Blind and Visually Impaired Community
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RP Gene Therapy 1 Monthish Later (self.Blind)
submitted by guyfierionlyfans
Howdy. I shared this in /r/retinitispigmentosa but figured I’d share here too in case any RP folks aren’t subbed there plus I think this treatment could be the future for other conditions.
On 2/3 I had gene therapy surgery on my right eye and on 2/17 I had the left eye done. When I posted last I was still recovering from the right eye and hadn’t had the left done yet so a lot’s changed since then.
Quick background, I have XLRP and this therapy basically injects a virus into the retina that is carrying healthy RPGR genes that are supposed to go in and replace the mutated genes. This is a phase 3 clinical trial so theoretically this treatment could be approved for anyone with this mutation to get in the future if it’s proven to be safe and effective.
Regarding safety, so far so good. The surgery itself is pretty quick and routine and the eye can see normally after about a week of blurriness. It’s a little uncomfortable for a while but not really painful at all. They have me on eye drops and steroids to prevent inflammation. No complications or side effects for me.
As far as effectiveness I’m happy to report that the treatment is working! About 2 weeks ago, once the left eye was healed and seeing as good as it was pre-op, I was able to really compare the right eye to the left and started noticing how much sharper and vivid things were. Now that the left eye is catching up to the right I’m noticing the same thing. I feel like at this rate my prescription is going to improve next time I have a proper eye exam. Colors aren’t blending together as much, things just look like they’re in a higher resolution now.
On top of that it’s helping with light and contrast sensitivity. I wouldn’t be able to walk around alone at night still but just around the house in dim lighting I’m noticing things I wouldn’t have been able to see before. When I come inside from walking the dog I can actually see without needing to adjust for 5 minutes. Previously I’d be completely blind coming inside. Things don’t blur together as much as they used to. I have to use high contrast mode on PC but when I do look at a normal white screen with black text it’s easier to read now. Even my phone looks brighter and clearer.
These are still relatively small changes; I still can’t drive a car and I’m not suddenly cured, but I wouldn’t be sharing this if these changes weren’t significant enough to notice. I honestly wasn’t expecting to notice any improvement at all, certainly not this soon, so this is all a big welcome surprise.
Since it’s a research study the Drs can’t speculate about how much more improvement I may see and I don’t redo my baseline tests for another 6 weeks so all I have to go by is my own observations til then. I’ll update here once I have more to share and in the meantime I’m happy to answer any questions folks may have.
Also, if you’re not a member already feel free to join our RP Discord server. Lots of fun banter and good info in there and I’ve also shared more detail about this experience there. https://discord.gg/GVb2vB8UJ5
On 2/3 I had gene therapy surgery on my right eye and on 2/17 I had the left eye done. When I posted last I was still recovering from the right eye and hadn’t had the left done yet so a lot’s changed since then.
Quick background, I have XLRP and this therapy basically injects a virus into the retina that is carrying healthy RPGR genes that are supposed to go in and replace the mutated genes. This is a phase 3 clinical trial so theoretically this treatment could be approved for anyone with this mutation to get in the future if it’s proven to be safe and effective.
Regarding safety, so far so good. The surgery itself is pretty quick and routine and the eye can see normally after about a week of blurriness. It’s a little uncomfortable for a while but not really painful at all. They have me on eye drops and steroids to prevent inflammation. No complications or side effects for me.
As far as effectiveness I’m happy to report that the treatment is working! About 2 weeks ago, once the left eye was healed and seeing as good as it was pre-op, I was able to really compare the right eye to the left and started noticing how much sharper and vivid things were. Now that the left eye is catching up to the right I’m noticing the same thing. I feel like at this rate my prescription is going to improve next time I have a proper eye exam. Colors aren’t blending together as much, things just look like they’re in a higher resolution now.
On top of that it’s helping with light and contrast sensitivity. I wouldn’t be able to walk around alone at night still but just around the house in dim lighting I’m noticing things I wouldn’t have been able to see before. When I come inside from walking the dog I can actually see without needing to adjust for 5 minutes. Previously I’d be completely blind coming inside. Things don’t blur together as much as they used to. I have to use high contrast mode on PC but when I do look at a normal white screen with black text it’s easier to read now. Even my phone looks brighter and clearer.
