First Cell Therapy for Type 1 Diabetes Receives FDA Approval, Made Possible by Startup’s Success

CellTrans, a small biotech company, has received the first FDA approval for a cell therapy for type 1 diabetes. The therapy, known as Lantidra, is intended for adults with type 1 diabetes who are unable to achieve their target blood glucose levels through regular insulin administration. Lantidra, which is made from pancreatic cells sourced from deceased donors, is administered via an infusion into the liver. The therapy aims to provide enough insulin for patients to stop using injections or an insulin pump. The effects of Lantidra can last for years in some patients.
The FDA approval of Lantidra is based on results from two non-randomized, single-arm studies, which involved a total of 30 type 1 diabetes patients with hypoglycemic unawareness. The studies showed that 21 patients were insulin independent for one year or more, 11 patients were insulin independent for one to five years, and 10 patients were insulin independent for more than five years. However, five participants did not achieve any insulin independence.
To prevent the body from rejecting the transplanted cells, Lantidra must be used in conjunction with immunosuppressive drugs. However, chronic immunosuppression can increase the risk of infections and cancer. The most common reactions reported in clinical trials of Lantidra included nausea, fatigue, anemia, diarrhea, and abdominal pain. A majority of participants experienced at least one serious adverse reaction related to the infusion procedure and the use of immunosuppressive medications. In some cases, the adverse reactions resulted in the discontinuation of the medications, rendering the transplanted islet cells ineffective.
In addition to CellTrans, other companies are also developing cell therapies for type 1 diabetes. Vertex Pharmaceuticals has a candidate called VX-880, which uses lab-grown islet cells derived from stem cells. Preliminary data showed that the therapy led to improvements in blood sugar control and reduced or eliminated the need for insulin shots or infusion pumps. Eli Lilly has acquired Sigilon Therapeutics, a company developing a therapeutic candidate that uses stem cell-derived islet cells protected from the immune system by a biocompatible sphere. Quell Therapeutics, a London-based startup, is using an autologous approach by engineering a patient’s own regulatory T cells. AstraZeneca has partnered with Quell to develop therapies for type 1 diabetes and inflammatory bowel disease.
While cell therapies hold promise for achieving insulin independence in type 1 diabetes, they come with risks and challenges. The use of immunosuppressive drugs can have serious side effects, and further research is needed to improve the safety and efficacy of these therapies. However, the FDA approval of Lantidra represents a significant milestone in the field of type 1 diabetes treatment and brings hope to patients who struggle to manage their blood sugar levels.