People with Severe Type 1 Diabetes are Cured in Small Trial of New Drug (courant.com) 63
"A single infusion of a stem cell-based treatment may have cured 10 out of 12 people with the most severe form of Type 1 diabetes," reports the New York Times.
"One year later, these 10 patients no longer need insulin. The other two patients need much lower doses." The experimental treatment, called zimislecel and made by Vertex Pharmaceuticals of Boston, involves stem cells that scientists prodded to turn into pancreatic islet cells, which regulate blood glucose levels. The new islet cells were infused and reached the pancreas, where they took up residence. The study was presented Friday evening at the annual meeting of the American Diabetes Association and published online by The New England Journal of Medicine...
Patients in the study began to need less insulin within a few months of being infused with new islet cells, and most stopped needing the hormone altogether at about six months [said Dr. Trevor Reichman, director of the pancreas and islet transplant program at University Health Network, a hospital in Toronto, and first author of the study]. He added that patients' episodes of hypoglycemia went away within the first 90 days of treatment.
If the study continues to show positive results, the company expects to submit an application to the FDA next year. "For the short term, this looks promising" for severely affected patients like those in the study," said Dr. Irl B. Hirsch, a diabetes expert at the University of Washington who was not involved in the study. But patients in the trial had to stay on drugs to prevent the immune system from destroying the new cells. Suppressing the immune system, he said, increases the risk of infections and, over the long term, can increase the risk of cancer... Patients may have to take the immunosuppressant drugs for the rest of their lives, the Vertex spokesperson said.
"One year later, these 10 patients no longer need insulin. The other two patients need much lower doses." The experimental treatment, called zimislecel and made by Vertex Pharmaceuticals of Boston, involves stem cells that scientists prodded to turn into pancreatic islet cells, which regulate blood glucose levels. The new islet cells were infused and reached the pancreas, where they took up residence. The study was presented Friday evening at the annual meeting of the American Diabetes Association and published online by The New England Journal of Medicine...
Patients in the study began to need less insulin within a few months of being infused with new islet cells, and most stopped needing the hormone altogether at about six months [said Dr. Trevor Reichman, director of the pancreas and islet transplant program at University Health Network, a hospital in Toronto, and first author of the study]. He added that patients' episodes of hypoglycemia went away within the first 90 days of treatment.
If the study continues to show positive results, the company expects to submit an application to the FDA next year. "For the short term, this looks promising" for severely affected patients like those in the study," said Dr. Irl B. Hirsch, a diabetes expert at the University of Washington who was not involved in the study. But patients in the trial had to stay on drugs to prevent the immune system from destroying the new cells. Suppressing the immune system, he said, increases the risk of infections and, over the long term, can increase the risk of cancer... Patients may have to take the immunosuppressant drugs for the rest of their lives, the Vertex spokesperson said.
Off Insulin onto immunosuppressants for life.... (Score:3)
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How are you controlling your diabetes? Are you able to control it, do you use an artificial pancreas? If so how is that working out? Be wary that one of the participants in the study died of cryptococcal meningitis which affects immunocompromised people.
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For any treatment, the question is always over whether the cure is better than the disease. I've been immunosuppressed going on 7.5 years now on account of a kidney transplant. From what I've seen of dietary restrictions of diabetic patients, they're almost as bad as dietary restrictions of kidney patients (kidney patients can't simply rely on food labels, they have to rely on a lot of guess work, and there aren't any finger stick measurements for potassium) but in addition to that, diabetic patients consta
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Most diabetics use a base long acting insulin. Insulin Glargine has a 24 hour action time, Insulin Degludec is 42 hours... So it's a once a day thing, and adjustments play out over days...
I have a relative that's been a kidney transplant patient since early childhood. They're in their 30's now, on their 3rd kidney. The suppressants aren't perfect, the match can be close, even familial, and the immune system slowly kills the organ. But here's the real kicker... They're now battling a blood cancer that ar
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Most diabetics use a base long acting insulin. Insulin Glargine has a 24 hour action time, Insulin Degludec is 42 hours... So it's a once a day thing, and adjustments play out over days...
