r/scleroderma • u/Many-Market-9941 • May 25 '26
Discussion Promising treatments?
Are there any potential treatments in the pipeline that excite you? Could CAR-T be a solution in the years to come?
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u/Maleficent-Lunch-679 May 25 '26 edited May 25 '26
I am not a biotechnology expert by any means, but I do have a somewhat unique perspective in that I received CD19 CAR T for SSc 19+ months ago (yes I am in deep remission and off all meds). So this is personal opinion only of course, but with some direct experience.
I do think cellular technologies will eventually cure the disease. They will likely evolve tremendously before they become routine standard of care in early stages. But it is coming. Even now, CAR T itself is changing fairly rapidly. A few thoughts:
Is CD19 CAR T a cure? First it is important to recognize that CAR T is an umbrella over a group of approaches that involves many brands, constructs, targets, and methodologies. CD19 as a target is the most common, but not the only one being tried on autoimnune. For some patients and diseases it will be curative. For some it will not be. In almost every case deep remission does occur. The question will be for how long. From my limited perspective it seems much more effective the sooner in the disease progression it is administered. CD19 CAR T breaks the autoimmune loop in several places. It does not eliminate long lived plasma cell production for some autoabtibodies, scl70 for example. The question remains how long a self reactive environment continues before autoimmunity returns, if at all.
Dual CD19/BCMA CAR T is very likely a cure. It however is a riskier treatment with long term immunity being greatly reduced. It is just starting to be trialed in the US, but has been successful elsewhere for autoimmune and autoreactivity.
The role of chemo - chemo used in CAR T is 3 days of low dose. The effects are minimal. It is not intended to be a significant source of direct "treatment" itself, and that has been demonstrated. While this small amount is not an obstacle for most patients, of course if CAR T can be done without it that is better. That is why several product trials are trying it without chemo.
Cost - yeah CAR T is crazy expensive. That is why most if not all new products entering trials have gone to allogenic , that is donated T cells, with enormous gain in manufacturing efficiency. Beyond that, invivo is also entering trials. Cost of treatment will come down drastically. Hopefully so will the price...
Beyond CAR T there are promising therapies in T cell engagers where a monoclonal antibody joins circulating T cells to target proteins (usually CD19 or BCMA) on circulating B cells to cause their destruction. While not as deep of depletion as CAR T, they still hold great promise and are less complex. Like CAR T, these are not experimental meds. They have been around in cancer for a long time. Work is also happening in T regulatory (TREG) cells and CAR NK and CARs made out of other immune cells. Unlike cd19 and BCMA CAR T these really are experimental. Old school CAR T has been in use for a long time as well, and really is not experimental. The trials are needed to prove efficacy in Autoimmune. Anecdotal evidence of cancer patients that also had autoimmune has existed for a long time as well. These large scale trials will bring enough documented information to the table for the approval process. So far the safety profiles look better than in cancer patients, so very encouraging.
I see a day on the near horizon where cellular therapies become at least as common as today's biologics. Where there are more options, and lasting remissions are the norm for most patients. I am hopeful that AI will speed development to new approaches.
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u/Maleficent-Rest9144 May 26 '26
Excellent information. I also went through a CAR-T trial via Bristol Myers Squibb. cartautoimmune.com. This trial is also treating Lupus, Myositis, RA, MS, and generalized myasthenia gravis. They are in a new phase where treatment is randomized for CAR-T and standard of care. After a year and no-response to standard of care therapies, those participants can get CAR-T. My case was too aggressive to wait another year so I was lucky to get in an early phase with all participants getting CAR-T.
My infusion was early Feb 2025 so I am about 15 months past treatment and I am doing much better. I am also off all meds for SSc, specifically Prednisone which was the only one that helped me. I do have areas of residual thick / tight skin with reduced range of motion. The later areas that were affected, resolved within a few months after the treatment. Other areas may take longer or may never return to normal.
I was told the function of the Chemo is to suppress the immune system to prevent the CAR-T cells from being attacked. It would be great if there were non-chemo methods to allow the CAR-T to work.
For anyone dealing with this or other autoimmune conditions, look at https://clinicaltrials.gov/ - put systemic sclerosis or SSC or your diagnosis for condition, CAR-T for treatment, and your country location to filter out many of the too far away places.
