A Call To Re-Evaluate Islet-Cell Regulations
By Tyler Menichiello, contributing editor
Autologous islet cell transplantation (AIT) is a procedure for patients who undergo a pancreatectomy, usually as a result of severe pancreatic inflammation/chronic pancreatitis. Insulin-producing islet cells are isolated from the removed pancreas and re-infused into patients, where they find a home in the sinusoids of the liver. There, they more-or-less become a de facto pancreas, preserving insulin production to stabilize patients’ blood-glucose levels.
One company that plays a key role in the isolation of islets for AIT is Pittsburgh-based Imagine Pharma. The Imagine Islet Center is one of only 10 islet-isolation centers designated by the Integrated Islet Distribution Program, and it is run by renowned islet-isolation expert Dr. Rita Bottino. Recently, I had the opportunity to speak with Bottino about the FDA’s regulations regarding islet-cell transplantation.
Under current FDA regulations, islets are considered a biologic drug, which Bottino doesn’t agree with — and she’s not alone. A number of experts believe the requirement of a BLA for islet allotransplantation has stalled the field for decades. Bottino and others in the field think that islets and islet allotransplantation should be treated more like organs for organ transplantation rather than a product that needs BLA approval. Bottino says addressing the (broken) regulatory framework around islets would provide more access to treatment and, ultimately, better serve patients living with Type 1 diabetes.
Why Islets Shouldn’t Require A BLA
After 35 years, Bottino has performed a lot of islet isolations — she estimates close to 2,000. The one thing she’s learned is that there’s not much consistency batch-to-batch. “Every islet preparation is different from the previous one, and it’s going to be different from the next one,” she tells me. Everything from their morphology to even how the islets are obtained differs by the batch.
“Islets are very different than single cells or drugs,” Bottino says. “One size doesn’t fit all. They’re not like a compound or one type of cell that you can characterize. It is very difficult to express characteristics in islets like purity, potency, sterility, or even the ability to cause a certain result or effect in a patient based on characteristics after isolation.”
It’s like islets have a unique signature, she says. Therein lies the logical fallacy of regulating them as a standardized, biologic drug product — they’re anything but. Requiring a BLA for allotransplantation imposes an inhibitive cost (to providers) on a treatment that can potentially help a lot of patients in need.
A New Regulatory Framework To Help Patients
I could hear the urgency in Bottino’s voice as she told me how limiting these regulations are for patients. “We have access to organs that could be used for clinical islet allotransplantation, but we can’t use them,” she says.
There’s a strong body of clinical trial data that supports the efficacy of islet allotransplantation, especially for Type 1 diabetics with hypoglycemia. Bottino thinks that regulating islet cells as any other organ for transplant would allow for even more supervision of clinical outcomes.
“It's really clear to us that allotransplantation works and can help patients,” Bottino tells me. “We have donor tissue that we could use to benefit patients. A change in regulation would indeed make a difference in the life of patients with Type 1 diabetes.”
Luckily, the push to re-evaluate the FDA’s regulation of islets is in progress. In 2023, The ISLET Act was introduced to U.S. House of Representatives. While it has yet to be passed (as of August 15, 2024), it’s a positive step in the right direction. Hopefully, The ISLET Act gets passed sooner rather than later, so the abundance of “beautiful” donor islets (as Bottino affectionately calls them) can help more patients in need.