It is emphatically not a once-a-day thing.Type 1 diabetics (who aren't on an insulin pump) use both a base long acting insulin AND a rapid mealtime/correction insulin. This can be 10+ shots per day, and requires constant vigilance to keep blood glucose in range. The insulin pumps use only rapid insulin, but have other downsides; scarring, internal 24/7 exposure to a teflon cannula, and 100% dependency on the technology working properly at all times to keep the patient safe. If the continuous glucose monitor
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It is emphatically not a once-a-day thing.Type 1 diabetics
Let me qualify... I have no experience with Type 1, only Type 2. I appreciate the info.
T
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To reduce the chance of this happening, most pumps are set to maintain an unhealthily high blood sugar level, which can have its own long term health consequences.
And they can be pretty nasty at that. Long-term, you slowly (but surely) lose peripheral nerve function (neurosis) renal function, eyesight, brain function, congestive heart disease, toes, even limbs if it gets bad enough.
I'd take the immunosuppressant, but that's just me.
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I have a relative that's been a kidney transplant patient since early childhood. They're in their 30's now, on their 3rd kidney. The suppressants aren't perfect, the match can be close, even familial, and the immune system slowly kills the organ. But here's the real kicker... They're now battling a blood cancer that arises from being immunosuppressed for decades. Supposedly it's a type of cancer that responds well to treatment, but... The battle must be fought.
Might be lymphoma, possibly caused by chronic EBV. I've been told I have chronic EBV and to watch for certain signs that it could turn cancerous. But there's no hard data on how often that happens as there have not been any studies. The bigger concern is actually melanoma.
Anyways, being on the third kidney tells me that whoever this is has been on dialysis a few times. I never had to do dialysis (waited 3.5 years for a cadaverous donor, maintained >17 eGFR throughout that period.) You can get away with d
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Might be lymphoma, possibly caused by chronic EBV. I've been told I have chronic EBV and to watch for certain signs that it could turn cancerous. But there's no hard data on how often that happens as there have not been any studies. The bigger concern is actually melanoma.
I believe it's one of the lymphoma's, H or non-H, but I don't know which one. And yea the skin cancer rates on transplants are a big deal too.
Anyways, being on the third kidney tells me that whoever this is has been on dialysis a few times.
They're actually one of the original infant dialysis patients, where they installed a shunt, and filled the abdomen with fluid, and then drained it back out. The original root cause was a congenital defect, a blockage that couldn't be imaged at the time. A day or two of dry diapers, and it was too late... I'm under the impression imaging has gotten so much better i
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Is there any correlation with family transplants?
No idea. It's only ever found out when the recipient just stops taking their medication for whatever reason, which needless to say is a very bad idea.
He worked in a chemical plant and the stuff he was manufacturing turns out to be one of the worst kidney toxins I've ever researched, styrene.
You sure it was styrene? Everybody gets exposed to trace amounts of it often, especially if you consume coffee or cinnamon. Compare cinnamaldehyde with styrene for example, it's damn near the same molecule.
Re:Off Insulin onto immunosuppressants for life... (Score:5, Informative)
I agree that this therapy is not without significant risks, so it's not to be taken lightly.
That said, the long-term health outcomes of T1DM are also significant. So the way I see this development is that it is one more step on the path toward finding a durable, safe, and effective cure. And if approved, it may offer some patients another choice, one that of course should involve an informed discussion with competent healthcare providers.
It's important to keep in mind that healthcare is not a "one size fits all" thing. Two patients that have the same condition can respond very differently to the same therapy. Before the discovery of insulin, diabetics literally just...died. So on the path to understanding this relationship between the individual patient and the selected therapy, medical science can only offer a range of treatment options. At one time, humans believed in bloodletting, lobotomies, and arsenic to treat various illnesses. We built leper colonies. And in some places in the world, menstruation is still considered "dirty." We have made many advances, but there are still many more to be discovered.
