Diabetic patient receives stem cell treatment for the first time in the world

A 25-year-old woman with type 1 diabetes began producing her own insulin less than three months after receiving a stem cell transplant. She became the first person with the disease to receive treatment using cells taken from her own body.

The patient, who asked to remain anonymous and lives in Tianjin Province in northern China, told Nature magazine that she can now eat sugar after undergoing the procedure more than a year ago.

James Shapiro, a transplant surgeon and researcher at the University of Alberta in Edmonton, Canada, describes the results as “amazing.” “These cells completely reversed the diabetes in the patient, who previously needed large amounts of insulin,” he says.

These studies are among a handful of pioneering trials using stem cells to treat diabetes, which affects nearly half a billion people worldwide.

Most people with type 2 diabetes either don't produce enough insulin or their body doesn't use the hormone well.

In type 1 diabetes, the body's immune system attacks what are called "islet cells," which are cells found in the pancreas and play a vital role in regulating blood sugar levels. They are also responsible for producing several hormones, the most important of which is insulin.

This type of disease can be treated, but there are not enough donors to meet the increasing demand, and recipients must use immunosuppressive drugs to prevent the body from rejecting the donor tissue.

So new research has turned to using stem cells to grow any tissue in the body that can be transplanted and potentially become an unlimited source of pancreatic tissue, for example. By using tissue made from a person’s own cells, researchers also hope to avoid the need for immunosuppressive drugs.

In the first experiment of its kind, Deng Hongkui, a cell scientist at Peking University, and his colleagues extracted cells from three people with type 1 diabetes and returned them to a “pluripotent state,” in which they can form any type of cell in the body.

This reprogramming technique was first developed by Shinya Yamanaka at Kyoto University in Japan about two decades ago. But Deng and his colleagues modified the technique: instead of introducing proteins that stimulate gene expression, as Yamanaka did, they exposed the cells to small molecules, which allowed for more control over the process.

The researchers then used chemically induced pluripotent stem (iPS) cells to generate 3D clumps and tested the safety and efficacy of the cells in mice and “non-human primates.”

In June 2023, in a procedure that took less than half an hour, the researchers injected the equivalent of about 1.5 million “islet cells” into the woman’s abdominal muscles — a new site for transplantation. Most transplants were performed in the liver, but by placing them in the abdomen, the researchers were able to monitor the cells using MRI, and potentially remove them if necessary.

After two and a half months, the woman was producing enough insulin to live without needing additional doses, and she maintained this production level for more than a year.

The results are exciting but need to be replicated in more people, says Jay Schizer, an endocrinologist at the University of Miami, Florida, who studies type 1 diabetes.

Schaiser also wants to see if the woman's cells will continue to produce insulin for up to five years before the trial can be considered a "cure" that can be approved.

Transplanting organs using human cells has many advantages, but the procedure is difficult to scale up and commercialize, the researchers say. Several groups have begun trials of islet cells created using stem cells from donors.

Preliminary results from one trial, led by Vertex Pharmaceuticals of Boston, Massachusetts, were reported in June.

Twenty participants with type 1 diabetes received “islets” derived from donated embryonic stem cells, which were injected into their livers. All were treated with immunosuppressive drugs. Three months after the transplant, all participants began producing insulin when glucose was present in the bloodstream, and some became insulin independent.

Last year, Vertex launched another trial in which islet cells derived from donated stem cells were placed in a device designed to protect them from immune system attacks. It was transplanted into a person with type 1 diabetes who was not receiving immunosuppressive drugs.

Other researchers, including Daisuke Yabe, a diabetes researcher at Kyoto University in Japan, are preparing to begin an experiment using islet cells produced from donated iPS cells. He plans to grow sheets of islets and surgically implant them into the abdominal tissue of three people with type 1 diabetes, who will receive immunosuppressive drugs.

The first transplants for participants are expected to take place early next year.