People with Severe Type 1 Diabetes are Cured in Small Trial of New Drug (courant.com) 16
"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:2)
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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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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 fi
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.
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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.
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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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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
A better solution is coming (Score:4, 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.
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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]
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.
I heard Elon Musk is looking for (Score:2)
The stem cells for personality