A world-first clinical trial led by Uppsala University Hospital has shown that gene-edited donor islet cells can survive and function inside a person with long-standing type 1 diabetes for at least 12 weeks without the need for immune-suppressing drugs.
Although intensive insulin therapy can reduce complications and extend life expectancy, people with early-onset type 1 diabetes still face a reduced quality of life, a higher risk of cardiovascular disease, and shorter lifespan. For transplant recipients, the toxicity of lifelong immunosuppression poses further risks.
- Inflammation triggered by stress linked to metabolic syndrome
- New form of type 1 diabetes identified in black patients
- Barbie doll with type 1 diabetes launched
The study, Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression, published in the New England Journal of Medicine, tested whether ‘hypoimmune’ engineered islet cells could avoid rejection by the immune system.
A single participant – a 42-year-old man who had lived with type 1 diabetes for 37 years – received islet cells from a blood group–matched deceased donor.
The cells were gene-edited using CRISPR-Cas12b to remove the B2M and CIITA genes and modified to overexpress CD47 – a protein that helps them evade immune attack.
They were then re-clustered and implanted into the muscle of his forearm under general anaesthetic.
No anti-inflammatories, glucocorticoids or systemic immunosuppressants were given.
Over the 84-day follow-up, the immune system attacked any residual unmodified cells but left the engineered cells untouched.
These hypoimmune cells produced insulin, with C-peptide levels remaining near or above 10 pmol/L and increasing during meal testing.
- Common blood glucose test fails to detect nearly three quarters of gestational diabetes cases
- Poor sleep linked to elevated HbA1c in people with type 2 diabetes
- Popular 5:2 fasting diet beneficial for people with type 2 diabetes and obesity
HbA1c fell by about 42%, and scans confirmed the grafts were healthy and free from inflammation.
Only mild, non–drug-related side effects were reported. As the implant represented just 7% of a full beta-cell replacement, the man still required insulin, but the results confirm both survival and function.
Researchers believe this approach could one day make curative beta-cell transplants possible without the dangers of lifelong immune suppression, transforming care for millions with type 1 diabetes.
