The FDA has approved Lantidra, a groundbreaking donor-derived pancreatic islet cell therapy for type 1 diabetes, providing new treatment options for adults who cannot control their blood sugar with insulin alone.
The FDA has given the green light to Lantidra (donislecel), the inaugural treatment utilizing donor-derived pancreatic islet cells for type 1 diabetes (T1D). Created by CellTrans, it’s designed for adults with T1D who struggle to control their blood sugar with insulin. Islet cell transplants have been accessible in the US for more than two decades, but only as experimental therapies in clinical trials. The approval of Lantidra confirms allogeneic cells as a therapeutic option for those who can’t detect blood sugar drops and encounter challenges with insulin dosing.
The decision to approve Lantidra was grounded in clinical trials involving 30 T1D patients who experienced frequent hypoglycemia and couldn’t effectively manage their condition. Participants underwent one to three cell infusions into the liver. Following the infusion, 21 participants didn’t need insulin for at least one year, 11 for one to five years, and 10 for more than five years. Most participants faced at least one serious side effect from the infusions and the immunosuppressive regimen required to protect the islet cells.
Vertex Pharmaceuticals is progressing with stem-cell-derived islets as a scalable and uniform alternative to donor islet cells. VX-880 is currently undergoing phase 1/2 trials for T1D patients, and recent findings revealed that all six tested individuals synthesized their own insulin. Two patients with extended follow-up experienced no severe hypoglycemic episodes and reduced or eliminated their use of external insulin. To enable large-scale production of VX-880, Vertex has collaborated with Lonza, which is erecting a dedicated production facility in the US. Others are exploring islet encapsulation strategies to protect the cells from immune rejection. Eli Lilly has acquired Sigilon Therapeutics and their preliminary, encapsulated stem-cell-derived islet therapy SIG-002 for $34 million plus additional milestones.
In conclusion, the FDA’s approval of Lantidra signifies a notable step forward in treating T1D. This pioneering therapy, utilizing donor-derived pancreatic islet cells, provides a new avenue of treatment for adults struggling with unmanageable blood sugar levels despite insulin therapy. The approval not only validates the feasibility of using allogeneic cells as a treatment option but also recognizes the challenges faced by individuals who have difficulty sensing blood sugar fluctuations and managing insulin doses.
While the approval is based on promising research demonstrating Lantidra’s effectiveness in improving glycemic control, it’s crucial to acknowledge the associated risks and adverse effects, as evidenced by participants’ experiences. Looking forward, ongoing research efforts by Vertex Pharmaceuticals and other entities offer hope for further advancements. These initiatives aim to develop scalable and standardized alternatives, such as stem-cell-derived islets like VX-880, and innovative methods like islet encapsulation to enhance the safety and effectiveness of cell-based therapies.
Through collaborations and investments from companies like Vertex Pharmaceuticals, Lonza, and Eli Lilly, the future of cell-based therapies for T1D holds promise, potentially transforming the quality of life for individuals managing this chronic condition.
By Miriana Braghin, Department of Pharmacological and Biomolecular Sciences “Rodolfo Paoletti”, University of Milan.