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.
“It could take months for her to be insulin independent, but her blood sugar function is already happening, and we are cautiously optimistic about the treatment,” said Posselt, who is also the co-director of UCSF’s Pancreas Islet Transplant Program. “This type of transplant is important because it allows near complete physiologic treatment of diabetes without need for invasive surgery (such as pancreas transplant) or waiting for a suitable organ as is required for pancreas or deceased donor islet transplants.”
Posselt adds that the lab-produced islet cells came from the human donor with blood type A (matching MJ’s blood type), but he hopes there will soon be islet cells derived from a blood type O donor, so the therapy can be used for any patient with T1D and is eligible for the transplant.
In the meantime, MJ is looking forward to seeing the results of her weekly blood tests, which are already showing improved glucose control. She is also thankful for the support she’s received during her transplant journey.
“I have had great support at UCSF and the staff there is A1,” said MJ. “I couldn’t be more grateful to them, to my wife, to my friends and family, who are supporting me every day. And I am hoping that one day soon, I won’t have to worry about my diabetes anymore.”
About UCSF Health: UCSF Health is recognized worldwide for its innovative patient care, reflecting the latest medical knowledge, advanced technologies and pioneering research. It includes the flagship UCSF Medical Center, which is a top-ranked specialty hospital, as well as UCSF Benioff Children’s Hospitals, with campuses in San Francisco and Oakland; two community hospitals, UCSF Health St. Mary's and UCSF Health Saint Francis; Langley Porter Psychiatric Hospital and Clinics; UCSF Benioff Children’s Physicians; and the UCSF Faculty Practice. These hospitals serve as the academic medical center of the University of California, San Francisco, which is world-renowned for its graduate-level health sciences education and biomedical research. UCSF Health has affiliations with hospitals and health organizations throughout the Bay Area. Visit http://www.ucsfhealth.org/. Follow UCSF Health on Facebook, Threads or LinkedIn.
