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A recent study shows that a new type of stem cell transplantation may help people with type 1 diabetes become insulin free and increase C-peptide levels.

Researchers have used a transplant of a patient’s own treated blood cells to increase and preserve beta cell function in young people recently diagnosed with type 1 diabetes.

The research team, from the US and Brazil, hoped that if they intervened early enough they could wipe out and then rebuild the body’s immune system by using stem cells, preserving a reservoir of beta cells and allowing them to regenerate.

They enrolled Brazilian diabetics aged between 14 and 31 who had been diagnosed within the previous six weeks. After stem cells had been harvested from their blood, they underwent a form of chemotherapy to eliminate the white blood cells causing damage to the pancreas. They were then given transfusions of their own stem cells to help rebuild their immune systems.

The trial results indicate that destroying and restarting the immune system may “retrain” or “reset” the immune system without the immune response that caused type 1 diabetes in these patients initially, at least for a period of time.

While the trial seems to provide proof of concept that the autoimmune response that causes diabetes can be overcome by resetting the immune system, there are a number of serious issues that will need to be addressed before this approach can become widely available.

JDRF Australia’s Research Development Manager Dr Dorota Pawlak said that “Stem cells have the science community very excited but there are still significant challenges.”

“Firstly, it is not known whether the treatment represents a “cure” or a temporary resetting of the immune system with an eventual slide back into the autoimmune destruction of the beta cells.”

“Secondly, the results of this study are only relevant to people who are newly diagnosed and may still have some beta cells function left.”

“Finally, and most importantly, the risks and long term side effects associated with this highly invasive treatment need to be better quantified, mitigated, and weighed against the benefits of the procedure.”

“This is a key point – to deliver a cure to people with type 1 diabetes we need stem cells that are pure, safe and that don’t require the use of heavy immunosuppressive drugs. This is especially important for children with type 1 diabetes.”

Dr Pawlak noted that the work complements the number of studies funded by JDRF that use stem cells both as a research tool, and as a potential therapy for type 1 diabetes.

Journal of the American Medical Association 297(14):1568-76.

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