Learn how islet transplantation can change the treatment of type 1 diabetes

Type 1 diabetes (T1D) is an autoimmune disease in which the body destroys the pancreatic cells responsible for producing insulin. Therefore, treatment requires daily injections of the hormone to control blood sugar levels. Without it, T1D can lead to serious complications, such as damage to the kidneys, eyes, and heart, and can even lead to death.

The Brazilian Diabetes Society (SBD) estimates that 600,000 Brazilians live with this condition daily, and nearly 9 million people worldwide are affected. Therefore, medicine has been seeking new, more effective treatment options. GLP-1 medications, such as Ozempic and Mounjaro, emerged precisely in this endeavor.

No treatment, however, has proven as effective as a technique that has been performed experimentally: pancreatic islet transplantation. These structures are precisely the group of cells that produce hormones—including insulin—destroyed by type 1 diabetes. For the procedure, islets are obtained from the pancreases of deceased donors, processed in a laboratory, and then implanted into the patient's body. Typically, they are injected into the liver's portal vein, where they can attach themselves and resume insulin production, but studies are also investigating implantation into muscles.

Unlike pancreas transplants, which require major surgery and are usually only recommended in the most severe cases, islet transplants are considered minimally invasive, as they are performed via infusion without major incisions. The most well-known experimental protocol, known as Lantidra, has been used in people who have not responded well to conventional treatments.

On September 8, the University of Illinois in the United States announced that a 69-year-old patient stopped insulin injections just one week after his transplant . The feat increased global interest in the technique and raised hopes among those with diabetes that the treatment could become a cure for the condition.

Endocrinologist Rafael Scarin Borges, medical coordinator at the Aparecida Municipal Hospital (HMAP) in Goiás, managed by Einstein Hospital Israelita, explains that it's still too early to consider islet transplantation a cure for diabetes. "We're still in the experimental stage. Researchers believe that, in the future, it may be possible to achieve insulin independence, but this hasn't yet been demonstrated in humans. The results are encouraging, but larger, longer trials with attention to safety are required before we can talk about a cure," he explains.

Gene editing broadens perspectives

One advantage of islet transplantation is that, with small genetic modifications, it can be performed without requiring the patient to use daily immunosuppressants, as is the case with pancreas transplant recipients. A study published in August in The New England Journal of Medicine revealed that the patient was able to go 12 weeks without using immunosuppressants after the forearm transplant. "Measurements showed stable, glucose-responsive insulin secretion," the article states.

The researchers tested genetically edited islet cells using the CRISPR technique. The strategy removed DNA markers that make the immune system recognize these cells as foreign and added a protein that acts as a sort of "peace signal" for the immune system. "It's an important proof of concept. The biggest challenge has always been avoiding rejection, and if this is confirmed in larger studies, it opens the door to longer-lasting implants," adds Rafael Borges about the study.

Future tests will require greater attention to durability. So far, the effect has only been monitored for three months. It will also be necessary to determine whether larger doses of cells can lead to complete insulin independence. Today, despite the positive results, the patient continued to require periodic injections.

In parallel, stem cell research suggests that, in the future, it will be possible to create "universal" beta cells, capable of producing insulin without being attacked and without the need for gene editing or compatibility studies for transplants. In animals, results have already shown prolonged survival in these cases without immunosuppression. But the transition to humans requires caution.

"It's plausible to imagine large-scale therapies without immunosuppression. But issues such as standardization of production, monitoring, and ways to deactivate the graft, if necessary, will still need to be resolved," said Borges. This advance represents hope, but only larger and longer studies will be able to confirm whether edited islet transplants can transform the treatment of type 1 diabetes.

Source: Einstein Agency

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