Drugs in Development and Next Steps


Tim Hagenacker, MD: There are still some unmet needs in treatment of the adult SMA [spinal muscular atrophy] population. The important point is that we still need the multidisciplinary treatment approach. The medication does not replace the multidisciplinary approach. It will still be essential. We have these patients who are very severely affected. They don’t have a good number of motor neurons and cannot be improved by a medical treatment focusing on the SMN protein. Stabilization of the muscle itself or improving the muscle function can be a good additional treatment option, in addition to SMN-focused drugs. There are ongoing studies on muscle enhancers and similar treatments. We need these combinations of drugs to get patients within their best motor function. 

James Wymer, MD, FAAN: There are several other drugs they are working their way through to try to develop. This is where the excitement happens. We have 2 drugs that we just talked about that are looking to correct the problem. We have an AAV vector that’s already approved for type 1 SMA. If you look down the pipeline, you see a combination of several. There are refinements to the medications that we have now, but we also have a whole other group of medications that are working their way through that to try to enhance the muscle function. 

These work across different neuromuscular diseases. Remember, earlier we said that the medications can arrest the process. We didn’t know if it was going to improve things, but the goal was simply to arrest the process. Now we want to get the muscles functioning better. We want to improve their function. There are medications in clinical trials that they have to sort out. If they work, they have to sort out their efficacy. There’s a long way to go before they can come out. But if they do show the benefit that we’re hoping we will see, then we’ll have something that we can put on top of the SMN2 medications to try to get the muscles to function better and stronger, and for people to do better.

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