Insulin producing cell transplantation treatment set to be licenced for type 1 diabetics

by Barbara Hewitt on April 20, 2016

Scientists in the United States are in a position to seek a licence for new treatment for type 1 diabetes involving the transplantation of islets that contain insulin producing cells.

In what is being described as a major step forward for this kind of treatment a new clinical trial results show that transplantation of pancreatic islets prevents severe, potentially life threatening drops in blood sugar in people with type 1 diabetes.

Researchers found that the treatment was effective for people who experienced episodes of severe hypoglycaemia, low blood sugar levels that can lead to seizures, loss of consciousness and death, despite receiving expert care.

laboratory-science

The Phase 3 trial was designed in consultation with the US Food and Drug Administration to enable potential future licensure of the manufacture of purified human pancreatic islets.

“The findings suggest that for people who continue to have life altering severe hypoglycaemia despite optimal medical management, islet transplantation offers a potentially lifesaving treatment that in the majority of cases eliminates severe hypoglycaemic events while conferring excellent control of blood sugar,” said Anthony Fauci director of the National Institute of Allergy and Infectious Diseases (NIAID).

He explained that the treatment carries risks, including infections and lowered kidney function as a result of people taking the immune suppressing drugs needed to prevent rejection of the donor islets. However, although some of the side effects were serious, none led to death or disability. Currently in the United States, islet transplantation is available only in clinical trials.

“While still experimental, and with risks that must be weighed carefully, the promise of islet transplantation is undeniable and encouraging,” said Griffin Rodgers, director of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDKD). He added that even with the best care, about 30% of people with type 1 diabetes aren’t aware of dangerous drops in blood glucose levels.

In type 1 diabetes, the immune system attacks and destroys insulin producing cells in the islets of the pancreas. People with type 1 diabetes need lifelong treatment with insulin, which helps transport the sugar glucose from the bloodstream into cells, where it serves as a key energy source. Even with insulin therapy, people with type 1 diabetes frequently experience fluctuations in blood sugar levels.

Hypoglycaemia, or low blood sugar, typically is accompanied by symptoms such as tremors, sweating and heart palpitations that prompt people to eat or drink to raise their blood sugar levels. Those who do not experience these early warning signs, a condition called impaired awareness of hypoglycaemia, are at increased risk for severe hypoglycaemic events, during which the person is unable to treat himself or herself.

Treatments such as behavioural therapies or continuous glucose monitoring systems can prevent these events in many but not all people with this impaired awareness, leaving a substantial number of people at risk.

The study involved 48 people who had persistent impaired awareness of hypoglycaemia and experienced severe hypoglycaemic events despite expert care by a diabetes specialist or endocrinologist.

Scientists at eight study sites in North America used a standardised manufacturing protocol to prepare purified islets from the pancreases of deceased human donors. All study participants received at least one transplant of islets injected into the portal vein, the major vessel that carries blood from the intestine into the liver. Islet recipients currently must take immunosuppressive drugs for the rest of their lives to prevent their immune systems from rejecting the transplanted cells.

One year after the first transplant, 88% of study participants were free of severe hypoglycaemic events, had established near normal control of glucose levels, and had restored hypoglycaemic awareness. After two years, 71% continued to meet these criteria for transplant success.

Even a small number of functioning, insulin producing cells can restore hypoglycaemic awareness, although transplant recipients may need to continue taking insulin to fully regulate blood glucose levels.

Participants who still needed insulin 75 days after transplant were eligible for another islet infusion and 25 received a second transplant, and one received three. After one year, 52% no longer needed insulin therapy.

“This is the first licence enabling trial of a cellular product for treatment of type 1 diabetes. Licensure is critical because it will ensure the quality, consistency and safety of the islet product, provide greater patient access to islet transplantation and accelerate continued research that we hope would make this procedure suitable for a broader population of people with type 1 diabetes,” said NIAID Transplantation Branch Chief Nancy Bridges.

The researchers are continuing to follow participants to determine whether the benefits of restoring near normal blood glucose control and protection from severe hypoglycaemic events will outweigh the risks associated with chronic immunosuppression.

“For people unable to safely control type 1 diabetes, islet transplantation offers real hope for preventing severe, life threatening hypoglycaemia. However, as immunosuppression drugs required for transplantation can have significant adverse side effects, the treatment only makes sense for people who have frequent severe hypoglycaemia despite optimal diabetes management, or for those already on immunosuppressant drugs for a kidney transplant, a group being studied in another Phase 3 trial,” said study co-author Tom Eggerman, NIDDK scientific officer.


The opinions expressed in this article do not necessarily reflect the views of the DiabetesForum.com Community and should not be interpreted as medical advice. Please see your doctor before making any changes to your diabetes management plan.

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