Breakthrough in Stem Cell Therapy Reverses Type 1 Diabetes in First Human Trial

In a groundbreaking medical achievement, a 25-year-old woman from Tianjin, China, has effectively reversed her type 1 diabetes following a transplant of insulin-producing cells derived from reprogrammed stem cells. The woman, who had relied on insulin injections to manage her condition, has now been insulin-free for over a year. This pioneering procedure, published in Cell, marks the first successful trial of its kind in which stem cells from the patient’s own body were used to treat diabetes.
The woman, who chose to remain anonymous, expressed her newfound freedom from the dietary restrictions that had governed her life for years: “I can eat sugar now,” she shared in an interview with Nature, adding that she now enjoys a wide variety of foods, particularly hotpot.

A Medical Milestone
James Shapiro, a renowned transplant surgeon and researcher at the University of Alberta, Canada, described the procedure’s outcome as “stunning.” He noted that the surgery has “completely reversed diabetes in the patient, who was requiring substantial amounts of insulin beforehand.”
This success comes amidst other pioneering efforts to harness stem cells for diabetes treatment. Earlier this year, a separate group in Shanghai successfully transplanted insulin-producing islets into a 59-year-old man with type 2 diabetes, also using reprogrammed stem cells. The patient has since stopped taking insulin, further highlighting the potential of stem cell therapies in treating different types of diabetes.

Stem Cells: A Solution to the Donor Shortage
The use of stem cells offers a revolutionary approach to treating diabetes, especially as the demand for islet transplants exceeds the availability of donors. Unlike donor-dependent transplants, stem cells can be cultured indefinitely in a lab, providing an unlimited source of pancreatic tissue. For individuals with type 1 diabetes, whose immune systems attack their insulin-producing cells, this could be life-changing.
Deng Hongkui, a cell biologist from Peking University, led the research that made this breakthrough possible. His team extracted cells from the patient and reprogrammed them into a pluripotent state, meaning they could develop into any cell type in the body. This technique was adapted from a method developed by Japanese scientist Shinya Yamanaka nearly two decades ago. Deng’s team further refined the process by using small molecules to control the reprogramming, offering greater precision.

A New Approach to Islet Transplants
In June 2023, after safety trials in animals, Deng’s team performed the procedure on the woman, injecting around 1.5 million islets into her abdominal muscles. Typically, islet transplants are done in the liver, but placing the cells in the abdomen allowed the researchers to monitor them more closely and potentially remove them if needed.
Remarkably, just two and a half months after the transplant, the woman was producing enough insulin to maintain normal blood glucose levels without supplementation. Over the past year, she has maintained stable blood glucose levels, avoiding the dangerous spikes and drops common in diabetes management.
Experts, including Daisuke Yabe from Kyoto University, have hailed the results as “remarkable.” If replicable, this procedure could have profound implications for millions of people living with type 1 diabetes.

Next Steps in the Research
While the results are promising, scientists emphasize the need for further trials to validate the treatment. Jay Skyler, an endocrinologist at the University of Miami, noted that it is crucial to see if the woman continues to produce insulin for several more years before declaring her “cured.”
Deng is optimistic about the future. The other two participants in this trial are also showing positive results and are expected to reach the one-year milestone soon. His team plans to expand the trial to include 10 to 20 more participants.

Addressing the Autoimmune Challenge
One of the primary challenges in treating type 1 diabetes is the autoimmune nature of the disease, which causes the body to attack its insulin-producing cells. In this case, the woman was already on immunosuppressants due to a previous liver transplant, so researchers couldn’t determine whether her own stem cells would lower the risk of rejection. Moving forward, the team hopes to develop cells that can evade autoimmune attacks without the need for immunosuppressants.

The Future of Stem Cell Transplants
While personalized cell transplants offer immense potential, scaling up and commercializing the process remains a challenge. Other research groups are exploring the use of donor stem cells as a more feasible alternative. For example, Vertex Pharmaceuticals in Boston is conducting trials using donor-derived islet cells. Preliminary results have been promising, with some participants achieving insulin independence.
Additional trials are also underway, including one led by Yabe, who is developing sheets of islets for transplant into the abdominal tissue of type 1 diabetes patients. The goal is to find a sustainable, scalable solution that could make stem cell therapies more widely available.

A New Era in Diabetes Treatment
This breakthrough marks a significant step toward a future where diabetes may no longer be a lifelong burden for millions. As Deng’s research progresses, and as other clinical trials advance, there is hope that stem cell therapies could soon offer a cure for this chronic disease.
For now, the woman from Tianjin is living proof that such a future might be closer than ever. “I enjoy eating everything,” she says, reflecting on her newfound freedom.