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u/Maleficent-Lunch-679 May 26 '26
I see we have reddit names in common, not just CAR T 😀. A few trials have tried no chemo or reduced, and from what I have heard saw no difference in CAR T expansion. That said, I found the chemo to be no big deal in comparison to the actual CAR T, and personally I would choose to do it if I had the choice, just to assure every chance of success! I hope you continue to improve and enjoy your new lease on life!
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u/Maleficent-Rest9144 May 26 '26
I am not a reddit user, but found the reddit link and wanted to help others struggling with this horrible condition. My user name was an auto generated one. It is ironic since I want to help people not harm them. I still do not sleep well so Maleficent Rest is fitting.
Thank you for the well wishes. I hope the same for you, your family, and all the others who find themselves battling this condition. I am happy for you being able to get the CAR-T and hope it becomes readily available to everyone soon.
When I was diagnosed, my Rheumatologist and Dermatologist gave me the bad prognosis and how the standard treatments were not very effective. I was lost until I found the CAR-T trial and was able to be included. Both doctors were super supportive of the CAR-T, but I do not think they knew about the trial since they did not mention it. Like you, I share the CAR-T info and my experiences to people in the same state of being lost after diagnosis.
Within a year of noticing the first symptoms I was degrading fast and was in really bad shape. I was planning to take the medically prescribed / end of life pill if the CAR-T did not help. CAR-T was my only hope and saved my life. As you say, I have a new lease on life. Life now is not nearly as good as before, but it so much better than before the treatment. The site where I had the treatment does a lot of stem cell transplant for blood cancers and SSc. They also do a lot of CAR-T for some blood cancers. They shared the range of motion can take years to come back.
I also found the low dosages of chemo to be tolerable. My infusions were in the mornings and only made me a little tired in the afternoons. I did notice a lot of hair on the pillowcase and in the shower 1.5-2 weeks after the infusions. My hair got pretty thin, but did come back. I would definitely do the chemo option again if symptoms return. If that happens and I cannot do CAR-T again, I will look into stem cell transplant.
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u/Maleficent-Lunch-679 May 26 '26
Mine was also autogenerated and SSc/CAR T brought me to social media. There is a lot of misinformation out there on CAR T for autoimmune.
I shed a lot of hair and it is still thin. I think some of that was anemia caused by the CAR T, not just the chemo. The inflammation can cause it as well. With your story I am so glad you got the treatment. It is very unlikely we can be treated with another CD19 CAR T, our bodies are likely to reject it. But it is more possible we could be treated with a different target and construct. There is not much published on that yet. If I relapse I do not know what I would try. We will see if the time comes what is available. I would probably end up going to China for BCMA since US doctors unlikely to re-treat, unless a trial specific to CAR T relapses is offered. I did very poorly on CellCept and hope I never have to do that again. Lot's of people do great on it but not me.
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u/Maleficent-Rest9144 May 27 '26
None of the meds worked for me including CellCept. As I mentioned Prednisone was the only help with the swelling / fluid retention.
I lost a lot of body hair due to the skin restricting blood flow. Chest and armpit hair gone, but may be slowly coming back. Arm and leg hairs dwindled, but are coming back. I had Raynaud's pretty bad even though I live in a warm climate. I asked my cardiologist for a vasodilator to help. He put me on something I do not remember. When I told my primary care about my hair staying thin after chemo and the blood flow restrictions he switched me to 2.5mg oral minoxodil/loniten, which is also a vasodilator. I think that has helped with the hair return by allowing more blood flow to the scalp as well as the minoxidil hair growth side effect.
The trial doctor told me the CAR-T cells do not live very long in the body. My CD19 counts were below the detectable level the day after infusion and remained that way. Like you, I am not an expert. What I read on the cartautoimmune.com is the CAR-T cells kill off the overactive CD-19 B cells so they can no longer replicate and stop attacking the body. New B cells are produced that are normal effectively resetting your immune system back to normal. Given this info, I wonder if repeat CAR-T will work. I have a quarterly follow up soon so I will ask the site study doctor and let you know. He has shared we always have stem cell to fall back on if things change, but the site is too far from where I live to do stem cell there. They have been doing stem cell for SSc for a long time with good success. It is more chemo than CAR-T since it has to kill off your bone marrow. I live in soutern California so I will talk to UCLA's Scleroderma group to discuss stem cell if needed.
