Pancreatic islet transplantation remains an experimental therapy for patients with type 1 diabetes, but studies continue to show promising results.
During the Joint ADA/Diabetes UK Symposium, four researchers will offer a snapshot of where islet transplantation stands today and offer perspective on what must happen in the future to advance the therapy. The two-hour session, which will begin at 2:15 p.m. CT Sunday, June 14, is titled Islet Transplantation—Quo Vadis?
“There may be other cell-based therapies ahead, but those are pretty far away and still works-in-progress. But islets are here now and they work,” said symposium Chair Andrew M. Posselt, MD, PhD. “We know they work. We just have to be able to implement them clinically without resorting to study protocols. I think we can help a lot of patients that way.”
Dr. Posselt, Director of the Pancreatic Islet Transplant Program at the University of California, San Francisco, will discuss the health economics of islet cell transplantation. Also during the symposium, Franca B. Barton, MS, a biostatisticianat The Emmes Company, will review outcomes data for islet cell transplantation. Rebecca S. Monson, MD, MPH, will discuss patient expectations, including questions about immunosuppression, risk of malignancy, and procedure invasiveness. And Ilan Irony, MD, Deputy Director of the Division of Clinical Evaluation and Pharmacology/Toxicology at the U.S. Food and Drug Administration, will discuss the timeline for FDA regulation of islet transplantation and FDA guidance for the development of allogeneic pancreatic islets.
“This symposium puts together different perspectives on this promising treatment, from the report of results from major studies in the field, to patient perspectives, to regulatory issues, and the health economics,” Dr. Irony said. “The aim is to provide a multifaceted understanding of the issues.”
Early trials indicated that efficacy decreased about a year after islet transplantation, but improved isolation techniques and immunosuppression have led to better results, Dr. Posselt said. More recent studies have shown that islet transplantation has efficacy similar to full pancreas transplantation in certain type 1 diabetes populations while being significantly less risky or invasive.
Studies also show that islet transplantation is cost-beneficial compared to insulin therapy after seven to eight years when using insulin independence as the target, Dr. Posselt said. But he noted that even when islet transplantation doesn’t lead to insulin independence, it can still improve A1C and overall diabetes management while preventing potentially fatal hypoglycemic episodes.
Islet transplantation also expands the pool of type 1 diabetes patients who are candidates for transplant therapy. A full pancreas transplant requires patients to be young and healthy. Dr. Posselt said nobody over 50 is considered for a full transplant at his facility.
“That excludes a lot of people who may have difficulties controlling their diabetes, who are older, or have comorbidities,” he said. “By allowing them to get an islet transplant, you would improve their quality of life and overall health dramatically.”
In the U.S., Dr. Posselt said the goal is for insurance to cover islet transplantation as a standard of care for certain type 1 diabetes. That has already occurred in Australia, Canada, and several European countries. The long regulatory process in the U.S. remains a big hurdle, Dr. Posselt said, even though the U.S. has performed the most islet transplantation procedures.
Dr. Irony will offer the FDA’s perspective on what is needed to move islet transplantation beyond the experimental stage in the U.S.
“The published reports suggest that islet transplantation could restore a state of control of blood sugars without the feared episodes of very low blood sugars in a portion of patients who are eligible for, and undergo these procedures,” Dr. Irony said. “However, the studies were conducted under investigational new drug applications and we are still waiting for submission of marketing applications for these products so that we can review independently and analyze the detailed results.”