Immune system enhancement through unseen cell technology could potentially provide a remedy for type 1 diabetes.
A groundbreaking treatment for type 1 diabetes has emerged, as a 42-year-old man with a long-standing history of the condition received a genetically modified cell transplant at the University Hospital of Uppsala in Sweden. The treatment, which has shown effectiveness in avoiding the need for lifelong immunosuppressive drugs, was published in The New England Journal of Medicine [1][2][3].
Gene Editing at the Forefront
The donor islet cells underwent targeted editing using CRISPR-Cas12b molecular scissors to create "hypoimmune" cells [2][3][4]. These cells are designed to evade the immune system, allowing the recipient to produce insulin without the need for immunosuppressive drugs.
Trial Results
Over a period of 12 weeks, the gene-edited cells survived and produced insulin in the patient's body, as confirmed by stable and increasing C-peptide levels (a marker of insulin secretion) and improved glycemic control (HbA1c fell by about 42%) [1][2]. The grafts showed no signs of inflammation or immune attack on advanced imaging tests, and there were only mild adverse events unrelated to the treatment [2].
Clinical Significance
This trial represents a "proof-of-survival" and "proof-of-function" validation of gene-edited islet cell transplantation without the need for immune suppression [1][2][3]. This could potentially revolutionise type 1 diabetes treatment by eliminating the need for systemic immunosuppressants that currently limit transplantation use.
Trial Specifics
The trial, which started in March 2024, enrolled two participants and is an early clinical proof-of-concept study using CRISPR-Cas12b editing technology [4].
A Safer Alternative
This treatment addresses the major challenge of transplant rejection in type 1 diabetes cell therapies by genetically engineering cells to become "invisible" to the recipient’s immune system [1][2][3]. The cells were modified to overexpress a protein that sends "don't eat me" signals to immune cells (CD47) [2].
Although the transplanted cells were initially attacked by the immune system for 84 days, they managed to maintain insulin production and improve glucose control.
Previous Progress
A study published in 'Cell' reported a case of a 25-year-old woman who produced insulin on her own and stopped injections for over a year after receiving a transplant of pancreatic islets generated from her own body's reprogrammed stem cells [5]. However, this approach requires immunosuppression.
References:
[1] The New England Journal of Medicine [2] Science Daily [3] Medical Xpress [4] Uppsala University [5] Cell (2023)
Film about the medical field could explore the story of the groundbreaking treatment for type 1 diabetes, which involves gene-edited cell transplants to create "hypoimmune" cells. This technique, potentially revolutionizing diabetes treatment by eliminating the need for systemic immunosuppressants, was tested on a 42-year-old man at the University Hospital of Uppsala in Sweden.
Science and health-and-wellness publications such as The New England Journal of Medicine, Science Daily, and Medical Xpress have reported on the promising results of this gene-editing approach, demonstrating its effectiveness in avoiding long-term immunosuppressive drug use.
In the future, medical-conditions like chronic diseases, including type 2 diabetes, may also benefit from this advancement, offering safer alternatives for cell therapies by genetically engineering cells to evade the immune system. Research in this area continues, with clinical trials focusing on type 1 diabetes and using CRISPR-Cas12b editing technology.
