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  • Which SMA Treatment Are You Currently Pursuing?

    Posted by ryan-berhar on July 11, 2019 at 7:03 pm

    Hello everyone. Hope you all are doing well.

    So I know this topic isn’t necessarily applicable to everyone. This is more a question directed at people who aren’t receiving Spinraza. I’m just wondering what people’s mindsets are regarding treatments.

    Is anyone still heavily pursuing Spinraza? Or are you holding out hope that the superior Zolgensma will soon become approved for adults? I fall into the latter category, but I just wanted to get other people’s thoughts.

    kristen-elaine replied 4 years, 11 months ago 6 Members · 9 Replies
  • 9 Replies
  • kristen-elaine

    July 11, 2019 at 7:43 pm

    So I’m curious, is there actually any reason to even suspect that Zolgensma will really be applicable to adults? Not older adolescents or essentially anyone still considered Pediatric, but to adults? The last I really read in to it, I remember seeing that there were trials being done to test the ability of zolgensma to be administered via intrathecal in to older children. But I don’t know, and maybe I’m wrong or just uninformed on any latest developments… but I don’t believe it will be a viable option for adults. Not even considering the insurance aspect, I mean, just biologically.

    But, to answer your question! I’m currently on Spinraza, FINALLY was able to start at the beginning of this year after about 1.5 years of obstacles, and currently weighing the possibility of stopping and taking what’s in store with that decision as it comes. Hopefully Risdiplam will be approved soon! If it is, I can definitely see that as an easier option, and supposedly more efficacious than Spinraza too, according to clinical studies, but I guess like everything in this new world of SMA treatment option, we’ll see?

  • michael-morale

    July 12, 2019 at 9:20 am

    I think it’s a little premature to say that Zolgensma is going to be the superior treatment. While Zolgensma is designed to replace the SMN1 gene, and Spinraza is designed to correct the SMN2 gene, both treatment options have their advantages. At the current time, AveXis is going through clinical trials with an IT or intrathecal delivery for Zolgensma so that it can become available to an older population, and while they are still going through clinical trials, I do have a gut feeling that it will become available sometime during the first part or middle part of 2020. Spinraza for me, has halted the progress of my disease, and I have seen a few benefits with regards to core strength and breathing. If anything, Spinraza has bought me more time until something else becomes available, like Zolgensma, Risdiplam, or maybe even the combination therapy between Cytokinetic’s Reldesemtiv and Biogen’s Spinraza.

    One thing that we need to remember is that Zolgensma does not repair the damage that has already been done in our bodies. It will probably show good benefits for those of us that are older, but nothing has been developed as of yet that will repair damage that has already taken place.

    • ryan-berhar

      July 12, 2019 at 5:16 pm

      Why do you think it’s premature? Zolgensma not only replaces the missing gene (Spinraza doesn’t), it’s also a one-time delivery. It’s objectively superior at least as I understand it.

  • michael-morale

    July 12, 2019 at 9:36 am

    Kristin – I wanted to address your comment and maybe answer the question that you had. AveXis is currently going through clinical trials of the IT or intrathecal delivery of Zolgensma. At the present time, Zolgensma is only available to infants and toddlers 2 years of age or younger, irregardless of the type of SMA that they have. (1, 2, 3 or 4) This treatment for these infants and toddlers is being done via an IV infusion. The reason why they are able to administer this treatment via the IV infusion, is that their bodies are so small, the treatment itself, doesn’t have any problem getting past the blood brain barrier and getting into their central nervous systems.

    AveXis is working on the IT or intrathecal delivery because this is the only way they are going to be able to administer this treatment to older children, teenagers and adults. They have to do it intrathecally for older children, teenagers and adults, because if they were to try to do it intravenously, they would not only have to push an exorbitant amount of drug through our bodies, it would probably not get past the blood brain barrier to get into the central nervous system.

    I’ve heard through the grapevine that AveXis is getting some good results so far in their clinical trials of the IT or intrathecal delivery method, and I truly believe that it will be available to those of us that are older. It may not be until the end of this year or maybe even the first part or middle part of 2020, but AveXis’ goal is to try to have this available to us as soon as possible.

    There is also a lot of work going on with another treatment called Risdiplam, and there is also a combination therapy animal study with regards to Cytokinetic’s Reldesemtiv and Biogen’s Spinraza. Reldesemtiv will work along the same lines as Risdiplam, but it will be in combination with Spinraza, and so far, in these animal trials, Cytokinetics and Biogen are reporting some pretty impressive results.

    All of these treatment options that are currently undergoing clinical trials and animal testing will be on our radar for discussion as soon as we know more information.

    I apologize for such a lengthy response, but I wanted to give you my opinions. Have a great day.

