2024-11-07 07:06:00
She is the first in the world to suffer from type 1 diabetes and be able to produce her own insulin. A 25-year-old woman from China was treated for her type 1 diabetes using her own reprogrammed stem cells. A technique that has allowed him to eat sugar and produce his own insulin for more than a year. The results of this pioneering experiment were detailed in the journal Cell.
Type 1 diabetes is an autoimmune disease that appears spontaneously in children or adolescents. T lymphocytes (white blood cells) begin to identify the β cells in the pancreas as cells foreign to the patient’s body and eliminate them. However, these ß cells produce a hormone essential for regulating blood sugar: insulin. Without insulin, the concentration of glucose (sugar) in the blood is abnormally high, leading to complications in the heart and blood vessels. Irreversible until then, the type 1 diabetes is treated with insulin treatment that patients must take for life.
An isolated but promising case
All people who suffer from type 1 diabetes, except this young woman from Tianjin in China, who was able to benefit from a transplant of her own stem cells. These stem cells are neutral cells, still undifferentiated, which subsequently transform into specialized cells.
In the case of the young woman, stem cells were developed from already specialized cells, we then speak of induced pluripotent stem cells. Concretely, “the researchers took adipose tissue from the patient, which they transformed into stem cells using a cocktail of small molecules. It’s a real challenge. These stem cells were then transformed into pancreatic endocrine cells according to a very specific protocol.“, explains Professor Raphaël Scharfmann, researcher at Inserm and pioneer in the field of pancreatic ß cells.
Thanks to these new endocrine cells in the pancreas, the patient began to produce her own insulin three months after the transplant.
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Although this first is encouraging in the field of research against type 1 diabetes, it nonetheless remains an isolated case and does not suggest a treatment that would be available for all patients. “It’s not for everyone.”warns Professor Scharfmann.
First of all, we must remember the very particular profile of this patient: “In addition to her type 1 diabetes that appeared in adolescence, she suffers from parallel pathologies, has already had a liver and pancreas transplant and is taking immunosuppressive treatment [pour maximiser les chances de réussite de la greffe de foie]“, explains the specialist.
The researchers themselves say they do not know exactly why this technique finally worked in this patient but never in previous trials with other patients. The key element could be the immunosuppressive treatment for his liver transplant, which would have allowed better assimilation of his own reprogrammed cells.
Perhaps the innovative injection site – the abdominal muscles – also played a role, with transplants usually being done into the liver.
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Of the “factories“high performance insulin
Professor Scharfmann highlights the colossal work carried out on the quality of stem cells before the transplant. “After numerous quality checks, they were first transplanted into more than 200 mice, which is enormous. The genome of these cells was then sequenced for analysis and only then were they transplanted into the patient.”
In total, 2 billion cells were transferred to it. “That’s twice as much as we have in the pancreas. The β cells that produce insulin are veritable factories. We have 1 billion of these cells in our pancreas and each of them produces 6,000 insulin molecules per second.” The twice as high number of pancreatic endocrine cells could also have been decisive.
In any case, “this is a big step. Especially if we look at where we were 25 years ago“, comments the specialist. The team that followed this patient could well publish other results soon. The researchers said they wanted to extend the trial to a group of 10 to 20 other people.
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Stem cell therapy for diabetes success rate
**Interview with Professor Raphaël Scharfmann: A Pioneer in Stem Cell Therapy for Type 1 Diabetes**
**Interviewer**: Good morning, Professor Scharfmann. Thank you for joining us today to discuss this groundbreaking case in stem cell therapy for Type 1 diabetes.
**Professor Scharfmann**: Good morning! Thank you for having me.
**Interviewer**: Let’s dive right in. Can you explain what makes this case of the 25-year-old woman from Tianjin, China, so unique?
**Professor Scharfmann**: Certainly. This young woman is the first known case in which an individual with Type 1 diabetes has regained the ability to produce insulin independently after undergoing a stem cell transplant. The researchers utilized a method that involved reprogramming her own adipose tissue into induced pluripotent stem cells, which were then differentiated into pancreatic endocrine cells.
**Interviewer**: That sounds remarkable. What were the initial results following the transplant?
**Professor Scharfmann**: Remarkably, just three months post-transplant, she showed signs of insulin production. This allowed her to regulate her blood sugar levels without needing daily insulin injections, which is a significant breakthrough for her personally.
**Interviewer**: While this is an exciting development, you mentioned in your earlier comments that this case is quite isolated. Can you elaborate on that?
**Professor Scharfmann**: Yes, it’s important to note that this success does not indicate that we have a one-size-fits-all treatment for Type 1 diabetes. This patient presented a unique profile, including having undergone a liver and pancreas transplant previously, along with being on immunosuppressive medication to maximize the chances of the initial surgeries’ success. Our understanding of why this worked for her, but not for others, is still limited.
**Interviewer**: What are the implications for future research and clinical applications based on this case?
**Professor Scharfmann**: This case certainly opens up new avenues for research, particularly regarding the potential of using stem cell therapy to regenerate insulin-producing cells in the pancreas. However, we must conduct extensive studies to understand the mechanisms behind this success. Until we have clearer insights, it’s crucial to approach this as a promising but preliminary step in Type 1 diabetes treatment.
**Interviewer**: Thank you for shedding light on this fascinating development, Professor. Before we wrap up, what message do you have for Type 1 diabetes patients who might be following this story?
**Professor Scharfmann**: I want patients to remain hopeful but also to understand that we are in the early stages of research. While advancements are promising, the journey to finding a widely applicable treatment is ongoing. Staying engaged with your healthcare providers and understanding the latest research can be empowering.
**Interviewer**: Thank you, Professor Scharfmann, for your insights and for your work in this critical field. We look forward to hearing more about future developments!
**Professor Scharfmann**: Thank you for having me!
