FDA Approves First Cell Therapy for Type 1 Diabetes

 In a growing string of regulatory milestones this year, the FDA on Wednesday approved the first cellular therapy for type 1 diabetes.

Lantidra (donislecel), developed by Chicago-based CellTrans, is a pancreatic islet cell therapy made from the pancreatic cells of deceased donors. It is authorized for adults with type 1 diabetes (T1D) whose repeated episodes of low blood sugar (hypoglycemia) leave them unable to hit average blood glucose levels.

Also called “brittle” diabetes, the condition is ultra-rare, affecting three in every 1,000 people with insulin-dependent diabetes overall, according to pharmaphorum.

In a prepared statement, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures. Wednesday’s approval “provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” Marks said.

Lantidra is believed to work through the secretion of insulin by the infused allogeneic islet beta cells. In some patients, it’s possible that these infused cells could produce enough insulin that the patient would no longer be beholden to insulin to control their sugar levels, according to the FDA’s press release.

The donislecel therapy was tested in two non-randomized, single-arm trials involving a total of 30 patients who found it difficult to detect the onset of hypoglycemia. The participants were given at least one infusion and a maximum of three. Of the 30 patients, 21 were insulin-free for at least a year; 11 didn’t require insulin for between one and five years and 10 were insulin-free for more than five years. Five patients failed to achieve any days of insulin independence.

Cell therapy appears to be picking up steam as a viable treatment for type 1 diabetes. On Friday, Vertex announced one-year follow-up data from a Phase I/II clinical trial of its investigational stem cell–based therapy VX-880 showing that two patients no longer required insulin injections.

VX-880 is an allogeneic stem cell therapy that delivers fully differentiated and insulin-producing islet cells to restore the body’s glucose-responsive capabilities to produce insulin.

Both patients on the therapy had strong reductions in hemoglobin A1c (HbA1c) one year after receiving the experimental cell therapy and spent 95% of their time within the target range of blood glucose levels, Vertex reported. The American Diabetes Association recommends that T1D patients spend at least 70% of their time within this target range. 

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