    • kristen-elaine

      July 12, 2019 at 6:15 pm

      Thanks Michael! I am aware of all of the different drugs in development, as well as their associated clinical trials and the data findings therein. What I was curious about was, why is there the idea among the adult population that Zolgensma will ever be something an adult SMA affected individual can receive? While I mentioned that I am familiar with the drugs and their published findings- I did not attend Conference this year, and I do know from past years that sometimes Drug companies reveal brand new data and findings that have not yet been published- so, my curiousity was regarding whether or not there was NEW information that I hadn’t heard of, or NEW assurances causing SMA adults to speculate Zolgensma would be available for prescription to adults. I do know that the current trials are testing intrathecal methods of administration for that particular drug to be administered to “an older population”, which yes, as you’ve mentioned is given via IV infusion to children under 2 years of age currently. I do not however believe that when they (they meaning, Avexis/Novartis affiliates) say “an older population”, an adult population is an actual potential. I believe when they say “an older population” they are referring to children older than 2 years, and possibly even older adolescents in their preteen years, but in all respects, not adults or anyone beyond puberty. I believe this because the viral vector used to carry the SMN1 gene into a human body, through whatever administration method, ie IV or intrathecal, has a HIGH propensity for immunity in an older population. That’s one of the MAIN reasons Zolgensma was approved with a label for babies under 2 years, because they are least likely to have incorporated that viral immunity yet, and yes, like you mentioned, because their smaller, require less drug, and the issues a more fully developed human has re: blood brain barrier is less of a concern. With adults also, as we’ve already seen with Spinraza, insurance approval for a drug as expensive as Zolgensma is an entirely different obstacle and one I’m not sure current payer policies and legislation will support in an older population. But that’s a whole other animal! So to say.



      I would also like to point out one part in your description of each current and currently developing drug, where you compare Risdiplam to Reldesemtiv. This is incorrect. Risdiplam is actually a small molecule drug designed to do what Spinraza does, act on the SMN2 gene slicing error, in order to create more full length SMN protein in the body. The major difference here is that Risdiplam is taken orally, every day, and Spinraza is an intrathecal injection every 4 months following the loading dose schedule. Risdiplam will most likely not be eligible to take alongside of Spinraza, as they function on the same biological mechanism. Reldesemtiv on the other hand, acts as a skeletal muscle growth agent, promoting muscle growth, which will hopefully enable it to be taken alongside of Spinraza and Risdiplam to help repair *some* of the atrophy muscle damage, or at the very least, help to encourage muscle development.

  • deann-r

    July 12, 2019 at 10:24 am

    Even though I’m currently on Spinraza I’m anxiously awaiting Risdiplam coming to market. Of course cost and access are among my biggest concern as in my opinion local pharmacies may be hesitant to carry a medication like that because of the cost. At least my pharmacy can be funny about things like that. Then there’s the question, will my insurance cover it? Hopefully since there are already treatments available it may bring costs down.

    Unfortunately if Zolgensma does become available to adults it would most likely be as an IT treatment from the sounds of it leading to the same roadblock that seems to be preventing some from pursuing Spinraza, access to the spine. Of course as a one time treatment maybe it would be more enticing to figure out that hurdle. I’m not as confident the label will be expanded as Michael expects. At least not as wide as the Spinraza label. I’m wondering if adults have built immunity to the viral vector they use. Total speculation on my part, I have not a shred of evidence to support it. Time will tell.

    • kristen-elaine

      July 12, 2019 at 5:47 pm

      DeAnn- I was considering the same as you mentioned! I’m also not confident that the label of Zolgensma will be expanded to include adults, additionally considering the high potential for immunity to the viral vector among the adult population.  While the potential efficacy of primary gene replacement (SMN1 primary gene vs the secondary SMN2, non homologous gene) would be superior in my own speculation, coupled with only a single dose, rather than guaranteed repeated treatments like in the case of Spinraza and Risdiplam, makes Zolgensma more appealing, I don’t think it will be a practical possibility for adults in reality. But like I also said in my post above- remains to be seen! There is also the researcher and designer concern that Zolgensma may in fact prove to be a drug that DOES require either repeated dosing in the long run or at the very least combination therapies like Cytokinetic’s drug treatment. That’s not something often mentioned in media posts or column write ups about Zolgensma, but it is a factually based consideration and concern that is repeatedly voiced by those that developed the biological mechanisms for Zolgensma. I think that’s also a point of contention many people/patients don’t want to consider or acknowledge, but the clinical trials were too short (less than a human lifetime, like Spinraza) to best assume what the long term results and effects will actually be.

  • rachel-markley

    July 12, 2019 at 12:06 pm

    I do believe Zolgensma will become available for adults, however, I have many concerns about it’s efficacy in those of us with neurons that are deader than dead. It took me a year and a half to come around to the idea of Spinraza and another year to get it. I get dose 4 Wednesday.

    • lupa-f

      July 12, 2019 at 1:27 pm

      Zolgensma isn’t going to be any different than Spinraza in that regard, Rachel. Neither one works on the nerves directly, both try to create more SMN1 which stops further destruction of the nerves, they just do it in different ways. The primary issue with Zolgensma and adults is how the virus they’re using to provide it works with an adult’s immune system.

      I’m not nearly as optimistic as Michael as far as the time frame for it being approved for adults. Spinraza had trials on older children alongside infants much earlier in the process than Zolgensma and didn’t have the same concerns about its delivery method which made a broader approval simpler. Zolgensma is just starting its trials for older children and adolescents and as a one time treatment they’re going to need data showing it remains effective for a long time.


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