At 2018 Cure SMA Conference, Research Presentations Project a Promising Future

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by Kevin Schaefer |

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Last week, I wrote about the personal impact that this year’s Cure SMA conference had on me, as well as the relationships I built during that week in Dallas, Texas. In this column, I’ll be sharing my perspective on the research component of the conference.

One of the most popular and important sessions at the conference each year is the researchers meeting, which consists of both presentations and a Q&A. This year, it was held on Saturday, June 16, in the hotel’s grand ballroom. The second half of this session provided the most information, as representatives from the major pharmaceutical companies working on SMA treatments were present. These companies included Biogen, AveXis, Cytokinetics, Genentech, and Novartis.

Since starting my Spinraza (nusinersen) treatments, I’ve tried to pay more attention to the developments going on with SMA-related research and I’ve looked at other drugs in the pipeline. Particularly, I was curious to hear an update from Cytokinetics about their clinical trial therapy — reldesemtiv (CK-2127107).

The representative from Cytokinetics said that reldesemtiv is showing positive results in its current clinical trial stage, particularly among ambulatory patients. While this doesn’t necessarily apply to me, it’s exciting to see other therapies getting closer to being FDA-approved.

During the Q&A portion of this session, SMA News Today’s YouTuber, Michael Morale, posed a great question to the spokeswoman from Cytokinetics. Given that reldesemtiv is designed to work at the muscular level and Spinraza works at the cellular level, he asked if there were any conversations going on between Cytokinetics and Biogen to develop some kind of medication that works on both levels.

Many people in the audience applauded Michael’s question, and I found the researcher’s response to be encouraging. Dr. Stacy Rudnicki, the Cytokinetics representative who spoke during this portion of the session, said that conversations are ongoing between these two pharmaceutical companies. She said they are working on an animal study to test out the kind of therapy that Michael proposed.

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Ultimately, I came out of the research symposium with a positive view of the state of today’s SMA research. While I’m content with just staying on Spinraza, the idea of future treatments is certainly promising for a number of people in the SMA community. When I think about the research status back when I was a kid going to my first SMA conferences, it paled in comparison to what it is today. Today, the resources, information, and community support available are exponentially greater than I ever anticipated.

This time a year ago, I was overly anxious about starting my Spinraza treatments, and ultimately I had no idea what to expect. Obviously, I wanted to stop the progression of my SMA, but I was still nervous about the injections and possible side effects.

Now, I’m quite glad that I did go through with it, and I am seeing improvements in my energy level and muscle strength. It’s been a whirlwind of a journey with plenty of ups and downs, but the results have made it well worth it. I’m also fortunate to have a fantastic Biogen case manager and neurologist, both of whom have supported me every step of the way.

At the same time, I know other adults with SMA who have been unable to acquire Spinraza, either due to financial obstacles or problems related to the actual injection process. My hope is that those individuals can find some encouragement from the research updates from this year’s conference. My takeaway from the research session was that other treatments are moving forward in the pipeline, and there is definitely the possibility of future FDA-approved therapies for SMA. The future is bright.

For more on this discussion, check out this topic in the SMA News Today forums, where you can share your thoughts and questions


Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.


Dante avatar


What kind of muscle strength improvements?

Kevin Schaefer avatar

Kevin Schaefer

Hi Dante. So, I’ve been able to move my arms a little bit more since starting Spinraza. I can’t lift objects or anything, but it’s easier to slide my hands across my tray. Improvements like that are significant for me.


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