Simplifying stem cell transplantation for newly diagnosed type 1 diabetes

This study investigated a stem cell transplant method using a less toxic and cheaper method in individuals with recently diagnosed type 1 diabetes. The study concluded that this therapy is a feasible and safe potential treatment for type 1 diabetes.

In patients with type 1 diabetes (T1D) the pancreas does not produce insulin,  the hormone needed to break down the glucose (sugar) taken in from food. Therefore, current treatment methods involve replacing the insulin that the body can no longer produce with insulin injections.

Stem cells have the potential to grow and develop into any cell type or tissue in the body, including cells of the pancreas. These cells could replace those that are no longer able to produce insulin. Patients who receive stem cell transplants receive drugs to block the immune system. This prevents the immune system from attacking and destroying the transplanted stem cells. 

Stem cell transplantation has required long hospitalizations and multiple medications to block the immune system, which can be very expensive and have serious side effects (such as infection). Simplified methods of stem cell transplantation are needed.

The current study examined a simplified stem cell transplantation method. 16 patients with recently diagnosed type 1 diabetes underwent stem cell transplantation. Patients were sent home following transplantation, under the care of a family member instructed in proper care. Patients were seen by doctors each day until the stem cells started growing. All patients were treated with fludarabine (Fludara), a less toxic and cheaper method of blocking the immune system than is generally used. Patients were followed for an average of 3 years after the transplant. 

By 6 months following transplant, 44% of patients achieved insulin independence. 37% of patients were able to reduce their total daily insulin requirement by an average of 46%. 19% of the patients did not respond. HbA1c levels showed an average decrease of 2.3% at the 6 months in those who were insulin independent.  

No side effects requiring hospitalization occured in any patient.

The study concluded that this therapy is a feasible and safe potential treatment for type 1 diabetes.

This study included a very small number of participants. Further studies with larger populations are required.

This study did not include a group of patients not treated, for use as a comparison. Instead, patients in this study were compared to a historical control group (patients included in other studies).