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Any updates in the treatment pipeline?
Hey all. I am checking in to see if anyone has heard any news on potential treatments in the pipeline. I just got my 30th Spinraza injection this week, which is unbelievably wild. I can’t believe I’ve had THIRTY doses!
This milestone prompted my curiosity about new treatments on the horizon. I have my eye on the larger dose of Spinraza and Apitegromab for different reasons.
Nevertheless, I’m grateful to have access to treatment in the first place.
Have you heard about progress made in the treatment pipeline? Are there any notable updates?
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10 Replies
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Something I’m keeping an eye on in addition to the Myostatin inhibitor you reference, is something called anthrobots. These have shown (in the lab, not animals – yet) to allow a certain type of cell (not stem cell) to generate new or regenerate dead motor neurons. This is a fascinating development as far as I’m concerned. The problem with SMA is the loss/death of motor neurons. You are born with the number you will have in your entire life. So, once the die, that’s it for that one. So, all the medication to me, and this is just they way I think about it, like higher doses of Spinraza, won’t really matter as much until something like this is developed. IOW, if you have 100 motor neurons in your arm, and 50 have died from SMA lacking SMN1 and the ineffectiveness of SMN2, even those modified by treatments such as Spinraza and Evrysdi, you still only have 50 to effectively communicate. If anthrobots work as the lab has shown so far, those 50 dead ones could be regenerated or new ones could be developed to replace them, giving you all 100 motor neurons in your arm. Sounds promising.
I also know someone who works for BioGen and although they couldn’t disclose specifics, when we were talking about the above back in March, her response was, “CRISPR gene editing is the thing to watch and may be available before any of those options are.”
So, that we have hundreds, if not thousands of people working every single day to improve our lives is an absolutely wonderful thing and something I am particularly thankful for.
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Yes, thankful is an understatement. I forgot about CRISPR when typing my original post. My dad has had his eye on this for years. Do you know if there is any recent literature on this? I’m going to have to look into it.
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Nothing new specifically, so I just keep an eye on research online and something seems to pop up from time to time. I was just excited when my friend at BioGen implied that this research is progressing well and may be available before the other products like Scholar Rock’s myostatin inhibitor. My question to her specifically was about the concern with loss of motor neurons and she said she believes CRISPR will help with that as well. Short of that, anthrobots look really promising for that.
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That’s really exciting to hear. I’ve been wondering about updates myself as well. I didn’t know about the other one he mentioned above. But I’m glad to know now. I hope some more information will be available at some point
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Has anybody received the email about the new pill form of Evrysdi? I just saw it in my inbox and apparently it has already been approved by the FDA so I’m not sure when it will become available to us. If it’s not already
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I saw that! I believe it’s a tablet, not a pill. I’m sure it will be much more convenient than refrigerated liquid. Are you on Evrysdi right now and thinking about making the switch?
I’m currently taking Spinraza, but Evrysdi is always in the back of my mind as spinal taps are getting more and more challenging for me. This tablet would be amazing, but I wonder: is it an uncoated tablet? I’m sure many SMA people have feeding tubes or trouble swallowing and need the tablet to be crushed. If it’s coated, I don’t think it can be? Don’t quote me on that, though. That’s just what I heard.
I believe it’s not available yet, but it has been approved.
Here’s an article Roche posted: https://www.roche.com/media/releases/med-cor-2025-02-12
roche.com
FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and...
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Update. I should have read the article first. 😂 You can disperse the tablet in water! And they said it will be available in the coming weeks.
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I switched to Evrysdi in September after 6 years on Spinraza. I spoke with my Genentech rep yesterday who said she’s receiving a lot of requests about this. a few things of note:
1. It HAS to be taken orally, even if diluted in non chlorinated water. If you use a feeding tube, you can’t take it in the pill form.
2. It may require a new prior authorization to be approved. This depends on your prescription benefit plan. If yours requires it, it may be worth continuing until your current PA expires (usually annually).
3. It’s a 30 day supply! Yay! No more of the nice round 24 day supply. 🤨
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Interesting. Why can’t it be administered through a feeding tube?
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Oh yeah, that’s what I meant when I said pill. Was just using that as a generalized term for capsule, tablet, etc. LOL. I can’t believe it’s going to be available in just a few weeks. That’s amazing! I wonder how the process will be for us to obtain this.
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