Diabetic man with cells published by genes produces his own insulin – no transplanted medication required

A new case study offers an enticing overview of the potential future of transplant medicine. A man with type 1 diabetes is now able to make his own insulin thanks to a pancreatic cell transplant edited with genes – a transplant that did not require typical drugs used to avoid rejection.

Scientists in Sweden and the United States have conducted research this week in the New England Journal of Medicine. The 42 -year -old man with long -standing diabetes has received given islet cells that have been genetically modified via CRISPR to prevent immune rejection. About four months after the procedure, its cells entirely published by the genes continued to produce insulin without causing an immune response.

“Our study, although preliminary, suggests that immunity is an alternative concept for bypassing the allowance,” wrote the authors in the article.

People with type 1 diabetes have immune systems that destroy pancreatic cells responsible for insulin. The condition can be managed with regular doses of synthetic insulin, but people’s health always tends to worsen over time. Recently, scientists have studied if the data transplants of data islands can provide a self -insulin replacement supply for type 1 diabetics – an effective remedy, in other words, provided that cells can survive in the long term.

Until now, the first clinical trials of technology have seemed promising, but these transplants still require immunosuppressive therapy for life to ensure that the body of the host does not go after the given cells. As effective as these drugs are, they have their well -known drawbacks, as weaken people ‘immunity to real threats such as infections.

Study researchers have experienced a unique approach to bypass the need for these drugs. They first tested it on mice and monkeys, this new case being the first test in humans.

They have created three specific modifications in the cells given, all intended to calm the most likely responses in the immune system. Two of the modifications exhaust the supply of HLA class I and II antigen cells, proteins that say to our T cells if another cell is foreign or not. A third edition means that given cells make more than another protein called CD47 which inhibits the activity of other immune cells which would normally target them.

The researchers injected the modified cells into the human forearm. The changes did not completely succeed in certain cells, and the human immune system quickly killed these cells. But as hoped for, his body left the cells entirely published alone, even without immunosuppressants. Surviving cells produced insulin as usual and man seemed to manage 12 weeks later. He lived several undesirable events, but none was serious or linked to the transplanted cells themselves (he had slightly inflamed veins where a catheter was placed, for example).

This study is only proof of concept, more important to show that the procedure can be carried out safely than to prove that it actually works. Man has received a relatively low dose of the given cells, probably too low for his body to be able to produce enough insulin to himself to no longer need treatment. Monitoring is also necessary to know if these cells can survive in the long term.

But there is a lot to encourage. And this is the last sign that scientists are really about to make type 1 diabetes curable. Other research teams have achieved early success in the use of similar transplants to cure the condition, including some that have also avoided requiring immunosuppress therapy.