Diabetes Research Institute participates in NIH-funded multi-center clinical trial that opens the door for application to the FDA for approval in the most severe cases of type 1 diabetes
Miami, FL – April 18, 2016 — An unprecedented collaborative study across North America was completed to determine safety and efficacy of islet transplantation – the transplant of the pancreatic cell clusters that contain insulin-producing cells in the pancreas – for treatment of the most severe forms of type 1 diabetes. Eight centers, including the Diabetes Research Institute (DRI) at the University of Miami Miller School of Medicine, found that the treatment was effective in preventing severe hypoglycemia – low blood sugar levels – which represents a particularly feared complication in type 1 diabetes that can lead to seizures, loss of consciousness and even death.
The Phase 3 trial was conducted by the National Institutes of Health (NIH)-sponsored Clinical Islet Transplantation (CIT) Consortium. The investigators designed the study in consultation with the U.S. Food and Drug Administration to enable potential future licensure of the manufacture of purified human pancreatic islets. The results appear online today in Diabetes Care.
“The findings suggest that for people who continue to have life-altering severe hypoglycemia despite optimal medical management, islet transplantation offers a potentially life-saving treatment that in the majority of cases eliminates severe hypoglycemic events while conferring excellent control of blood sugar,” said Anthony S. Fauci, M.D., Director of the National Institute of Allergy and Infectious Diseases (NIAID), one of the NIH centers that funded the study.
“This phase 3 trial could allow the US centers to file a Biologic License Application (BLA) with the FDA for islet transplantation for treatment of the most severe cases of type 1 diabetes, for what could become the first biologically active cell product ever approved in the USA, ” said Camillo Ricordi, M.D., Director of the Diabetes Research Institute and Chairman of the Steering Committee of the NIH CIT Consortium.
“At this time, islet transplantation still requires the use of immunosuppression and therefore is indicated in a very small group of subjects that, despite management of diabetes mellitus under the care of diabetes specialist, continue to have life-threatening, severe hypoglycemia,” said Rodolfo Alejandro, M.D., Professor of Medicine and Director of the DRI Clinical Cell Transplant Program.
To eliminate the need for immunosuppression and make islet transplantation available to all patients who can benefit, the DRI is focused on finding better ways to protect transplanted cells and prevent the autoimmune attack that caused the onset of the disease.
Currently in the U.S., islet transplantation remains an experimental procedure, but this cell replacement therapy is already available to patients in other countries, where it is an approved and reimbursable procedure through one’s health insurance.
The Diabetes Research Institute has been contributing technology and equipment for clinical islet transplantation to academic centers worldwide, including this NIH Consortium that completed this unprecedented effort to standardize protocols across North America. Among the equipment used by each center was the Ricordi Chamber, the core element of an automated method for isolating human pancreatic islets, which was invented by Dr. Ricordi and collaborators, significantly improving the success of islet transplantation.
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.
Hypoglycemia, 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 hypoglycemia—are at increased risk for severe hypoglycemic events, during which the person is unable to treat himself or herself. Treatments such as behavioral 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.
One year after the first transplant, 88 percent of study participants were free of severe hypoglycemic events, had established near-normal control of glucose levels, and had restored hypoglycemic awareness. After two years, 71 percent of participants continued to meet these criteria for transplant success.
Even a small number of functioning, insulin-producing cells can restore hypoglycemic 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. Twenty-five participants received a second transplant, and one received three. After one year, 52 percent of study participants no longer needed insulin therapy.
The researchers are continuing to follow participants to determine whether the benefits of restoring near-normal blood glucose control and protection from severe hypoglycemic events will outweigh the risks associated with chronic immunosuppression.
About the Diabetes Research Institute (DRI)
The Diabetes Research Institute at the University of Miami Miller School of Medicine leads the world in cure-focused research. As the largest and most comprehensive research center dedicated to curing diabetes, the DRI is aggressively working to develop a biological cure by restoring natural insulin production and normalizing blood sugar levels without imposing other risks. Researchers have already shown that transplanted islet cells allow patients to live without the need for insulin therapy. Some study participants have maintained insulin independence for more than 10 years. The DRI is now building upon these promising outcomes by developing a DRI BioHub, a bioengineered “mini organ” that mimics the native pancreas. While various BioHub platforms are being tested in preclinical and clinical studies, the DRI is also developing strategies to eliminate the need for anti-rejection drugs and reset the immune system to block autoimmunity. For more information, please visit DiabetesResearch.org, call 800-321-3437, or Tweet @Diabetes_DRI.
About the National Institutes of Health (NIH)
NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.