Stem cell therapy to treat Type I diabetes

Marcos Malumbres

Centro Nacional de Investigaciones Oncológicas (CNIO), Spain

Type 1 diabetes has no cure and affects more that 10.2 million people all over the world, causes premature death and costs more that 600 billion USD. Today, the best option consists merely of controlling disease progression through insulin injections and maintenance of a healthy diet and lifestyle. Stem cell-based therapies have emerged as an encouraging alternative, based on the use of reprogrammed cells derived from the diabetic patient. This field is rapidly evolving, with several ongoing clinical trials exploring its effectiveness.

However, because of the intrinsic difficulties in obtaining gold-standard pluripotent cells by reprogramming, this strategy yields poor-quality cells with poor differentiation potential and lack of functionality. Thus, there is a need to find an improved approach to easily generate high-quality pluripotent cells from patients, capable of differentiating to pancreatic beta cells, mature and functional, long-term producers of insulin.

If solved, it would have an enormous socio-economic impact: not only for patients, their families and healthcare professionals, but also for the global healthcare system, health insurance and governments worldwide, which are struggling to meet the costs of diabetes care.

Solving this need can be achieved by using the project's novel methodology: a brief exposure to a single microRNA makes any stem cell tested so far (independently of its origin) more prone to efficiently differentiate into the desired specialized tissue: in this case, pancreatic beta cells, mature and producers of insulin.