T1D Patient Achieves Full Insulin Independence After Stem Cell Transplant: case study
A stem-cell treatment restored insulin production and glucose control in two patients with type 1 diabetes (T1D), a researcher reported during the American Diabetes Association (ADA) annual meeting.
Cadaveric islet transplantation can achieve glycemic control in T1D, but cadaveric islet quantity and quality are limiting. We report the first patient-administered VX-880, an investigational allogeneic stem cell-derived, fully differentiated, pancreatic islet cell replacement therapy.
Case:
A 64-year-old male with a 40-year history of T1D complicated by impaired awareness of hypoglycemia with 5 severe hypoglycemic events (SHEs) the year before VX-880 was receiving 34U insulin/day at baseline (HbA1c 8.6%; undetectable fasting and stimulated C-peptide). After a single VX-880 infusion at half the target dose, fasting C-peptide was detected by Day 29 and increased rapidly; HbA1c and daily insulin decreased in parallel. At Day 90, robust increases in fasting and stimulated C-peptide, improved glycemic control, and a substantial reduction in exogenous insulin administration were observed and continued to improve through the last time point assessed
VX-880 was generally safe and well-tolerated; most AEs were mild or moderate and consistent with immunosuppression. The most common AEs were SHEs (not serious or related to VX-880), which occurred in the perioperative period. There was 1 serious AE of rash (mild, unrelated to VX-880), which was resolved.
Thus, these unprecedented results are the first evidence that stem cell-derived islets can restore insulin production and glucose control in T1D. The study continues to enrol and dose patients.
Reference:
259-OR: Stem Cell-Derived, Fully Differentiated Islet Cells for Type 1 Diabetes by JAMES F. MARKMANN et al. published in the American Diabetes Association.
https://doi.org/10.2337/db22-259-OR
Keywords:
American Diabetes Association, 259-OR, Stem Cell–Derived, Fully, Differentiated, Islet, Cells, Type 1 Diabetes, JAMES F. MARKMANN, ALI NAJI; MICHAEL R. RICKELS; MARIA ALBA; GAUTHAM MARIGOWDA; LEORAH ROSS; CHENKUN WANG; FELICIA PAGLIUCA; BASTIANO SANNA; LESLIE S. KEAN; ANNE L. PETERS; PIOTR WITKOWSKI; CAMILLO RICORDI