These are still relatively small changes; I still can’t drive a car and I’m not suddenly cured, but I wouldn’t be sharing this if these changes weren’t significant enough to notice. I honestly wasn’t expecting to notice any improvement at all, certainly not this soon, so this is all a big welcome surprise.
Since it’s a research study the Drs can’t speculate about how much more improvement I may see and I don’t redo my baseline tests for another 6 weeks so all I have to go by is my own observations til then. I’ll update here once I have more to share and in the meantime I’m happy to answer any questions folks may have.
Also, if you’re not a member already feel free to join our RP Discord server. Lots of fun banter and good info in there and I’ve also shared more detail about this experience there. https://discord.gg/GVb2vB8UJ5
yourmommaisaunicorn 6 points 1y ago
Thank you for posting here! I’m part of the other sub but didn’t see it in my feed.
Was the procedure a shot in your eye?
Was the procedure a shot in your eye?
guyfierionlyfans [OP] 5 points 1y ago
Yup! They make a small incision and inject some goo into the retina which is carrying the good genes. They actually stitch up the incision after which is really unsettling to think about but you don’t really feel it much.
MacaroniGlutenFree 5 points 1y ago
As a father to a RP daughter, this is beautiful news. The basis of this treatment could practically apply to every RP gene, eventually.
Questions : how old are you if you don't mind? Is the treatment for life or do you need yearly injections? In which country are you?
Questions : how old are you if you don't mind? Is the treatment for life or do you need yearly injections? In which country are you?
guyfierionlyfans [OP] 3 points 1y ago
31 y/o male in US. Yea there’s similar treatments for other genes, Luxturna is already approved and available for RPE65. Obviously this is new so impossible to say if it’ll actually be permanent but yes that’s the plan - a one time injection that should preserve and improve vision for life. Pretty miraculous
MacaroniGlutenFree 3 points 1y ago
Well I sincerely wish you all the best. I hope they can apply the principle to more and more genes in the upcoming years.
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guyfierionlyfans [OP] 2 points 1y ago
No stars yet but we have light pollution here and honestly I haven’t looked. Might need to let the treatment do it’s thing a bit longer until I’m seeing that good. The hospital hosting the trial is only about a 2 hour drive so my wife’s been taking me but yes they cover hotels and parking and everything.
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guyfierionlyfans [OP] 2 points 1y ago
The gene was actually created synthetically in the hospital but I guess the virus itself was made elsewhere. There really aren’t serious risks. The procedure (vitrectomy) is relatively routine and this has been done for other types of gene therapy so it’s not a new surgery just the actual medication is new. There’s risk of inflammation which they mitigate with steroids and antibiotics. Theoretically the surgeon could botch it somehow but I trust them. Other than that they tell you the virus could theoretically spread outside of the eye and have unknown effects but they test for that and no issues. They did say I’m more likely to develop cataracts but that’s treatable. So the rewards outweighed the risks for me. Didn’t wanna have to wait another 5-10 years. I still see OK now so I wanted to preserve /improve what I have now rather than do this when I’m almost blind and it may not be as helpful long term.
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violetsocks 3 points 1y ago
Congratulations! These are amazing steps in the right direction. I don’t have RP, but I’m hoping there is a gene therapy for us Stargardt’s people soon.
synthpopolis 3 points 1y ago
This is fantastic news! I’m so excited for you.
I know you’ve been getting hammered with questions, and I’m going to ask a few of my own if you will be so kind as to indulge me. :)
- Is the goal of this treatment to stop the progression, slow the progression or reverse it?
- Do you know if it is expected to have any positive impact on peripheral vision and/or night vision?
I have a form of X-linked RP but beyond that, I don’t know which specific mutation is involved. Considering this is already in phase 3 trials though, that’s definitely impetus for me to get a genetic test to learn more about my condition.
Thanks again for sharing! Keep us updated
I know you’ve been getting hammered with questions, and I’m going to ask a few of my own if you will be so kind as to indulge me. :)
- Is the goal of this treatment to stop the progression, slow the progression or reverse it?
- Do you know if it is expected to have any positive impact on peripheral vision and/or night vision?
I have a form of X-linked RP but beyond that, I don’t know which specific mutation is involved. Considering this is already in phase 3 trials though, that’s definitely impetus for me to get a genetic test to learn more about my condition.
