Type 1 diabetes (T1D) is an autoimmune disease characterized by progressive destruction of insulin-producing β cells in the pancreas, leading to pathological increase of blood sugar (glycemia). It affects 35 million people worldwide and is associated with a risk of multi-organ failure (kidney, heart) despite continuous progress in its treatment (i.e. insulin therapy). Finding a cure for T1D means finding strategies to restore spontaneous insulin secretion capacity. In this way, two possible areas of investigation exist: a) the preservation of the function of β cells after the onset of the disease; b) the restoration of the mass of β cells by transplantation of new β cells or β-like cells.
Current strategies for β-cell mass preservation – including immunotherapy, and vaccination – have globally failed to show significant effects on disease control in terms of HbA1C levels. Replacement of pancreatic β cells is an attractive therapy for T1D1 and proof-of-concept has been provided with islet transplantation, which is dependent on the availability of organ donors. Therefore, there is a need to find efficient ways to derive β cells from other cell sources whose use is unarguably compatible with clinical procedures.
In this context, our work focuses on: a) the study of β-cell mass evolution after T1D and of potential mechanisms of β-cell preservation; b) the transplantation of new β-like cells in order to replenish the insulin-secreting capacity that is lacking in T1D.
We characterized the evolution of T1D in a pediatric cohort of patients2 and we are now studying the principal determinants of the partial remission occurring after diagnosis (in 60% of patients), with the aim of identifying patients that could benefit from treatments to preserve β-cell function and insulin secretion.
Our group has discovered a new pancreatic cell progenitor with the potential to produce new β cells in vitro3 and to decrease glucose levels of diabetic animals after transplantation4. In our current studies, we are evaluating novel technologies to preserve β-cell mass after the onset of T1D and to foster insulin secretion from our β cell-like pancreas progenitors. Our techniques are designed with the aim of being compatible with clinical-grade procedures and potential translational approaches for patients with diabetes.