For type 1 diabetes, insulin has long been the essential treatment method. Blood glucose monitoring, frequent insulin injections, even insulin pumps are used to help diabetics control their glucose levels and avoid dangerous spikes and dips in their blood sugar.
But for some, being captive to the use of insulin may soon be a thing of the past.
One patient, Erika Totten, who was part of a key phase III clinical trial at Penn's Institute for Diabetes, Obesity and Metabolism, received transplanted pancreatic islet cells and is no longer insulin-dependent. In fact, now seven years removed from her transplant, Erika doesn't need insulin at all.
"The procedure gave me back my pre-diabetes life," said Totten in a recent Philadelphia Inquirer article.
Investigators on the trial, including Ali Naji, MD, PhD, a professor of Surgery, and Michael R. Rickels, MD, MS, an associate professor of Medicine, both of the Type 1 Diabetes Unit at Penn, found that transplanting purified human pancreatic islet cells into type 1 diabetics can lead to nearly normal glycemic control and no longer being reliant on insulin.
“This phase III clinical trial examined the longer term effects of islet cell transplantation, aiming to secure [U.S. Food and Drug Administration] approval of islet cells as a biological product for treating type 1 diabetes," Rickels said.
Researchers from the multi-institutional study found that transplanted islets provide better glycemic control, improved hypoglycemia awareness, and durable protection against severe hypoglycemic events in type 1 diabetics who have otherwise experienced significant glucose instability with other types of insulin delivery methods. Patients with this severe type 1 diabetes can experience such dangerous highs and lows that they can become disoriented, confused, and in extreme cases, some have even been pulled over for suspected drunk driving.
Type 1 diabetes is a disease in which the body attacks its own insulin-producing cells, so it can no longer regulate insulin production or glucose absorption properly.
"Pancreatic islets are tiny clusters of cells scattered throughout the pancreas, which produce the hormone insulin," Rickels said. "This insulin helps cells in the body absorb glucose [sugar] from the bloodstream and use it for energy."
This study reports that at one year following islet transplantation nearly 88 percent of the islet cell recipients were free of severe hypoglycemic events, had restored hypoglycemia awareness, and had excellent glycemic control. And at two years, numbers remained positively high at 77 percent.
In the simplest terms, this means that patients who receive transplanted pancreatic islet cells may be less likely to experience the normal side effects of their diabetes, such as dangerously high or low blood glucose.
And these results certainly put the procedure steps closer to FDA approval.
Naji notes that "the next step in this research is to begin the process of applying for a BLA, or Biologic License, from the FDA which is needed to take this therapy from an experimental stage to direct patient treatment."
In order for the researchers to obtain FDA approval, there is a comprehensive application process that needs to be followed. This encompasses a pre-screening evaluation, or GAP analysis, to ensure there are no inefficiencies in an institution's facilities, to review the research data for safety and efficacy, and to evaluate the staff who will work directly to prepare human islet cells for transplantation. Following the pre-screening and once there is a standard operating procedure in place, the full FDA application can be submitted.
"I am encouraged that Penn Medicine has expressed interest in establishing the islet cell transplantation program as a novel therapy for our patients," said Naji. "But given the significant efforts that go into applying for FDA approval, I believe we are still a few years away from this therapy being FDA approved and available to patients."
Pancreatic islet cell translation is currently a standard of care - approved and covered by insurers – for patients with type 1 diabetes with severe hyperglycemic events in Australia, Canada, the UK, and the European Union.
"Our hope is that this therapy will allow us to look beyond addressing complications of unstable glucose control, and focus on the long term benefits of normalizing glucose levels entirely in type 1 diabetes," Rickels said.