[kristen-elaine]

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?

[kristen-elaine]

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.

 

*edit

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.

[kristen-elaine]

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.