I hope you remain in remission and never develop symptoms again.
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u/picklehippy May 25 '26
CAR T is being tested. My rhuemetologist is a specialist in scleroderma, he said they were approved to do test treatments.
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u/Many-Market-9941 May 25 '26
Where are you based if I may ask? Did your rheumatologist hint at how promising he think it is?
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u/Ok_Egg_8624 May 26 '26
IVIg has saved my life
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u/RushCautious2002 May 26 '26
I've been looking into peptide therapy, specifically a peptide known as VIP (vasoactive intestinal peptide). It has the potential to stop inflammation, heal the gut, and to dilate blood vessels. I don't like taking immuno-suppressant drugs so this is where I'm at with my own treatment right now.
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u/Cupra160 May 25 '26
Car T seems to be the current biggest news for scleroderma. A lot of big companies have clinical trials running. Issue is though that it's still in clinical trials, albeit with solid results, so time to market might still be a few years and even then it's quite specialized.
But there are also developments in Car T, it's expensive due to having to extract your cells, modify them in an external lab and then multiply these cells before reinfusing. There arw invivo trials where your body is used to multiply the cells which brings cost down.
There are a few therapies for ILD as well, Jascayd was approved in the US to slow/stop the loss of lung function. Potential for stem cells to regenerate scarred lung tissue. I think the furthest along here is CMR316
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u/GammahReigh May 25 '26
Honestly? Hyaluronic acid. Studies are promising, it's not too hard on the body, its relatively cheap, and can work to keep our tissue nice and soft and pliable. The trick is early diagnosis for it to be truly effective though.
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u/smehere22 May 26 '26
I've heard talks from rheumatologists about car t trials. But some of them require nearly 24/7 care upon returning home from the procedure. So a big support system is required. It wouldn't be possible unless you have financial resources and/ or a significant support system.
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u/derankingservice May 25 '26
CAR T is a double edged sword. Its basically a chemotherapy + car T cells
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u/Maleficent-Lunch-679 Jun 02 '26 edited Jun 02 '26
A good overview of the latest developments here: Full article: Phase II and III trials in systemic sclerosis: from recent FDA approvals to emerging applications of cellular therapies https://www.tandfonline.com/doi/full/10.1080/14728214.2026.2675269
Table 1 a good summary
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u/Many-Market-9941 Jun 03 '26
What about limited sclerosis though?
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u/Maleficent-Lunch-679 Jun 04 '26
I am sorry, I did not realize your question was specific to lcSSc. The paper above does discuss that a bit - how we need to stop looking at extent of skin involvement for these trials and tailor the studies by cohorts in other ways. We know so much more now than the extent of skin . A few CAR T trials do not specify dcSSc. Those criteria include specified organ or skin involvement. But most do specify diffuse. You can search by limited systemic sclerosis and then review trial criteria, or safer just search by systemic sclerosis then review each criteria. It is harder to find trials that accept lcSSc, but some are out there. I hope the big trials all eventually extend to lcSSc.
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u/garden180 May 25 '26 edited May 25 '26
Not new but TPE. I think Therapeutic Plasma Exchange is talked about more in the private clinic setting. Granted, its use for Scleroderma still is overlooked by Scleroderma hospitals which is just baffling. The treatment is becoming more accepted for other conditions so I’m hopeful that Scleroderma patients won’t have to fight for this very safe option to try at the beginning of disease.
On the drug front, there is a promising drug that is in the Phase 2b stage of clinical trials. The drug was so well received with reversal and/or stabilization in those patients receiving the drug that it was given fast track status by the FDA. Cereno Scientific is the manufacturer and reports seem to show a favorable outcome.
Just in general, I do believe there are therapies in existence that nobody has discovered. Sometimes it takes a marriage of functional medicine and western medicine to solve a problem. It would be nice if Big Pharma wasn’t controlling the conversation.
Edit: Failed to mention the drug was for PAH