An Illinois woman has been cured of type 1 diabetes after 25 years of insulin dependence, thanks to a pioneering stem cell therapy. Marlaina Goedel, 30, received an islet cell transplant as part of a clinical trial at the University of Chicago Medicine Transplant Institute, freeing her from daily insulin shots and allowing her to enjoy a normal life.
Islet Cell Transplantation: A 'Lifechanging' Infusion
The treatment involved a one-off infusion of islet cells from a deceased donor's pancreas into her liver. These cells then lodged into the blood vessels and started producing insulin. Within four weeks, Ms. Goedel no longer needed insulin injections. According to Dr. Witkowski, who conducted the trial, she was effectively cured.
The Role of Tegoprubart
Patients in the trial were administered tegoprubart, an experimental drug composed of lab-made antibodies. This drug is designed to trick the immune system into accepting the transplanted cells as its own, preventing rejection. Tegoprubart is currently in Phase 2 testing for preventing organ rejection in kidney transplant patients.
Clinical Outcomes and Future Prospects
Another patient in the trial has reduced their insulin dose to one-third of the original amount, while a third patient was completely weaned off insulin within two weeks. Researchers noted that the transplant function was three to five times higher compared to patients receiving a different type of immunosuppression, suggesting that tegoprubart may be less toxic to transplanted islets.
Dr. Piotr Witkowski, lead researcher of the trial, stated that these results represent 'another step in our quest to achieve a path for functional cures in type 1 diabetes.' Additional trials are planned to further test the treatment and expand its availability.
Patient Perspective
Ms. Goedel, who was diagnosed with type 1 diabetes at the age of five, described the condition as significantly impacting her childhood and adult life. She experienced frequent hospitalizations due to life-threatening diabetic ketoacidosis and faced challenges in family planning due to blood sugar fluctuations. Now cured, she plans to return to school and pursue activities without the constant worry of a blood sugar crash.
Expert Commentary
Dr. David-Alexandre C Gros, CEO of tegoprubart manufacturer Eledon Pharmaceuticals, explained that this treatment is intended for type 1 diabetics with severely unstable blood sugars, a condition known as brittle diabetes, which affects approximately three in every 1,000 type 1 diabetics. He added that islet transplantation could restore endogenous insulin production, normalize glucose control, and potentially free patients from daily insulin dependence.
Considerations and Costs
While the treatment shows promise, health experts note that scaling up personalized transplants using the recipient's own cells remains challenging and potentially costly. Currently, islet cell transplants are estimated to cost around $100,000. Because islet cell transplants are regulated through the FDA as a biologic drug instead of a transplant, it could prevent people from accessing them outside of a clinical trial.