Re:Off Insulin onto immunosuppressants for life... (Score:4, Insightful)
If they used the patient's own stem cells as the initial source, then the immune-suppressants wouldn't be needed, but that would DRASTICALLY raise the cost.
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If they used the patient's own stem cells as the initial source, then the immune-suppressants wouldn't be needed, but that would DRASTICALLY raise the cost.
Ironically this is not the case.Type 1 Diabetes by definition is an autoimmune attack by the body on its own cells. Using more of those same cells won't stop the immune attack. It's possible that a more selectively targeted immune suppressant may be sufficient to use if it's the patient's own cells; e.g. the calcium-channel blocker Verapamil has been shown to slow down the attack, but periodic re-infusions of beta cells would probably still be necessary.
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Well, it's not a complete answer, but it's a large part of the answer. There's work in process on retraining the immune system to avoid autoimmune diseases that would also need to succeed. But even so, customized treatment with the patient's own cells would drastically raise the costs at just about every step of the operation.
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The immune system problem can be fixed by using each patient's own stem cells.
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The immune system problem can be fixed by using each patient's own stem cells.
Type 1 Diabetes is thought to be caused by an autoimmune reaction that destroys the cells in the pancreas that make insulin, so immune-suppressants would be needed anyway. From Type 1 Diabetes [cdc.gov]:
Type 1 diabetes is thought to be caused by an autoimmune reaction (the body attacks itself by mistake). This reaction destroys the cells in the pancreas that make insulin, called beta cells. This process can go on for months or years before any symptoms appear.
Some people have certain genes (traits passed on from parent to child) that increase their chance of developing type 1 diabetes. However, many of them won't go on to have type 1 diabetes even if they have the genes. A trigger in the environment, such as a virus, may also play a part in developing type 1 diabetes. Diet and lifestyle habits don't cause type 1 diabetes.
The main problem here seems to be that people can have the genetic defect and not have any symptoms until that defect is triggered, like with psoriasis (which I have). I imagine it would be possible to detect this early, if they even know which gene defect it is, and start giving people immune-suppressants to prev
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Swapping one drug for another, and the second one can lead to direct cancer. Seems smart.
Although... people do this all the time. All those biologic medicines advertised on TV to treat autoimmune conditions like Crohn's disease, rheumatoid arthritis, psoriatic arthritis, psoriasis, and some types of cancer etc... work by suppressing parts of the immune system. The list possible side-effects almost always include, "... tuberculosis and death." Okay, those are super rare, but, for me anyway, my psoriasis isn't bad enough to risk those.
Biologic Medications (Drugs) and Side Effects [webmd.com]
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If you had bothered to read the article, you will see this was given to hypoglycemic unaware diabetics. These people have zero warning that their insulin levels are low, they don't get shakes, they don't sweat, they just suddenly pass out, have a seizure, or possibly die.
Having to take a drug that might make you sick later is a pretty good upgrade from having to take a drug that you could literally die without warning if you got the variable dosage wrong, or from a bad batch of test strips (my friend went t
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Yeah a friend of mine at university died from that type of T1 diabetes. Prior to that. he was partly blind, and had damage to various organs.
Diabetes 1 does not mess around, its a serious medical emerhency that *will* kill if left untreated.
This is a potentially epic treatment if it manages to avoid the autoimmunge response, and that could lsave the health system billions of dollars, hundreds of millions per year.
Stem cells? (Score:1, Informative)
Not in Christofascist america. You'll buy your insulin at maximum gouge prices while saluting from your $10k a day hospital bed.
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You are right on the "cristo" part. Trump is not a Christ, he is an atheist that has no honor and integrity and hence is not above pretending. There are more fake "chistians" in his administration as well. But the "fascist" part is true, even if it is only in its early stages. That can happen when about half of the voters are as dumb as bread.
Re: Stem cells? (Score:2)
Two thirds of the voters are that dumb, as the ones who didn't vote are also idiots. The only message you send by not voting is that you will roll over.
