Making for another landmark cell and gene therapy approval this month, the US Food and Drug Administration (FDA) has signed off on CellTrans’ Lantidra (donislecel), the first cell therapy for type 1 diabetes.
The treatment is derived from deceased donor pancreatic cells and is indicated for patients who are unable to achieve average blood glucose levels (glycated hemoglobin) with daily insulin injections or with continuous infusion through a pump because of repeated episodes of severe hypoglycemia (low blood sugar). For these patients, insulin dosing becomes difficult because they cannot be sure when their blood glucose levels are dropping too low, something known as hypoglycemia unawareness.
Lantidra is thought to increase insulin secretion through the infusion of allogeneic insulin-producing islet beta cells. Given as a single infusion into the hepatic (liver) portal vein, the infused cells make enough insulin so that patients no longer have to take it by injection or pump. A second infusion may be required depending on how a patient responds to the first dose.
Type 1 diabetes is a chronic autoimmune disease that requires routine administration of insulin through multiple daily injections or continuous infusion using a pump. To dose their insulin, individuals with type 1 diabetes have to continuously monitor their blood glucose levels throughout the day.
In the US, there are currently 1.9 million people who have type 1 diabetes.
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“Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the FDA’s news release. “Today’s approval … provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels.”
The safety and effectiveness of Lantidra were evaluated in two non-randomized, single-arm studies involving a total of 30 participants with type 1 diabetes and hypoglycemic unawareness. Patients received at least one infusion of the treatment and up to a maximum of three infusions.
Results showed that 21 participants that received Lantidra did not need to take insulin for one year or more, with 11 participants not needing insulin for one to five years and ten participants not requiring it for more than five years.
Five participants did not achieve any days of insulin independence but despite this, the therapy still shows promise as it can help achieve more stable insulin baseline levels with fewer fluctuations, making it easier to manage the condition.
Lantidra has been in the making for two decades since CellTrans was first established in 2003 by Professor Jose Oberholzer at the University of Illinois.
In April 2021, an FDA advisory committee voted 12-4 in favor of the therapy, saying that the benefits of the therapy outweighed the risks.
Vertex Pharmaceuticals has been in the spotlight for its work in developing a stem cell therapy for type 1 diabetes. Last week, the company reported that two patients who had received the stem cell treatment more than a year ago no longer needed injections.
Stem cell treatments are deemed to be more reliable and healthier compared with allogenic cells derived from deceased donors.
While acknowledging their potential, some islet and pancreas transplantation experts part of . The group blasts the “outdated” regulations around the treatment, saying that because cells come from deceased donors, the FDA should treat islet transplantation as an organ transplant rather than a drug, which other regulatory bodies have.
In a 2021 paper, the group claimed that “final islet product sterility and potency cannot be confirmed” under the current regulations.
To overcome these concerns, there are new technologies being developed that could bypass the need of a donor. For example, Novo Nordisk recently partnered with Canadian biotech Aspect Biosystems to develop 3D-printed beta cells in a $75 million deal.