Advancing toward a curative frontier: an updated narrative review on stem-cell therapy in pediatric type 1 diabetes

Type 1 diabetes (T1D) is a chronic autoimmune disease primarily diagnosed in childhood, characterized by pancreatic β-cell destruction, severe insulin deficiency, and hyperglycemia. Current treatments, including insulin therapy and glucose-lowering medications, manage the condition but fall short of offering a cure. In this review we explore the potential of stem-cell therapy as a transformative and curative approach for T1D, focusing on its promise in regenerating β-cells and addressing challenges specific to the pediatric population.

A comprehensive review of the literature was conducted to evaluate stem-cell types: embryonic, perinatal, adult, induced pluripotent and cancer stem cells, and their role in T1D treatment. Particular emphasis was placed on methods for β-cell differentiation, advancements in autologous and allogeneic stem-cell transplantation and emerging strategies to overcome safety, efficacy, and economic barriers. Challenges such as immune rejection, tumorigenicity, and cost-effectiveness were analyzed, alongside novel solutions like immune-shielding and clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein-9 (Cas9) technology.

Stem-cell therapy presents a promising avenue for curing T1D, offering potential for β-cell regeneration and reduced dependence on exogenous insulin. However, challenges such as delayed β-cell functionality, immune responses, tumor risks, and high costs hinder widespread application.

Advancements in personalized medicine, immune-shielding strategies, and cost reduction may pave the way for clinical success, especially in pediatric populations. Further research addressing these barriers is essential to establish stem-cell therapy as a viable and equitable treatment option.