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Indeed. Not voting is also voting. Obviously, the US has made it hard for many people to vote and that is one factor in the current problems. But even if it is hard, you become complicit if you do not vote.
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Well, when the only choices we're allowed to vote for are 'fast fascism' or 'slow fascism' it's easy to understand why a lot of people don't vote. As someone here said in 2016, "If two people are sawing off my legs why do you think I should thank the one who's doing it slower?"
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Remember when Kamala was VP and she was having people snatched from the streets and even courthouses? Or that time she pledged "no more wars" and months later started bombing Iran? Wait, I don't recall that either.
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In all fairness, she didn't get the chance, but she said that she would not change policies from those followed by Biden in any noticeable way. He was having people "snatched off the street" in anti-genocide protests, and they both fully supported the Gaza genocide and ethnic cleansing.
Why not use the patients own cells? (Score:2)
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I was wondering the same thing. Probably a hell of a lot more expensive and complicated. But I bet that is the direction this is going.
Suppressing the immune system "forever" is a very bad thing, probably as bad as diabetes.
Re:Why not use the patients own cells? (Score:4, Insightful)
Using the patient's immune cells won't work. They got diabetes because their immune system destroyed its own cells. The clearest viable path is a technology called encapsulated beta cells. See my other comment for references.
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Using the patient's immune cells won't work. They got diabetes because their immune system destroyed its own cells. The clearest viable path is a technology called encapsulated beta cells. See my other comment for references.
This is true, for now. Targeted immunosuppression therapies may eventually make patient-derived cells a more viable option; e.g. Anokion is currently working on "inverse vaccines" [anokion.com] for T1D, which in conjunction with patient-derived stem-cell-based beta cells may provide an effective cure. Verapamil (a calcium-channel blocker) has also shown effectiveness in slowing down beta cell destruction, without broadly compromising the immune system. That sort of drug, combined with periodic stem-cell reinfusions, coul
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I would like to see the Anokion tech succeed, because it would be broadly applicable. It will be interesting to see which comes to market first .. tolerizing vaccines, or encapsulated beta cells.
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Interesting point. So this really is an early experiment, not a "trial" or a "cure".
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Seems like that would solve the immunity problem.
It's not (necessarily) about the foreign cells, but the genetic autoimmune condition, triggered by something, that caused their Type 1 Diabetes in the first place.
From this CDC page on Type 1 Diabetes [cdc.gov]
Causes
Type 1 diabetes is thought to be caused by an autoimmune reaction (the body attacks itself by mistake). This reaction destroys the cells in the pancreas that make insulin, called beta cells. This process can go on for months or years before any symptoms appear.
Some people have certain genes (traits passed on from parent to child) that increase their chance of developing type 1 diabetes. However, many of them won't go on to have type 1 diabetes even if they have the genes. A trigger in the environment, such as a virus, may also play a part in developing type 1 diabetes. Diet and lifestyle habits don't cause type 1 diabetes.
Not a doctor, but I'm guessing it's like Psoriasis (which I have), which is an autoimmune issue enabled by a genetic defect; people can have the defect, but no symptoms until triggered by something, like an injury. This is why drugs and treatments that suppress the immune system work on psoriasis. The di
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It's not (necessarily) about the foreign cells, but the genetic autoimmune condition, triggered by something, that caused their Type 1 Diabetes in the first place.
Yeah, potentially. That said, it might be possible to train the immune system to ignore those cells by repeated injection of whichever protein is triggering the immune response in combination with phosphatidylserine [sciencedirect.com]. This approach seems more likely to be successful if it is done with the patient's own stem cells, rather than a potentially highly alien cell line. :-)
Then again, if that approach works and you catch the diabetes early enough, it could potentially stop type 1 diabetes progression entirely wit
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1) That would DRASTICALLY raise the costs.
