Can stem-cell therapy successfully treat type 1 diabetes?

• A woman has undergone a stem-cell therapy made from her own cells, to treat her type 1 diabetes.
• Researchers in China discovered the woman did not need to use insulin 75 days after the procedure, and that the stem-cell derived islet cells she was injected with had been engrafted inside her abdomen.
• More people have been enrolled in this trial and other stem-cell therapy trials at several sites around the world.

A woman with type 1 diabetes started producing her own insulin less than 3 months after receiving an injection of a stem-cell derived treatment. She remained free from insulin injections 1 year after treatment.

The case is the first of its kind, and two more people have been enrolled in the clinical trial in China since, researchers involved in the study told Medical News Today.

Hongkui Deng, PhD, lead author of the new study — which appears in Cell — and cell biologist at Peking University, Beijing, China, who has previously done groundbreaking work developing induced pluripotent stem cells told MNT that:

“The trial is ongoing and includes three patients in total. After [an] interim analysis of the [data of the] first patient and the submission of this work, the second and third patients were enrolled. Follow-up with these patients is ongoing, as they were sequentially enrolled in accordance with regulatory safety requirements. [A] long-term follow-up of at least 2 years will be conducted.”

Other stem-cell based therapies for type 1 and type 2 diabetes are also currently in development and in trials.

For this case study, researchers based in Tianjin First Central Hospital, Nankai University, Tianjin, China took fat cells from a 25 year-old woman with type 1 diabetes, and chemically induced them to behave as pluripotent stem cells, a type of cell that can develop into other types of cell.

They then used these to create islet cells, which typically exist in the pancreas and create insulin, a hormone that regulates levels of glucose (sugar) in the bloodstream.

Type 1 diabetes is an autoimmune condition that results in the destruction of these islet cells by the immune system. This means that the body can not create as much insulin as needed which results in chronic, high blood glucose which can lead to complications including vascular problems that affect eye sight, can cause nerve and kidney damage.

The patient in this case study had previously had two liver transplants and a failed pancreas transplant due to complications that had arisen due to her diabetes.

The induced islet cells made from the patient’s own cells were then injected between the skin and abdominal muscles. Researchers discovered that these successfully engrafted in the patient, including growing their own vasculature.

Researchers found that the woman no longer needed insulin injections from around 2 and a half months after they had carried out the procedure, and at the 1-year mark she still had no need for insulin injections.

Before the procedure she produced enough insulin to reach her target glycemic range 43.18% of the time, and 4 months later this increased to 96.2% of the time. She was also shown to have lower glycated hemoglobin, which indicated long-term systemic glucose levels at a non-diabetic level.

Though this is the first case study available of a person who has continued to produce insulin 1 year after receiving stem cell-based therapies, there are other trials that are ongoing to develop a stem cell treatment for people with type 1 and 2 diabetes.

In June 2024, the pharmaceutical company Vertex announced results from its phase 1/2 clinical trial on the use of stem-cell derived type 1 diabetes treatments at the American Diabetes Association 84th Scientific Sessions.

The trial results, based on the data of 12 people with type 1 diabetes, looked at the introduction of stem-cell derived insulin-producing islet cells. The trial was expanded to a total of 37 people at the time the company reported the result in June.

Previously, safety data from a trial in Canada, which aimed to determine the safety of engrafting insulin-producing stem-cell derived pancreatic endoderm cells into 17 participants, appeared in Cell Reports Medicine in 2021.

The first author of the Cell Reports Medicine study, James Shapiro, who is a professor of surgery at the University of Alberta, shared with MNT his thoughts on the latest breakthrough now reported in Cell.

“I’m not at all surprised [by the results] — we have been working on this also for the past 5 years, making insulin-producing islets and reversing diabetes in mice reliably using human stem cell islets. The beauty of this approach is that they are the patients’ own cells — so organ and tissue rejection is not a concern, and no or far less anti rejection medications are needed. But I am indeed exceedingly impressed with the stunning results the Tianjin Team achieved in their first patient. This is truly incredible.”

Shapiro told us he was interested in continuing to work with other teams to overcome some of the challenges that were still faced in making stem-cell treatments for people with type 1 diabetes.

“There are still lots of things to sort out,“ he pointed out. “Firstly, how can this process be expanded and accelerated to treat many more patients?“

“Secondly, can this really be done without any medications for patients with autoimmune (type 1) diabetes? Third, more time and safety testing will be needed to make sure there are no unwanted — or ‘off target’ — cells generated in the process,” noted Shapiro.

Deng concurred, telling MNT that the “challenges that need to be addressed in the field of stem cell-derived islet transplantation for diabetes therapy in order to extend its clinical benefits to many more patients include [the] development of appropriate immunomodulatory strategies, and the clinical manufacture of stem cell-derived islets in an efficient and cost-effective manner.”