Clinical Trial Tests Type 1 Diabetes Stem Cell Derived Therapy
Clinical Trial Tests Type 1 Diabetes Stem Cell Derived Therapy – Researchers at the University of California, San Diego School of Medicine, in partnership with ViaCyte, Inc., a San Diego-based biotechnology firm specializing in regenerative medicine, have launched the first-ever human Phase I/II clinical trial of a stem cell-derived therapy for patients with Type 1 diabetes.
The trial will assess the safety and efficacy of a new investigational drug called VC-01, which was recently approved for testing by the U.S. Food and Drug Administration. The 2-year trial will involve four to six testing sites, the first being at UC San Diego, and will recruit approximately 40 study participants.
“The goal, first and foremost, of this unprecedented human trial is to evaluate the safety, tolerability and efficacy of various doses of VC-01 among patients with type 1 diabetes mellitus,” said principal investigator Robert R. Henry, MD, professor of medicine in the Division of Endocrinology and Metabolism at UC San Diego and chief of the Section of Endocrinology, Metabolism & Diabetes at the Veterans Affairs San Diego Healthcare System.
“We will be implanting specially encapsulated stem cell-derived cells under the skin of patients where it’s believed they will mature into pancreatic beta cells able to produce a continuous supply of needed insulin. Previous tests in animals showed promising results. We now need to determine that this approach is safe in people.”
Type 1 diabetes mellitus is a life-threatening chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow glucose to enter cells to produce energy. It is typically diagnosed during childhood or adolescence, though it can also begin in adults. Though far less common than Type 2 diabetes, which occurs when the body becomes resistant to insulin, Type 1 may affect up to 3 million Americans, according to the JDRF. Among Americans age 20 and younger, prevalence rose 23 percent between 2000 and 2009 and continues to rise. Currently, there is no cure. Standard treatment involves daily injections of insulin and rigorous management of diet and lifestyle.
Phase I/II clinical trials are designed to assess basic safety and efficacy of therapies never before tested in humans, uncovering unforeseen risks or complications. Unpredictable outcomes are possible. Such testing is essential to ensure that the new therapy is developed responsibly with appropriate management of risks that all medical treatments may present.
“This is not yet a cure for diabetes,” said Henry. “The hope, nonetheless, is that this approach will ultimately transform the way individuals with Type 1 diabetes manage their disease by providing an alternative source of insulin-producing cells, potentially freeing them from daily insulin injections or external pumps.”
The VC-01 therapy is the combination of:
- PEC-01 cells: A proprietary pancreatic endoderm cell product derived through directed differentiation of an inexhaustible human embryonic stem cell line, and
- Encaptra drug delivery system: A proprietary immune-protecting and retrievable encapsulation medical device.
According to Viacyte, “The unique combination of these cells with this device results in rapid and extensive growth of blood vessels around the device, providing a plentiful oxygen source and rapid distribution of insulin to the body. Just weeks after implantation, the host responds to chemical signals from the VC-01 combination product by developing an extensive network of blood vessels.”
Their goal is to develop “a transformative cell replacement therapy to free people with insulin-requiring diabetes from painful and frequent insulin injections, strict diet regimens, constant blood sugar monitoring, and serious chronic health conditions associated with the disease.”
This treatment has potential. It is still years away from approval, but if it becomes a viable treatment, it appears to be minimally invasive and does not require the use of long term immunosuppressive drugs.
LCT’s Diabcell is similar to ViaCyte’s VC-01, in that they are both encapsulated beta cell devices. They do use different encapsulation coatings, and Diabcell uses pig beta cells, while VC-01 uses beta cells grown from human embryonic stem cells. LCT has been tested in people for over 6 years, and is currently in phase-II trials. (At one time it had approval to be sold in Russia, but it never was sold there.) There is also a device being tested at the University Clinical Hospital Saint-Luc in Belgium, which uses human beta cells (from cadavers) and a different encapsulation coating.
Several organizations are doing animal tests on various encapsulated beta cell devices. These include Cerco Medical, Beta-O2, DRI, and several more.
Finally, several organizations are doing human tests on beta cell devices which are not (yet) encapsulated, but they hope to encapsulate in the future. DRI is doing work like this, as is Serova. If beta cells are not encapsulated, then you must take immunosuppressives for the rest of your life.
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University of California, San Diego Health Sciences. “Clinical trial to test safety of stem cell-derived therapy for type 1 diabetes.” ScienceDaily. ScienceDaily, 10 September 2014.