2) That wouldn't eliminate the need for immune-suppressants, though it might well drastically lower it. Since type 1 diabetes is probably caused by the immune system attacking the beta cells, that would still need to be suppressed somehow. But the suppression could be more tightly targeted. (Actually, there are signs that the immune system can be retrained...but I believe those are still at the "lab experiment" stage.)
A better solution is coming (Score:5, Interesting)
Encapsulated beta cells. No immunosuppressants needed. Immunosuppressants for life may be worse than diabetes because there are already good "artificial pancreas" systems (continuous glucose monitoring with a Dexcom G7 + a Tandem Insulin pump). The full solution though will be encapsulated beta cells.
Re:A better solution is coming (Score:5, Informative)
Here are a couple of references about encapsulated beta cells tech that I mentioned:
A readable article that describes the technology in detail: https://advanced.onlinelibrary... [wiley.com]
Here's a couple articles about a couple of the companies (there are about 7, outside of academia) working on development and testing (which takes about a year or two per iteration to watch for failure etc.) of encapsulated beta cell technology:
Major company: https://pharmaphorum.com/news/... [pharmaphorum.com]
Startup: https://www.pharmavoice.com/ne... [pharmavoice.com]
Re: A better solution is coming (Score:2)
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I think I first heard of the "encapsulated beta cells" approach over a decade ago. It still sounds like a good idea, but there appear to be lots of "implementation problems".
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They've been working on this or something like that since I was a kid. For reference: I'm 52 now; 51 of those with T1D. The encapsulation idea isn't new at all, it's just not been scientifically feasible.
First step. (Score:2)
This is the first step toward a full cure for everyone with Type 1 diabetes. With our increasing ability to manipulate genes, it may come to the point where they grow a batch of modified cells that are based on patient's DNA so that no immunosuppressive drugs are needed. If you're a billionaire then this is within reach but for the rest of us, it will take some time.
The future is already here... it’s just not very evenly distributed.
Re: First step. (Score:2)
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That is grossly underestimating things. At that price-point, you cannot even identify stemcells.
Re: First step. (Score:2)
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The future is already here... it’s just not very evenly distributed.
Given observable reality, this is not likely to change. There are just too many assholes that desperately need to mave mich, much, much more money than everybody else. Hence this will become available to the general population only if it becomes dirt cheap.
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Or if it's developed in China, where gouging for healthcare is prohibited. Already a lot of new drugs are being developed in China, licensed by US Pharma, produced mostly in China, imported to the US, and sold for 10-100 times the price.
https://open.substack.com/pub/... [substack.com]
I heard Elon Musk is looking for (Score:3)
The stem cells for personality
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Unfortunately, doctors determined that the treatment would kill him.
Better sell (Score:2)
So not their own stem-cells... (Score:2)
That is not a "trial", that is properly called an "experiment" and in a sense it was done on the cheap, to the detriment of the experimental subjects. Yes, it is possioble that staying on immunosupressants is less bad medically, but it definitely is not a "cure". It is replacing one evil with a smaller (?) different one.
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Good job, showing him how it's done. The positivity balance for Anonymous Coward is worse.
gweihir nice tag line, sorry I couldn't resist! Apparently AC took offense!
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Hahaha, thanks. AC = fuckup with no insight and no honor or decency. They have standpoints so broken that they are not worth even considering and they are too cowardly to even give you a pseudonym to respond. The value of people does not get much lower than that.
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Neither the summary, nor the article, nor the linked study, make it clear that the stem cells came from the patients themselves.
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That's because they did not come from the patients themselves. See my post above.
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Thanks, I did not know the meaning of allogeneic, that does indeed make it clear.
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The stem cells were from a separate donor. The very first sentence of the study's abstract is: "Zimislecel is an allogeneic stem cell–derived islet-cell therapy."
allogeneic: denoting, relating to, or involving tissues or cells that are genetically dissimilar and hence immunologically incompatible, although from individuals of the same species.
Infused (Score:2)
> The new islet cells were infused and reached the pancreas, where they took up residence
This is pretty amazing - assuming they only took up residence in the pancreas.
It could be a major facet of affordable immunotherapies.