New hope is on the horizon for people with type 1 diabetes (T1D). For the first time, a person with T1D was implanted with an experimental treatment that may replace damaged insulin-producing cells in the pancreas.
According to JDRF, the study of ViaCyte’s experimental therapy, VC-01, is initially being conducted at the University of California at San Diego (UCSD). The experimental product encapsulates immature human islets derived from a stem cell source in an immune-protective device called the Encaptra drug delivery system.
At first, a small group of people in the study will be followed for several months to ensure the safety of the therapy. After the initial evaluation of this first group of participants at the UCSD site, it is expected that additional sites will be activated to enroll more people in early 2015.
“JDRF is excited about the historic research advance of this encapsulated cell replacement therapy for type 1 diabetes,” said Derek Rapp, JDRF president and CEO. “Encapsulated cell therapies have the potential to fundamentally transform the lives of people with type 1 diabetes by restoring a person’s independence from insulin injections and reducing concerns of the complications that arise from living with the disease."
"We are enthusiastic about the progress of research in this field," added Mr. Rapp. "We look forward to the full enrollment in the trial and to results from this study which could move us one step closer to a new and effective treatment for type 1 diabetes. This day would not have been possible without the basic stem cell research and the insights learned to protect insulin-producing cells from the autoimmune attack that JDRF has been funding for years.”
Commenting on the study, DiabeticLifestyle Editorial Board Member, Grazia Aleppo, MD, noted that the clinical trial opens the horizon to a new minimally invasive method of beta cell replacement.
The primary goal of this first study is to evaluate the safety of the VC-01 product candidate in people who have had T1D for at least 3 years; not to make them insulin independent. However, the study is also designed to evaluate the effectiveness of the VC-01 product candidate in replacing the lost insulin-production function that is central to T1D, by measuring C-peptide levels as a marker of insulin production.
This study will provide researchers with critical information about the functioning of the encapsulation device at the implantation site and about the maturation and survival of the implanted cells, noted a press release from JDRF.
Dr. Paul Laikind, President and CEO of ViaCyte, said, “Treating the first patient with our stem cell-derived islet replacement product candidate is an exciting next step in our quest to transform the way patients with type 1 diabetes are impacted by the disease. Moving from a promising idea to a new medicine is a long and challenging journey and we are grateful to JDRF, and all its supporters, for the tremendous and continued support they have provided. At ViaCyte we have been working over a decade to reach this milestone, and JDRF has been there every step of the way, providing critical funding, excellent advice, and advocating on our behalf.”
People in the study will go about their daily lives with the implanted product for up to 2 years. They will be closely monitored by clinicians during this time. The encapsulated cell therapy here involves putting cells with the potential to mature and produce insulin in a protective barrier and implanting them in the body using a minimally invasive procedure.
Once matured these encapsulated immature human islets might sense a person’s glucose levels and produce insulin while the barrier shields them from the body’s T1D autoimmune attack as well as foreign graft rejection. If they perform as they have in animal studies, the cells will continuously assess the amount of glucose in the blood and release the appropriate amount of insulin.
Importantly, encapsulated cell replacement therapies have the potential to overcome the major obstacles that have limited the use of human islet transplantation in people with T1D: limited donated islets and the need for lifelong administration of immunosuppressive drugs to prevent destruction of the newly introduced islets.
"Although I am always optimistic about new therapies, it is imperative to gather more understanding regarding the safety of this type of therapy, the durability of the encapsulated cells/ drug delivery system, and the response of the immune system overtime towards these encapsulated beta cells," said Dr. Aleppo, Associate Professor in Medicine-Endocrinology, Northwestern University, Chicago.
"I feel that this study will provide substantial information when the data will become available regarding this new therapy and I look forward to seeing the progress and eventually the results," noted Dr. Aleppo.
Stem cell-derived islet sources may represent an unlimited supply of replacement insulin-producing cells and the protective devices eliminate the need for immunosuppressive drugs, noted the JDRF press release.