First Cellular Therapy for Type 1 Diabetes Receives FDA Approval: Lantidra

Diabetes is a major disease that reduces quality of life worldwide. Type 1 diabetes, one of its forms, begins at a younger age and can be difficult to manage, particularly for patients with severe fluctuations between high and low blood sugar. However, a new FDA (Food and Drug Administration of the United States) approval offers important hope for these patients. The FDA has approved the first cellular therapy used to treat patients with Type 1 diabetes. This approval is an important step in diabetes treatment and could significantly improve patients’ quality of life in the future.

Historic FDA Approval: Lantidra

On 28 June 2023, the FDA approved Lantidra, the first allogeneic pancreatic islet-cell therapy made from deceased donors’ pancreatic cells, for the treatment of Type 1 diabetes. Lantidra was approved for adults with Type 1 diabetes who cannot reach target glycated hemoglobin levels (average blood glucose levels) because of severe hypoglycemia (low blood sugar) episodes despite intensive diabetes management and education.

Severe hypoglycemia is a dangerous condition that can cause injuries resulting from loss of consciousness or seizures. This approval is for the first cellular therapy to treat patients with Type 1 diabetes. It offers an additional treatment option to help individuals with Type 1 diabetes and recurrent severe hypoglycemia achieve target blood glucose levels.

Type 1 diabetes is a chronic autoimmune disease requiring lifelong care: the person’s immune system attacks insulin-producing pancreatic islet cells. People with diabetes need to check their blood glucose and use insulin every day. However, some people with Type 1 diabetes may struggle to manage the amount of insulin they need each day to prevent hyperglycemia (high blood sugar). Lantidra offers a potential treatment option for these patients.

How Lantidra Works

Lantidra’s main mechanism of action involves giving patients allogeneic islet beta cells (obtained from another individual), which then secrete insulin. These cells are injected into the body with Lantidra. In some patients, they can produce enough insulin to eliminate the need for externally administered insulin, through injections or pumps, to control blood sugar.

These allogeneic islet beta cells are obtained from pancreatic cells taken from deceased donors. Infusing Lantidra into the body, specifically into the hepatic portal vein, allows the islet beta cells to enter the bloodstream and reach the liver.

The Clinical Research Behind Lantidra’s FDA Approval 

Lantidra’s safety and efficacy were evaluated in two nonrandomized, single-arm studies in which a total of 30 participants with type 1 diabetes and hypoglycemia unawareness received at least one and no more than three infusions.

The studies’ efficacy results were as follows:

• 21 participants did not need to take insulin for a year or longer;
• 11 participants did not need insulin for one to five years, and 10 participants for more than five years.
• 5 participants never achieved insulin independence.

The side effects associated with Lantidra varied with the number of infusions each participant received and the duration of follow-up, and may not reflect the rates observed in practice. The most common side effects included nausea, fatigue, anemia, diarrhea and abdominal pain. Most participants experienced at least one serious adverse effect related to the procedure for infusing Lantidra into the hepatic portal vein or the use of immunosuppressive medicines needed to keep the islet cells alive. Some serious adverse effects required immunosuppressive medicines to be stopped, resulting in loss of islet-cell function and insulin independence. These effects should be considered when evaluating Lantidra’s benefits and risks for each patient.

The FDA granted approval of Lantidra to CellTrans Inc.

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A Brief History of Pancreatic Islet-Cell Transplantation

Pancreatic islet-cell transplantation is not a new concept. Early investigations were conducted in the late 19th century by researchers such as British surgeon Charles Pybus. However, modern islet (pancreatic islet-cell) transplantation research is generally traced to Paul Lacy’s work beginning in the late 1960s. Lacy’s team developed a collagenase-based method for isolating islets, paving the way for subsequent in vitro and in vivo islet experiments. Later studies showed that transplanted islets could reverse diabetes in rodents and nonhuman primates.

The first successful trial of human islet-cell transplantation took place at the University of Pittsburgh in 1990. However, despite continuous improvements in practice, only around 10% of islet recipients achieved normal blood glucose levels (euglycemia) in the late 1990s.

In 2000, Dr. James Shapiro and colleagues published a report on seven people who achieved euglycemia after islet transplantation using a steroid (cortisone)-free protocol and large numbers of donor islets. Known as the “Edmonton protocol,” it was adopted by islet transplantation centers worldwide, greatly increasing transplantation success.

From this historical perspective, Lantidra differs from earlier islet transplantation efforts in being the first FDA-approved cellular therapy and potentially being applicable to a broader patient population. Its method of infusing islet cells into the hepatic portal vein also has particular significance. However, it is important to remember that both approaches require immunosuppressive medicines to suppress the immune system, and these medicines can have side effects.

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