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Dr Vamshi Rao Discusses Possible Future Guidelines for SMA Care


Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago discusses how new treatments will influence future care guidelines for spinal muscular atrophy (SMA).

Vamshi Rao, MD, is an attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine.


Now that there are 3 treatments options for patients with spinal muscular atrophy [SMA], do you foresee any clinical guidelines being developed and if so, what do you think would be their focus?

Rao: I think we'd have been, over time, modifying our standard of care guidelines. The most recent modification was 2 to 3 years ago and that happened after a long time after the previous modification. This modification to the standard of care guidelines was pushed because nusinersen [Spinraza] was approved. Now, we have 2 drugs [approved] after that. So, I'm confident that we need more modification or change in our standard of care, especially because our natural history, in terms of what an individual with SMA looks like, is absolutely modified. It's different. And thank goodness, it's different. What do we do with these modified natural histories is going to be the subject of the new standard of care guidelines. The child that was not destined to sit up or walk is now sitting up and walking. What does that mean for bone health? What does that mean for their orthopedic manipulations in the future? How do we deal with scoliosis in children who were not supposed to get scoliosis? And vice versa? It's a very vast question and I don't think we'll be able to deal with every aspect of it today, but I think we will need to change and modify our standard of care guidelines. And those standard of care guidelines will also need to talk about or touch upon what we think about combination therapies.
My analogy to that I use comes from the world of oncology. When we started looking at cancers in children, we started using drugs and we found that those drugs were riddled with side effects, but they had some benefit of prolonging life. So, we kept using them. Then, we found out that we could make better drugs but we realized that we actually had to combine those drugs. Over time, we realized we not only had to combine them, but we also had to look at the genetics of the particular cancer to personalize those drugs. I think we're moving in that same direction with SMA. We started with a drug, and there were nowhere close to the side effects that we experienced with the cancer drugs, so that's definitely better. The cost of the drugs is sometimes limiting in terms of how the insurance companies allow us to combine drugs. We have to look at each child and say what stage they are, what developmental milestones they haven't hit. What is the gap that we still have in terms of these children attaining the next level? What standard of care guidelines should be put into place, as well as combination therapies and potentially new therapies in the future? So, it's a very it's a very hopeful world, but it's a world and a future filled with a ton of questions that we need to answer before we get there.

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