SMA News Forums Forums Treatments and Research Any updates in the treatment pipeline?

  • Any updates in the treatment pipeline?

    Posted by alyssa-silva on May 10, 2024 at 1:34 pm

    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?

    mike-huddleston replied 1 month, 2 weeks ago 2 Members · 3 Replies
  • 3 Replies
  • mike-huddleston

    May 14, 2024 at 6:27 pm

    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.

  • alyssa-silva

    May 19, 2024 at 2:07 pm

    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.

    • mike-huddleston

      May 26, 2024 at 7:32 am

      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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