UCSF providing laboratory-produced islet cell therapy as part of clinical trial for Type 1 diabetes patients.
UCSF Health transplant specialists recently performed an innovative, minimally invasive pancreatic islet transplant designed to enable a patient with Type 1 diabetes to become insulin independent.
The patient received insulin-producing islets created in a laboratory and derived from stem cells of an individual with the same blood type. While UCSF Health is internationally known for islet transplantation, the islets have previously come from deceased donor pancreases.
“This is the first time we have done an allogeneic transplant of islets produced in a lab from stem cells of a single human donor,” said Andrew Posselt, MD, a transplant specialist who supervised the transplant and is the principal investigator of the study. “It will take months for the islets to function, but the patient is doing well and there is some preliminary evidence that the islets are already helping with glucose control.”
Islets are made up of 1,000 cells in the pancreas that produce insulin, which helps to regulate blood sugar levels in the body. In Type 1 diabetes (T1D), the body’s immune system destroys these insulin-producing cells, thus requiring people with T1D daily, life-long use of insulin therapy as well as modifications to diet and exercise.
The islet cell therapy in this case, called zimislecel, is produced by Vertex Pharmaceuticals and was sent to UCSF Health as part of a phase 3 clinical trial among select medical centers in the U.S. and abroad. The islet cells were infused into the hepatic portal vein and disbursed in the liver as they establish their own blood supply and begin to make insulin.
In this case, the patient, MJ, is a 62-year-old woman from California with T1D. Despite having T1D since she was 17 years old, she has led an active lifestyle, teaching horseback riding and doing Pilates and yoga for many years. But about 18 months ago, she found her diabetes becoming increasingly challenging to control. While still maintaining her regular combination of insulin, diet, and exercise, MJ said she developed “brittle diabetes” which is characterized by extreme changes in blood sugar levels.
“My blood sugar would drop significantly if I went for a walk and it would take me longer to recover,” said MJ. “Even when I ate, my blood sugar would still crash. I felt like I was on a roller coaster going off the rails.”
No need for invasive surgery
While listening to a podcast about new therapies for T1D, she learned more about Vertex’s islet cell transplant and UCSF Health’s potential to offer this procedure.
“I live in the Central Valley, and I was told by my physician that UCSF was the best for this,” she said. “I reached out to see if I qualified. After all the necessary testing, including heart and liver tests, I had my transplant at UCSF in mid-July.”
The procedure took about 30 minutes, but MJ stayed in the hospital following the procedure for about a week so that she could be monitored for any signs of rejection as well as for rates of insulin use. In addition to check-ups and blood tests in the coming months, she will also continue to take immunosuppression medication to prevent rejection of the islet cells.