GMO cell transplant helps diabetic man produce insulin

A man with type 1 diabetes has become the first patient whose body began producing its own insulin after receiving a genetically modified cell transplant, without the need for anti-rejection drugs, writes Life Science.

Photo: Mikkolem | Dreamstime.com

The case, reported in the New England Journal of Medicine, could be a potential breakthrough in treating the disease, which affects 9,5 million people worldwide.

Type 1 diabetes occurs when a patient’s immune system destroys specialized cells in the pancreas called islet cells. These are responsible for producing insulin, a hormone that regulates blood sugar levels. The condition can be controlled with regular doses of synthetic insulin, but there is no cure for the disease.

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Islet cell transplants can provide longer-term insulin production in people with type 1 diabetes. However, the patient's immune system may then recognize the transplanted cells as foreign and destroy them.

As a result, transplant patients must take immunosuppressants for life, making them vulnerable to infections.

To overcome these difficulties, scientists from Sweden and the US transplanted islet cells from a donor's pancreas. The cells were genetically modified using CRISPR technology to suppress the recipient's immune system's rejection reaction. This is the first time this method has been used in humans.

The authors of the paper say their study, although preliminary, shows that genetically modifying transplanted cells to evade attack by the recipient's immune system is a valuable tool for preventing the immune system from rejecting new cells or organs.

In this new approach, the researchers used CRISPR to make three changes to the genetic code of donor cells to reduce the likelihood of an immune reaction.

Two of these modifications reduced the amount of proteins on the surface of cells that signal our white blood cells whether the cells are foreign. The third change boosted production of a protein called CD47, which prevents other immune cells from attacking.

The genetically edited cells were then injected into the man's forearm. His body did not attack the modified cells, and they produced insulin as normal.

Although the patient was injected with a small dose of the modified cells and still requires daily insulin injections, the case suggests the procedure may be safe.

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The scientists plan to conduct follow-up studies to see if the cells can survive long-term, which would make it easier to control the disease and potentially cure it. Further tests are also needed to determine if the method will work for other patients.

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