Phase II clinical trial to test safety and efficacy of transplanted encapsulated islets

The Juvenile Diabetes Research Foundation announced today that it is partnering with Living Cell Technologies (LCT), a New Zealand-based biotechnology company focused on developing cell based therapeutics, in a Phase II clinical trial to study the safety and effectiveness of transplanting encapsulated insulin-producing cells from pigs as a treatment for type 1 diabetes with significant hypoglycemia unawareness.

Through its Industry Discovery and Development Partnership program, JDRF will support the second part of a Phase II trial in New Zealand of LCT's DIABECELL- product; the trial will test the effectiveness of the transplant product when given in higher amounts in people with established and unstable type 1 diabetes who suffer from frequent hypoglycemia and hypoglycemia unawareness - an inability to sense when blood sugar is trending dangerously low.

The DIABECELL- product comprises encapsulated, insulin-producing pancreatic islets derived from specially bred pigs. They are implanted into the abdomens of people with diabetes through a laparoscopic procedure, and work by sensing blood glucose and secreting insulin in response to high blood glucose levels. The encapsulation technology developed by LCT in DIABECELL- does not require immunosuppressant drugs to suppress the rejection of the islets by the immune system.

In addition to assessing the safety of the treatment, the trial will also study whether the transplanted islets improve blood glucose control, reduce hypoglycemic episodes, reduce daily insulin requirements, and improve the quality of life of participants in the trial.

According to Professor Bob Elliott, Medical Director of LCT, early results from the clinical trial, which began in October 2009, showed that the cell implants eliminated hypoglycemia unawareness in one of the trial participants -- a complication that occurs in as many as 2 in 10 people with long-term type 1 diabetes, and can be fatal.

Type 1 diabetes is an autoimmune disease in which a person's pancreas stops producing insulin, a hormone that enables people to get energy from food. Type 1 diabetes strikes children and adults suddenly, and lasts a lifetime. People with type 1 diabetes must take multiple injections of insulin daily or continuous infusion of insulin through a pump to survive. Intensive insulin treatment to normalize blood glucose levels and prevent future eye, kidney, nervous system and cardiovascular complications can sometimes result in hypoglycemic unawareness, a loss of the normal stress response to low blood glucose levels. People with type 1 diabetes and suffer from hypoglycemic unawareness feel no warning symptoms when their blood glucose levels drop to low levels that can impair consciousness and can lead to accidents and coma.

The insulin-producing islets used in the study come from pigs grown and harvested in optimal conditions whose health status was closely controlled and monitored, reducing the risk of safety threats. As pig insulin has been known to work in man, pigs are considered ideal candidates to provide islets for xenotransplantation (the transplantation of insulin-producing islets from non-human species into people with type 1 diabetes), the type of procedure described in the trial.

Pancreatic islet transplantation, a major research focus within JDRF's Beta Cell Therapies program, has the potential to normalize blood glucose levels by restoring insulin production for patients with type 1 diabetes. But because of a limited supply of human donors for pancreatic islets, scientists have investigated other possible sources of insulin-producing cells, which include the transplantation of insulin-producing islets from animals into people with type 1 diabetes (xenotransplantation). In addition, encapsulating islets has the potential to protect the transplant from immune rejection and eliminate the need for chronic immunosuppressive drugs in islet transplantation. Xenotransplantation and islet encapsulation are important areas of research interest to JDRF, funding approximately $3 million in xenotransplantation, and $3.5 million in islet encapsulation last year.

"The initial results from the first phase of the trial showed us a significant feature of LCT's product in its ability to protect the transplanted cells from immune rejection without the use of immunosuppressive anti-rejection drugs," said Julia Greenstein, Ph.D., Director of Beta Cell Therapies at JDRF. "Encapsulation for immuno-protection is one of JDRF's research priorities, and the success of this trial could have an important impact on this field of research and the development of a commercially-viable cell replacement therapy for people with type 1 diabetes."

"We are delighted that the JDRF is supporting the progress we are making to advance DIABECELL- towards normalizing the lives of people with type 1 diabetes," said Dr. Paul Tan, Chief Executive Officer for LCT.

The collaboration with JDRF and LCT Technologies for the Phase II trial is part of JDRF's innovative Industry and Discovery and Development Partnership program. Through the program, JDRF partners with pharmaceutical, biotech, and medical device businesses that seek to develop drugs, treatments, technologies, and other therapeutics leading to a cure, reversal, or prevention of type 1 diabetes and its complications. To date, JDRF has 27 industry partners across a range of research areas since the IDDP program was established in 2004.