[PDF][PDF] Implanted pluripotent stem-cell-derived pancreatic endoderm cells secrete glucose-responsive C-peptide in patients with type 1 diabetes

A Ramzy, DM Thompson, KA Ward-Hartstonge… - Cell stem cell, 2021 - cell.com
A Ramzy, DM Thompson, KA Ward-Hartstonge, S Ivison, L Cook, RV Garcia, J Loyal
Cell stem cell, 2021cell.com
An open-label, first-in-human phase 1/2 study is being conducted to evaluate the safety and
efficacy of pancreatic endoderm cells (PECs) implanted in non-immunoprotective
macroencapsulation devices for the treatment of type 1 diabetes. We report an analysis on 1
year of data from the first cohort of 15 patients from a single trial site that received
subcutaneous implantation of cell products combined with an immunosuppressive regimen.
Implants were well tolerated with no teratoma formation or severe graft-related adverse …
Summary
An open-label, first-in-human phase 1/2 study is being conducted to evaluate the safety and efficacy of pancreatic endoderm cells (PECs) implanted in non-immunoprotective macroencapsulation devices for the treatment of type 1 diabetes. We report an analysis on 1 year of data from the first cohort of 15 patients from a single trial site that received subcutaneous implantation of cell products combined with an immunosuppressive regimen. Implants were well tolerated with no teratoma formation or severe graft-related adverse events. After implantation, patients had increased fasting C-peptide levels and increased glucose-responsive C-peptide levels and developed mixed meal-stimulated C-peptide secretion. There were immunosuppression-related transient increases in circulating regulatory T cells, PD1high T cells, and IL17A+CD4+ T cells. Explanted grafts contained cells with a mature β cell phenotype that were immunoreactive for insulin, islet amyloid polypeptide, and MAFA. These data, and associated findings (Shapiro et al., 2021), are the first reported evidence of meal-regulated insulin secretion by differentiated stem cells in patients.
cell.com