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type 1 diabetes is an autoimmune disease in which the patient's pancreatic beta cells are destroyed, resulting in insufficient insulin secretion. scientists have long been looking for ways to effectively replace damaged pancreatic islet cells. recently, the team of shen zhongyang and wang shusen from nankai university/tianjin first central hospital and the team of deng hongkui from peking university/changping laboratory collaborated to publish a breakthrough research result in the top international academic journal "cell". for the first time, pancreatic islet cells differentiated from chemically reprogrammed pluripotent stem cells (cipscs) were used to successfully treat patients with type 1 diabetes.

pluripotent stem cells (pscs) are considered an important tool in the field of regenerative medicine because of their ability to self-renew and differentiate into a variety of functional cell types. in 2006, professor shinya yamanaka of kyoto university first induced induced pluripotent stem cells (ipscs) from human somatic cells. this discovery brought revolutionary changes to regenerative medicine. however, traditional ipsc preparation methods have some limitations, such as the risk of genomic integration. in 2022,beijing universityprofessor deng hongkui's team at the university of california, berkeley, developed chemically induced pluripotent stem cells (cipscs) by inducing human somatic cells to transform into pluripotent stem cells entirely using chemical small molecules. this new technology not only avoids the risk of genomic integration, but also has the advantages of easy manufacturing and standardization.

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the researchers used chemical small molecules to induce human somatic cells to transform into pluripotent stem cells (cipscs), and then used an optimized differentiation protocol to differentiate cipscs into pancreatic islet-like cells (cipsc-islets). these cipsc-islets are comparable to natural human islets in terms of transcriptome characteristics, composition, and insulin secretion function.

preclinical studies in nonhuman primates have shown that cipsc-islets can significantly improve diabetic symptoms without causingtumorin addition, the researchers found that transplanting cipsc-islets beneath the anterior rectus sheath resulted in better islet survival and maturation, with secretion levels of c-peptide (a marker of endogenous insulin secretion) approaching those of non-diabetic patients.

in this latest study, the research team conducted an exploratory clinical trial, the first use of a patient's own cipsc-derived islet transplantation to treat an immunosuppressed type 1 diabetic patient. the patient was a 25-year-old woman who was diagnosed with type 1 diabetes in 2012 and underwent multiple liver transplants and whole pancreas transplants, but ultimately had complications that led to the removal of the pancreas graft. in june 2023, she received a transplant of cipsc-islets.

after one year of follow-up, the patient no longer needed insulin treatment on the 75th day after transplantation. at the fourth month after transplantation, the target blood sugar range was achieved from 43.18% of the baseline value to 96.21%, and the glycated hemoglobin (hba1c) index also dropped significantly, reaching the long-term systemic blood sugar level of non-diabetic patients. since then, the patient's blood sugar control has been stable, with blood sugar reaching the target time for more than 98% and glycated hemoglobin level of about 5%.

clinical data from this year showed that all study endpoints had been achieved, with no abnormalities related to transplantation. the patient's quality of life had been significantly improved, he was no longer dependent on exogenous insulin, and his blood sugar was well controlled.

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this study is the world's first successful case of using pancreatic islet cells differentiated from chemically reprogrammed pluripotent stem cells (cipsc) to treat type 1 diabetes. the results not only confirm the safety and effectiveness of cipsc-islet transplantation, but also provide hope for the treatment of more patients in the future. future studies will further evaluate the effects of cipsc-islet transplantation in larger sample sizes and longer periods of time, in order to bring good news to more patients with type 1 diabetes.

with the continuous advancement of science and technology, stem cells and regenerative medicine are gradually changing our medical practice. the success of this study not only brings new hope to patients with type 1 diabetes, but also provides new ideas for the treatment of other chronic diseases. we look forward to more similar innovative achievements in the future to jointly promote the development of global public health.

refer to

Wang, C., et al. (2024). "Transplantation of chemically induced pluripotent stem-cell-derived islet under the abdominal anterior rectus sheath in a type 1 diabetes patient." Cell. doi:10.1016/j.cell.2024.09.004