Thanks again for sharing! Keep us updated
guyfierionlyfans [OP] 3 points 1y ago
Yea the goal is to reverse the progression. So far it’s doing that. It won’t affect the peripheral really since it only goes into the center of the retina but yes it’s helping with night vision and all other RP symptoms. Definitely get genetic testing done because if this or another treatment gets approved you’re gonna wanna get in line. It’s already worth it and who knows how much better things might get. Very exciting stuff :)
synthpopolis 1 points 1y ago
Thank you for the reply. Just to make sure I understand, when you say reverse the progression, does this mean the treatment could potentially restore already lost vision over time?
Last time I spoke to my eye doctor, he basically said the near term goal of these advances would be to stop the progression but that restoring vision that was already lost would be unlikely, at least with the studies currently underway. Thanks again
Last time I spoke to my eye doctor, he basically said the near term goal of these advances would be to stop the progression but that restoring vision that was already lost would be unlikely, at least with the studies currently underway. Thanks again
guyfierionlyfans [OP] 2 points 1y ago
Yes it’s already improved my vision from how it was before the surgery. I still won’t be “cured” but I’m definitely seeing significantly clearer in all lighting conditions and not having as much of an issue with light sensitivity and adaptation. I wasn’t blind though so yeah restoring completely lost vision is gonna be a bigger challenge. Hopefully at least with this gene therapy future generations can get it before they ever lose that much vision in the first place
synthpopolis 2 points 1y ago
That’s incredible! I hope you continue to experience benefits, and thanks again for sharing your story
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TechnicalPragmatist 1 points 1y ago
I was like it would be something from no vision to vision especially never seen before or not seen in a long time that must be a shocker. I have been totally blind for 20 years now hardly can imagine life otherwise. I also don’t have rp though and a cure or fix is probably not possible here. But I could just imagine learning to see.
SugarPie89 2 points 1y ago
Hi I just got surgery in my left eye on Monday. I am a little curious as to what you received because you said it's in stage 3 trials. I got Luxturna which to my knowledge is FDA approved and is out and considered safe and effective gene therapy for RP caused by mutations in both copies of the RPE65 gene. I'm excited that more people are getting this treatment though. Of course I wish I could've been younger when it came out but beggars can't be choosers I suppose. I will gladly follow your story! Very excited it's working for you.
Nevermind i read your message over lol you have a different mutation and this is a different treatment. This is great I am glad they are already expanding to different genes.
Nevermind i read your message over lol you have a different mutation and this is a different treatment. This is great I am glad they are already expanding to different genes.
guyfierionlyfans [OP] 2 points 1y ago
Yup it’s essentially the same idea as luxturna. I think all the big mutations will have similar treatment soon. Hope it works out for you! I started seeing minor improvements after like 7-10 days. Kinda freaky but so exciting.
SugarPie89 1 points 1y ago
Thank you! I've been a little freaked out about how blurry my left eye has been post surgery. The doctors said that was normal but somehow I wasn't expecting my vision to be like that. Maybe I just didn't ask enough questions about what I should expect post surgery lol. Of course it makes sense I mean i just got operated on but yeah was a bit shocking for me. Do you notice any improvement regarding floaters and flickering lights/pin wheels?
guyfierionlyfans [OP] 1 points 1y ago
Yeah it took about a week to see somewhat normally after the surgery. Days 1-5 were very blurry with gradual improvement then days like 6 and 7 got dramatically better overnight. I’m sure you’ll be fine in a few days. I didn’t have super bad floaters or flashes or anything before seems about the same now I still see things from time to time but idk if there’s any improvement there or not.
SugarPie89 1 points 1y ago
Well i will just have to see then about the floaters. Would be nice to get rid of them lol though I am not expecting them to go away completely. About how long did it take for you to be able to wash your hair? I know that's random but my hair is starting to smell lmao. I should've went with my gut and washed it the weekened before but I didn't. I'm excited to start seeing improvement soon.
Iamheno 2 points 1y ago
Good news! Was this a company based in Dallas by chance?
guyfierionlyfans [OP] 3 points 1y ago
The study is in Boston but the company is based in London
bongunk 1 points 1y ago
Reading your post and comments and it's so exciting! Are you at liberty to say who the company is and do you know whether they are doing any trials in the UK too? I also have RPGR XLRP and would be very interested in finding out more.
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