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Gene therapy approved for patients 2 and older 🎉
If you haven’t heard the news yet, the FDA has approved gene therapy for anyone over the age of 2 for all disease types. *Cue my jaw dropping* You can read all about this huge milestone here, but I would love to hear your thoughts as well!
Are you considering exploring the potential of receiving gene therapy?
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15 Replies
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I am completely shocked by the news. In fact, I didn’t believe it at first and had to do my own research. To be honest, I hadn’t heard much about the clinical trials, so this came as a huge surprise. I am definitely going to discuss this with my neurologist to see if gene therapy would be a fit for someone like me. I am 35 years old and have type 1.
Regardless of whether or not this is a good fit for me, this is such a major accomplishment that I didn’t think I’d see in this lifetime. Right now, I am just in complete awe of science, the people who research our disease, and all those who are part of the journey to discovering new treatments. It’s hard to put into words how I feel right now, but thankful would be a good place to start.
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This is amazing news! And it’s the latest example of why, for years, I have said I am so thankful for the hundreds, if not literally thousands, of people who work every single day to make our lives better. I know there’s someone on here who I’ve known for about 5 years (predating my time here) who is very negative about his SMA diagnosis and how “unfair” it is. I hope he sees this article – or is aware of it – and develops a better sense of appreciation. This is exactly why I have hope and personally, have always profoundly felt that whatever can be done for the benefit of the youngest amongst us warms my heart. If I benefit as a byproduct of that, even better. Understanding that because of research and treatments that SMA is no longer the leading cause of death in children under the age of 2 is beyond adequate words.
That said, I already contacted my neurologist about this and although it is unlikely a viable option for me, it doesn’t change my appreciation. For me, my age (62) and the fact that I was untreated for about 40 years before starting Spinraza in 2018 before switching to Evrysdi in 2024. Also, since this addresses the faulty exon 7, though it would significantly reduce if not eliminate further progression, there is no indication that it will address areas with the most/more significant weakness as that is an indication of more sever motor neuron loss (I word it that way intentionally as Zolgensma has resulted in some patients needing either Spinraza or Evrysdi as ongoing maintenance). In fairness, this is as expected and as with other currently available treatments since there is currently no viable treatment that repairs or creates new motor neurons. As with ALL treatment options, the earlier in our diagnosis, the less motor neuron damage, thus the more effective any treatment will be. Hopefully, and I don’t doubt this will happen at some point, science will get to a better place with motor neuron repair or creation so we can all gain the most benefit for all treatment options. And finally, due to my age, it will be a battle to justify the $2.59M price tag.
All said, this doesn’t mean I will not pursue it, just setting my expectations accordingly. It also does not diminish my heartfelt thanks for all of the efforts of so many to bring this, and other treatment options, into reality. 💜
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This is HUGE.
As you say, it is unlikely to benefit many of us, but the changed lives of those newly diagnosed could be amazing. To think that until recently there was nothing that could help and now we have multiple choices. We will need to trust our providers and hope that there are well trained therapists available.
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It is so amazing. Sometimes it’s even hard to wrap my brain around. I wonder what SMA will look like in 50 years… 🤯
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I am so eager to try this therapy! I cannot wait and big cheers to our dedicated scientists and donors who have funded this incredible journey
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Good luck! You’ll have to let us know how it goes!
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I’m just waiting to hear back from my neurologist about it. Thank you I will!
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I am going to do it. I have my next Spinraza appointment next month, so I am going to do it after Spinraza shot and have doctor submit to insurance company. Also, I found and article a group is doing research on reversing atrophy with SMA specifically.
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Really? I’m curious
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I am, too. Please share it here if you get a chance!
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To be clear, in yet another helpful webinar yesterday sponsored by MDA on gene therapy, no existing treatments reverse damage done prior to treatment. If you’re using Evrysdi or Spinraza, neither of those completely stop damage to all motor neurons but rather (and hopefully) slow the effect. There are many groups announcing work (they’re trying to get funding) but I’d use caution in assuming this will be quick, if effective. When members here post up statements, it would be helpful if links to this research (what you read that lead to the statement) so others can access and read the info for themselves. We should all maintain hope in reasonable amounts, especially in light of how things have progressed just in the last 10 years. For SMA, there must be the ability to create or repair dead or dying motor neurons. Until that happens, any treatment will be limited in its effectiveness. MDA said the recording of this presentation will be on their site soon. This typically is within a week or two.
They also explained why these treatments working on the AAV vector (like Zolgensma and Itvisma) are one time only as our bodies develop antibodies. They also discussed why these treatments are not always effective for all patients in the same manner, with some Zolgensma patients now on maintenance with either Spinraza or Evrysdi. This was more technical than I captured. Also of note, some folks will already have these antibodies so tests will need to be done for each individual seeking this new treatment for their existence as that would prevent it from working and it would not be approved.
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@Chrisr TLDR version: Please post up a link to the article you mention.
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new treatment areas to try to regain function articles. the SMA foundation is putting money into moonshot projects to try to reverse course in the disease:
https://hscrb.harvard.edu/news/regenerative-therapy-spinal-muscular-atrophy/
hscrb.harvard.edu
Regenerative Therapy Project to Revolutionize Spinal Muscular Atrophy Treatment | HSCRB
Regenerative Therapy Project to Revolutionize Spinal Muscular Atrophy Treatment | HSCRB
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Thanks for sharing, Chris. That’s really interesting. I wonder how this project is going as I see the article was published in 2021.
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Appreciated. There’s just so much to weed through these days, which can be good and bad. Knowing what others see or reference is helpful.
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