In worlds first, gene-edited cells help diabetic man produce insulin

A 42-year-old man with type 1 diabetes has become the first person to receive a transplant of genetically modified insulin-producing cells designed to evade the body's immune system – a development scientists say could pave the way toward a cure for the disease.

The procedure, carried out by researchers at Uppsala University in Sweden, allowed the patient to begin producing small amounts of his own insulin for the first time in years, without the need for immunosuppressant drugs that normally accompany cell transplants. The results were published this month in the New England Journal of Medicine, according to Scientific American.

"This is the most exciting moment of my scientific career," said Per-Ola Carlsson, a cell biologist at Uppsala who co-led the study. "It opens the future possibility of treating not only diabetes but other autoimmune diseases."

Scientists injected nearly 80 million genetically engineered cells into the patient's forearm muscle. Twelve weeks later, the cells were still alive and releasing insulin in response to meals. Although the man still required insulin injections, researchers said the cells showed no signs of rejection – a major step forward after decades of failed attempts to replace destroyed beta cells in type 1 diabetics.

Type 1 diabetes affects about 2 million people in the United States. The autoimmune disease destroys insulin-producing beta cells in the pancreas, forcing patients to rely on lifelong insulin injections and constant blood sugar monitoring. Without control, the condition can lead to heart disease, kidney failure, nerve damage and blindness.

For years, scientists have explored cell transplants as a potential cure. But past efforts using donor islets or stem-cell-derived cells triggered aggressive immune responses, requiring recipients to take drugs that increase risks of infection and cancer.

To overcome this hurdle, Carlsson's team used CRISPR gene-editing to inactivate two genes that normally flag donor cells to the immune system. They also boosted expression of another gene that suppresses attack from immune cells.

Laura Alonso, head of endocrinology, diabetes and metabolism at Weill Cornell Medicine, called the results "amazing," though she urged caution. "It's one person, it's early, and the insulin produced was only about 7% of what's needed," she said. "But cell-based therapy is where we need to go."

evan Herold, an endocrinologist at Yale School of Medicine, also noted the limits. "The participant still required a substantial amount of insulin after the transplant, so the clinical significance is somewhat unproven," he said.

Carlsson said his team will continue monitoring the patient and may increase the dose of modified cells if no side effects occur. The group is also working on producing insulin-producing cells from stem cells, which could scale the therapy, says Scientific American.

"The combination of those two advances would create the possibility of a cure for type 1 diabetes," Carlsson said.

Herold was more cautious but acknowledged the breakthrough. "It's still too early to call this a cure," he said. "But these options are now here to change the disease in ways that have never been possible before. There's tremendous hope."