medwireNews: Early results suggest that transplantation of fully differentiated stem cell-derived islet cells is a promising approach for achieving glycemic control in people with unstable type 1 diabetes.
Speaking at the 82nd ADA Scientific Sessions in New Orleans, Louisiana, James Markmann (Massachusetts General Hospital, Boston, USA) presented findings from a trial of the technology in one man and one woman with type 1 diabetes lasting 42 and 11 years, respectively.
Both participants had improved outcomes, he reported, with one able to discontinue insulin from about 200 days after receiving the cells.
The treatment, called VX-880, is an islet cell therapy derived from stem cells, rather than from deceased donors. Similar to donor islet transplantation, the cells are delivered to the liver via the hepatic portal vein.
Addressing reporters at the conference, Markmann noted that the stem-cell technology was developed with the aim of producing cells that are of consistent quality, unlike those from donors.
And donor cells are in a short supply, he said, noting that “one of the most important aspects of this work is that there can be an unlimited supply of cells for transplantation.”
The cells are allogeneic, however, so recipients require immunosuppression; the current trial used the regimen developed for the Clinical Islet Transplantation Consortium phase 3 trials.
The man and woman in the current study had undetectable C-peptide levels, impaired hypoglycemia awareness, and had respectively experienced five and three severe hypoglycemia episodes in the year before enrollment. These participants both received half of the target dose of VX-880.
During 270 days of follow-up, the male participant’s time in range (TIR) rose from 40.1% at baseline to 81.4% during days 121–150 and to 99.9% at days 241–270.
In parallel, his glycated hemoglobin (HbA1c) fell from 8.6% to 5.2% (70 to 33 mmol/mol) and his daily insulin dose from 34.0 to 0.0 units.
And during 150 days of follow-up, the female participant’s TIR increased from 35.9% at baseline to 51.9% during days 121–150. Her HbA1c fell from 7.5% to 7.1% (58 to 54 mmol/mol), although it was as low as 6.5% (48 mmol/mol) at day 120, and her daily insulin dose fell from 25.9 to 18.2 units.
The male participant had a number of severe hypoglycemia episodes during the perioperative period, which were not considered to be related to the procedure, and one at day 35, after which he had no further episodes. The female participant had no severe episodes after the infusion.
Just one adverse event, of a “mild” rise in liver enzymes post-infusion, was considered related to the treatment.
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