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Dr Vamshi Rao Discusses the Future of Combination Therapy in SMA


Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses the future of combination therapies in the spinal muscular atrophy (SMA) space.

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.


What future do you see for combination therapies in SMA and how will they affect patient outcomes?

Rao: I think we are definitely seeing a push for combination therapies from the patient point of view but also from the scientific community in general. I think what we are struggling with, of course, is lack of data in terms of using combination therapies in the real world. We have 3 drugs approved. You would think that we would have the discretion of prescribing those drugs as a combination, but it's hard because there is no clinical trial evidence of that. So, I think most of the sponsors and pharmaceutical companies that own these drugs are looking forward to some data out there that can result in a combination product, whether it is a trial where their drug is the first to be given or whether their drug is the one that follows a drug. For example, because nusinersen [Spinraza] was approved in 2016 and most folks started with nusinersen, there are now trials looking at a whether coming in with a drug after nusinersen, such as, Zogensma [onasemnogene abeparvovec] or Evrysdi [risdiplam], would be more beneficial. More so, I think people are also looking at safety of when to use combination therapy.

Right now, there isn't much data out there, [except for] some publications, about real world experiences. A group that I work with published a paper where we looked at children who had nusinersen or Zolgensma and then got the other drug, whether it was nusinersen or Zolgensma. So, it's small studies. I don't think we can generalize results yet. Definitely, I do see that combination therapies are going to be the wave of the future and I say that because unless you have 1 drug that has been instituted early in life, that leads to a child or an individual who looks like a non-SMA child, there is going to be a quest for something better, something more. So, if you have had a drug that makes you attain motor milestones, and jumpstarts your development, but you don't reach the curve or follow the curve of development of a non-SMA trial, then there's still some scope to bridge the gap. That's where the question about what's next, whether it's a new drug or a combination of drugs, is going to happen.

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