Stem Cells Transformed in Type 1 Diabetes

The production of insulin-secreting cells from the stem cells of patients with type 1 diabetes may signal a potential new approach to treating diabetes, according to a new study.

The finding suggests a personalized treatment approach to diabetes in which the patients' own stem cells could be used to manufacture new cells that make insulin.

"In theory, if we could replace the damaged cells in these individuals with new pancreatic beta cells -- whose primary function is to store and release insulin to control blood glucose -- patients with type 1 diabetes would not need insulin shots anymore," said lead author Jeffrey R. Millman, PhD, assistant professor of medicine and of biomedical engineering at Washington University School of Medicine. "The cells we have manufactured sense the presence of glucose and secrete insulin in response. And beta cells do a much better job controlling blood sugar than diabetic patients can."

Millman had made beta cells from stem cells derived from people who did not have diabetes. In the new experiment, the beta cells came from tissue taken from the skin of patients with diabetes.

"There had been questions about whether we could make these cells from people with type 1 diabetes," said Millman. "Some scientists thought that because the tissue would be coming from diabetes patients, there might be defects to prevent us from helping the stem cells differentiate into beta cells. It turns out that is not the case."

More research is needed to ensure that the beta cells made from patient-derived stem cells do not lead to the development of tumors, but there has been no evidence of tumors in the mouse studies, even up to one year after the cells were implanted.

The stem cell-derived beta cells could be ready for human research in three to five years, Millman said. At that time, he expects the cells would be implanted under the skin of diabetes patients in a minimally invasive surgical procedure that would allow the beta cells access to a patient's blood supply.

"What we are envisioning is an outpatient procedure in which some sort of device filled with the cells would be placed just beneath the skin," said Millman.

More than two decades ago, researchers began transplanting beta cells into patients with type 1 diabetes. Patients in several clinical trials have received beta cell transplants from pancreas tissue provided by organ donors, with some success. As with all types of organ donation, the need for islet beta cells greatly exceeds their availability.

The new technique also could be used in other ways. These experiments have proven the possibility of making beta cells from the tissue of patients with type 1 diabetes. The technique would also likely work in patients with other forms of the disease -- including type 2 diabetes mellitus, neonatal diabetes, and Wolfram syndrome. It would be possible to test the effects of diabetes drugs on the beta cells of patients with various forms of the disease, he said.

Reference: Millman JR, et al. Generation of stem cell-derived β-cells from patients with type 1 diabetes. Nat Commun. 2016 May 10;7:11463.