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Medicine

Stem Cell Therapy Frees Woman From Diabetes (washingtonpost.com) 81

Amanda Smith, a 35-year-old nurse from London with Type 1 diabetes, "is at the forefront of a medical experiment that seeks to treat the root cause of diabetes by replacing the cells the disease destroys," reports Carolyn Y. Johnson for the Washington Post. "On Valentine's Day 2023, doctors transplanted replacement islet cells, grown in a lab from embryonic stem cells, into a blood vessel that feeds Smith's liver. By August, she no longer needed insulin. Her new cells were churning it out." From the report: Smith is one of a dozen patients who have received a full dose of islet cells generated in a laboratory from stem cells. Eleven of the patients in the clinical trial drastically reduced taking insulin or stopped altogether, according to data presented at an American Diabetes Association meeting in June. Despite the promise, the therapy developed by Vertex Pharmaceuticals remains in early stages, and many experts consider it a major step forward, not the finish line.

No one knows how long these cells will keep churning out insulin or whether the therapy is safe long-term until it is tested and followed up in more patients, who must take immune-suppressing drugs to prevent their body from rejecting the foreign cells. One patient died of an infection caused by a complication of sinus surgery, highlighting the risk of immunosuppressive medications, which were among the factors contributing to the patient's death. [...]

Smith credits her insulin pump with keeping her alive but was elated to banish it to the back of a kitchen cabinet. She no longer has to plan her life around her illness. "I pray this gets to everyone," Smith said. "My life has changed."

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Stem Cell Therapy Frees Woman From Diabetes

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  • Wilford Brimley is stirring. DIAAAA....BEAT.....US!

    • I'm sure you're aware, but Wilford Brimley's "diabeetus" refers to Type 2, a metabolic disorder. This article (and this therapy) is specific to Type 1 diabetes, an autoimmune disorder. (Technically this type of therapy might also be helpful for some Type 2 cases, but it's initially focused on type 1.)

  • by wildsurf ( 535389 ) on Tuesday August 27, 2024 @03:50AM (#64738690) Homepage

    My 6yo daughter has T1D, and we banked her cord blood when she was born. If her own stem cells could be used to generate these insulin-producing cells, it would avoid the need for broad-spectrum immunosuppressive drugs. (Although the autoimmune attack itself would also need to be selectively suppressed to prevent the T1D recurring.) Holding out hope that these therapies start to become available sooner than later!

    • by Skinkie ( 815924 ) on Tuesday August 27, 2024 @04:47AM (#64738770) Homepage
      I think there are multilpe things to consider. If this is a genetic defect, ones own stemcells must be first repaired, that may be a crispr-cas9 kind of technique. And the stemcells must be put in an envionment where they start to produce Islets of Langerhans. I wonder why the article mentions immunosuppressants, in case they are the stemcells created from skin, they are body native.
      • by silentbozo ( 542534 ) on Tuesday August 27, 2024 @04:56AM (#64738790) Journal

        The treatment in the article is derived from embryonic stem cells taken from a donor. So they trade needing an insulin pump and constant monitoring for low blood sugar for taking immunosuppressants and risking complications related to a less active immune system.

        But this does get you closer to the day when they can re-engineer your own stem cells to fix the problem...

        • by Barny ( 103770 )

          As someone with Crohn's disease, who is on immunosuppressants and has to deal with a craptacular immune system, I can say this is likely a far less terrible fate than type 1 diabetes. There are some neat new drug therapies where using azathioprine and allopurinol together, in much lower doses, can tank the immune system without eventual liver damage.

          • by rykin ( 836525 )
            Crohn's disease can be treated with the carnivore diet. Same with ulcerative colitis. I've read many testimonies from others about it curing these issues and have personally resolved the chronic stomach issues that I had by following the diet for 6 months. I think you should look into it.
            • by Barny ( 103770 )

              I wish I had a dollar for every time someone told me an autoimmune disease could be cured with diet change.

      • I wonder why the article mentions immunosuppressants, in case they are the stemcells created from skin, they are body native.

        From the article

        Instead of transplanting the whole organ, scientists wondered, what if they could use islets harvested from organ donors?

        Appears to be cells from another person.

      • I think there are multilpe things to consider. If this is a genetic defect, ones own stemcells must be first repaired, that may be a crispr-cas9 kind of technique. And the stemcells must be put in an envionment where they start to produce Islets of Langerhans. I wonder why the article mentions immunosuppressants, in case they are the stemcells created from skin, they are body native.

        Because Type 1 (Juvenile) diabetes is in the autoimmune family of diseases. The pancreas is attacked by your own body because, for reasons no one yet understands, when some kids are in a given age range, suddenly their immune systems see the pancreas as an enemy.

        Before I even mention this to my own son with T1, I want to see how these patients deal with the autoimmune issues. Is it worth it to get the procedure if you're getting sick all the time because of immunity issues?

      • If this is a genetic defect, ones own stemcells must be first repaired, that may be a crispr-cas9 kind of technique.

        The "genetic defect" (to the extent T1D has a genetic predisposition) relates to the immune system, not the beta cells themselves. So no "repair" would be needed to create workable beta cells this way.

        FWIW, the genetic susceptibility to T1D is present in about 1/4 of the population, but only 1-2% of those will ever develop the disease, based on unknown (likely environmental or probabilistic) factors.

