Stem cell transplant findings could lead to personalised type 1 diabetes treatments

Jack Woodfield
Wed, 08 Mar 2017
Stem cell transplant findings could lead to personalised type 1 diabetes treatments
A clinical trial has shown promising findings from stem cell transplants in people with type 1 diabetes, with some patients coming off insulin injections for several years.

This international research could also lead to better outcomes for more patients who undergo stem cell transplantation, according to senior author Dr Bart Roep, City of Hope and director of the Wanek Family Project for Type 1 Diabetes.

"This study paves the way for personalised therapy in type 1 diabetes," Roep said. "We now understand stem cell transplants can succeed in treating diabetes for some, but not in others, and we can predict either outcome before the therapy is administered by 'reading' the immune signature of the patient."

Roep and colleagues were testing a type of stem cell transplantation called autologous hematopoietic stem cell transplantation (AHSCT).

This involves a person's own stem cells being transplanted with researchers able to see how much insulin is then made by the pancreas.

The researchers' aim was for the procedure to induce insulin independence in participants, and understand why some patients can achieve long-term clinical benefits from stem cell transplantation and others cannot.

Twenty-one participants with type 1 diabetes received this transplantation in Brazil. They were then monitored and assessed every six months.

Most patients were able to come off insulin for an average of 3.5 years, while one patient was free of insulin for more than eight years after transplantation, without any major side effects.

Roep added that some participants received little to no benefit from transplantation. "However, we discovered the immune signature predicting these outcomes - either favourable or not - which is the first step towards personalised medicine in type 1 diabetes. We have a foot in the door," he said.

"Understanding why it sometimes fails will allow us to design new treatment strategies for those less fortunate patients."

Roep also acknowledged that because immunosuppressive drugs are required before transplantation, this operation remains risky and will unlikely become the first line of defence for type 1 diabetes.

The findings appear online in the journal Frontiers in Immunology.
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