T1D Patient Achieves Full Insulin Independence After Stem Cell Transplant: case study

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