        • by Skinkie ( 815924 )
          Have you got any insights why they would have started with embrionic stem cells if they seem know who they can force a stemcell into an islet?
    • Did you know before she was born that she had diabetes? If not, how did you think to save the cord blood? Granted, I had one kid, and that was nearly 40 years ago before saving cord blood was even considered, I'd imagine. Even so, we had no idea that there might even be a reason to do so.
      • by wildsurf ( 535389 ) on Tuesday August 27, 2024 @03:15PM (#64740672) Homepage

        Did you know before she was born that she had diabetes? If not, how did you think to save the cord blood? Granted, I had one kid, and that was nearly 40 years ago before saving cord blood was even considered, I'd imagine. Even so, we had no idea that there might even be a reason to do so.

        There is no T1D in our family histories; in fact, ironically, 23andMe had identified both of us (mother and father) as having substantially below-average risk for T1D. We banked the cord blood (in 2018) mostly on a "why not?" basis, thinking that it might possibly turn out useful for other family members in the future, or perhaps something we could donate for science or for someone else's use. We never expected that our daughter herself might benefit from it! And she can't (yet), but maybe one of these days.

  • I'll Pass (Score:5, Interesting)

    by jvp ( 27996 ) on Tuesday August 27, 2024 @05:00AM (#64738794)

    As a T1D patient of over 50 years, I'm happy to see these various attempts at cures. But until they can do it without the need for immuno-suppressants, count me out. As much as I hate this disease, I've managed it incredibly well with my pump, exercise, and eating well. I really don't look forward to a day when my diabetes are gone, but it's replaced by the fear that I could die from a silly infection that I'd otherwise fight off.

    I'll pass.

    • by e3m4n ( 947977 )

      In a way it would be like getting AIDS. I could not imagine volunteering for that. Imagine a sick joke were curing one disease meant contracting AIDS as the fix.

      • by HiThere ( 15173 )

        IIUC (I can't remember the disease) there *IS* a disease for which that is true...at least there's a large statistical correlation.

    • by Barny ( 103770 )

      That's what it will come down to. I've been on immunosuppressants (azathioprine) for 15 years now. While at times it can suck a big one (infections, flu, etc) it's not that terrible. QOL will always be a concern with it, though.

      • by jvp ( 27996 )

        While at times it can suck a big one (infections, flu, etc) it's not that terrible. QOL will always be a concern with it, though.

        I'm never sick. Well, almost never. I actually appreciate my immune system even if it's the cause of my T1D. Personally I'd rather not trade the devil I know for one I don't. The researchers are still hard at work trying to figure out ways to implant beta cells that can't or won't trigger the auto-immune system. I'd consider that the cure, and the only cure worthwhile. Until then: insulin pump, finger pricking, eating well, etc.

    • I am also T1D. I agree. Trading Insulin for Pills is a non-cure.

  • Whatever triggers the immune cells to attack is the root cause. And we still donâ(TM)t know, nor are we looking.

    • by HiThere ( 15173 )

      You are wrong when you say "nor are we looking". You may not feel we're looking in the right place, but lots of people are looking.

    • Some scientists, like Hanan Polansky think the answer is viruses. Some we inherit, some acquire. Its taboo to talk about because many of them have been label sexually transmitted diseases (like herpes).
  • It was probably something else and I can't be arsed to check.

  • Since when does diabetes destroy cells? With at least Type II, it's each cell's resistance building up from to too much insulin. Is this Type I and it works differently or something? What cells did they even replace? Pancreas?
    • It sounds like bad wording. Type 1, genetic/autoimmune diabetes, is the destruction of the islet cells in the pancreas that produce insulin.

      They really need to develop a method using the patient's own cells.
      "No one knows how long these cells will keep churning out insulin or whether the therapy is safe long-term until it is tested and followed up in more patients, who must take immune-suppressing drugs to prevent their body from rejecting the foreign cells."

  • This is type 1, an important point for anyone mixing it up with type 2. That is insulin resistance and low insulin mainly driven by obesity.

  • I recall that they experimented with implanting new islets into Type 1 Diabetics, way back in the late 1990s. They produced their own insulin for a while, but those islets were attacked by the immune system, and they had to return to insulin therapy after a few months. Replacing the cells doesn't address the core problem, which is the immune system attacking them. It seems like just suppressing the immune system should be good enough to start producing insulin again, but then you've just traded insulin inje

    • There has been significant research into finding ways to get the beta cells to regenerate themselves (e.g. https://www.ncbi.nlm.nih.gov/p... [nih.gov]), as well as "inverse vaccines" (e.g. Anokion: https://anokion.com/pipeline/ [anokion.com]) to selectively "untrain" the immune system to attack the pancreas. Also research into ways to physically shield transplanted beta cells from both immune rejection and autoimmune attack. (e.g. Vertex Pharmaceuticals: https://www.vrtx.com/our-scien... [vrtx.com])

      Meanwhile, there are selective, relatively

      • as well as "inverse vaccines" (e.g. Anokion: https://anokion.com/pipeline/ [anokion.com]) to selectively "untrain" the immune system to attack the pancreas.

        This is the strategy that I feel like is the most promising. Maybe implanting cloned islets will be necessary in the process, but this technique is attempting to fix the problem at its source. I'm eager to see how it progresses.

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