Autologous Umbilical Cord Blood Transfusion in Very Young Children With Type 1 Diabetes

Abstract

OBJECTIVEInterest continues to grow regarding the therapeutic potential for umbilical cord blood therapies to modulate autoimmune disease. We conducted an open-label phase I study using autologous umbilical cord blood infusion to ameliorate type 1 diabetes.RESEARCH DESIGN AND METHODSFifteen patients diagnosed with type 1 diabetes and for whom autologous umbilical cord blood was stored underwent a single intravenous infusion of autologous cells and completed 1 year of postinfusion follow-up. Intensive insulin regimens were used to optimize glycemic control. Metabolic and immunologic assessments were performed before infusion and at established time periods thereafter.RESULTSMedian (interquartile range [IQR]) age at infusion was 5.25 (3.1–7.3) years, with a median postdiagnosis time to infusion of 17.7 (10.9–26.5) weeks. No infusion-related adverse events were observed. Metabolic indexes 1 year postinfusion were peak C-peptide median 0.50 ng/ml (IQR 0.26–1.30), P = 0.002; A1C 7.0% (IQR 6.5–7.7), P = 0.97; and insulin dose 0.67 units · kg−1 · day−1 (IQR 0.55–0.77), P = 0.009. One year postinfusion, no changes were observed in autoantibody titers, regulatory T-cell numbers, CD4-to-CD8 ratio, or other T-cell phenotypes.CONCLUSIONSAutologous umbilical cord blood transfusion in children with type 1 diabetes is safe but has yet to demonstrate efficacy in preserving C-peptide. Larger randomized studies as well as 2-year postinfusion follow-up of this cohort are needed to determine whether autologous cord blood–based approaches can be used to slow the decline of endogenous insulin production in children with type 1 diabetes.

Regeneration of insulin production by autologous bone marrow blood autotransplantation in patients with type 1 diabetes

“…Our data, obviously, cannot rule out the possibility that bone marrow transplantation could be effective in preserving or even restoring beta cell function in recently diagnosed type 1 diabetes patients. However, a recent report, in which autologous umbilical cord blood infusion failed to preserve C-peptide levels in young newly diagnosed children 1 year after infusion, does not support this hypothesis [10].…”

Type 1 Diabetes: Etiology, Immunology, and Therapeutic Strategies

“…Stem cells can regenerate the beta-cell mass in vivo, as shown for bone marrow-derived stem cell transfers in immunodeficient mice with chemically induced pancreatic damage (498). But in a phase I/II trial, stem cells from umbilical cord blood assisted TYPE 1 DIABETES: FROM CAUSE TO CURE the preservation of C-peptide levels poorly (175). Autologous nonmyeloablative hematopoietic stem cell transplantation (HSCT) yielded better results in newly diagnosed T1D (100, 459).…”

Tertiärprävention des Typ-1-Diabetes

“…Alternativ könnten sie im Sinne einer Wiederherstellung der Treg-Aktivität eine Immunmodulation bewirken. Studien aus den USA zeigten, dass diese Therapie zwar nicht kurativ wirkt, offenbar aber auch nicht schadet [35]. Inzwischen wird dieser Therapieansatz in den USA mit zusätzlichen Maßnah-men (Gabe von Vitamin D, ω3-Fettsäuren) kombiniert; auch in Deutschland wird die Behandlung mit Nabelschnurvenenblut bei Kindern mit neu diagnostiziertem Typ-1-Diabetes ab dem ersten Lebensjahr angeboten (.…”