of people with spinal muscular atrophy identified the risk of severe adverse events and the need for permanent ventilation as the most important factors in treatment decisions. Access to treatment, including cost and availability, ranked third.
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