First Cell Therapy for Type 1 Diabetes Receives FDA Approval

The FDA achieved another regulatory milestone this year by granting approval on Wednesday 28th June to the first cellular therapy for type 1 diabetes. Developed by CellTrans, a Chicago-based company, Lantidra (donislecel) is a therapy derived from pancreatic islet cells obtained from deceased donors. It is specifically authorized for adults with type 1 diabetes (T1D) who experience recurrent episodes of low blood sugar (hypoglycemia) and struggle to maintain average blood glucose levels. Referred to as "brittle" diabetes, this condition is extremely rare, affecting only three out of every 1,000 individuals with insulin-dependent diabetes, as reported by pharmaphorum. Peter Marks, the director of the FDA's Center for Biologics Evaluation and Research (CBER), emphasized the danger of hypoglycemia, which can lead to injuries resulting from loss of consciousness or seizures. Marks stated in a prepared statement that the approval of Lantidra offers an additional treatment option for individuals living with type 1 diabetes and experiencing recurrent severe hypoglycemia, helping them achieve target blood glucose levels. Lantidra is believed to function by stimulating insulin secretion through the infusion of allogeneic islet beta cells. In some cases, these infused cells may produce sufficient insulin to free the patient from relying on external insulin to control their sugar levels, according to the FDA's press release. The efficacy of donislecel therapy was assessed through two non-randomized, single-arm trials involving a total of 30 patients who had difficulty detecting the onset of hypoglycemia. Each participant received at least one infusion and up to three infusions. Among the 30 patients, 21 were insulin-free for at least one year, 11 did not require insulin for a period ranging from one to five years, and 10 were insulin-free for over five years. However, five patients did not achieve any days of insulin independence. Cell therapy is emerging as a promising treatment option for type 1 diabetes. Vertex recently announced one-year follow-up data from a Phase I/II clinical trial of their investigational stem cell-based therapy, VX-880, revealing that two patients no longer needed insulin injections. VX-880 is an allogeneic stem cell therapy that delivers fully differentiated and insulin-producing islet cells to restore the body's ability to produce insulin in response to glucose levels. Both patients who received the VX-880 therapy experienced significant reductions in hemoglobin A1c (HbA1c) levels one year after receiving the experimental cell therapy. They spent 95% of their time within the target range for blood glucose levels, as reported by Vertex. The American Diabetes Association recommends that individuals with type 1 diabetes spend at least 70% of their time within this target range. Looking to hire within the Cell and Gene space, contact [email protected] today or click the following link https://barringtonjames.com/our-services