One of CIRM’s greatest strengths is supporting projects throughout the development process.
Many promising ideas begin in the laboratory. Few advance to human clinical trials. CIRM helps bridge that gap by supporting research from early discovery through clinical testing.
A recent announcement from ViaCyte highlights the value of that approach.
A New Approach to Type 1 Diabetes
ViaCyte, a company focused on treating Type 1 diabetes (T1D), has received more than $72 million in support from CIRM.
Unlike Type 2 diabetes, which often develops over time, Type 1 diabetes is an autoimmune disease. In people with T1D, the immune system attacks and destroys insulin-producing cells in the pancreas.
Without those cells, the body cannot regulate blood sugar levels. Over time, high blood sugar can damage the heart, kidneys, eyes, and nerves. In severe cases, the disease can become life-threatening.
Current treatments help patients manage their condition. However, they do not replace the lost insulin-producing cells.
ViaCyte aims to change that.
Stem Cells That Respond to Blood Sugar
The company has developed an implantable device designed to replace the cells destroyed by Type 1 diabetes.
Doctors place the small pouch under the skin in a patient’s back. The device contains stem cells that mature over several months into pancreatic islet cells.
These are the same cells that normally produce insulin.
The goal is straightforward. The new cells would monitor glucose levels in the bloodstream. When blood sugar rises, the cells would release insulin to bring those levels back into a healthy range.
If successful, the therapy could provide patients with a more natural way to control blood sugar. It could also reduce the burden of constant monitoring and insulin management.
Supporting Therapies Every Step of the Way
ViaCyte’s progress demonstrates the importance of long-term support for innovative science.
Developing a new therapy requires years of research, testing, and refinement. Moving from a scientific concept to a treatment ready for patients is a difficult journey.
CIRM’s mission is to help promising therapies make that journey.
By supporting projects through multiple stages of development, CIRM helps researchers turn groundbreaking ideas into potential treatments for people living with serious diseases, including Type 1 diabetes.
Clinical trial
They tested this approach in 15 patients in a Phase 1 clinical trial in Canada. Their findings, published in the journals Cell Stem Cell and Cell Reports Medicine, show that six months after implantation, the cells had turned into insulin-producing islet cells. They also showed a rise in C-peptide levels after patients ate a meal. C-peptides are a sign your body is producing insulin, so the rise was a good indication the implanted cells were boosting insulin production.
As Dr. James Shapiro, the Chair of Canada Research and one of the lead authors of the study, says, that’s no small achievement: “The data from these papers represent a significant scientific advance. It is the first reported evidence that differentiated stem cells implanted in patients can generate meal-regulated insulin secretion, offering real hope for the incredible potential of this treatment.”
And that wasn’t all. The researchers say that patients spent 13 percent more time in the target range for blood sugar levels than before the treatment, and some were even able to reduce the amount of insulin they injected.
This is only a Phase 1 clinical trial, so the goal was to test the safety of the pouch, called PEC-Direct (VC-02), to see whether the body would tolerate implantation and whether it is effective. The beauty of this method is that the device is implanted under the skin so it can be removed easily if any problems emerge. So far, none have.
Ultimately, the hope is that this approach will help patients with T1D better regulate their blood sugar levels, improve their health outcomes, and one day even achieve independence from the burden of daily insulin injections.
Stem cells are great advantage for treatment of many diseases. In many adult tissues, stem cells are ease to be isolated, cultures and availability in many tissues. Their immunomodulatory properties and lack of ethical issues giving hope for both autologous and allogenic transplantation. However, stem cell -based therapy may produce undesirable effects. Many cumulative factors can give a response in the form of spontaneous tumor transformation, such as, 1)The ability of stem cells to proliferate for long periods of time, high viability and resistance to apoptosis can easily develop tumorigenesis. 2)Donor’s age, host tissue, growth regulators expressed by recipient tissues and mechanism that control behavior of stem cells at the target site. 3)Manipulation and longterm in vitro cultures of stem cells can cause genetic instability and chromosome aberration
Current clinical trial by using PSC-derived pancreatic progenitors which were fully encapsulated device and implanted into patients with T1D. The observation revealed that implanted device acquired neovasculature and PEC-01 cells matured into insulin-expressing β cells in T1D patients. The results showed that 63% of trial patients had stimulated C-peptide response which was indicator to determine endogenous β cell function. The unequivalent response of T1D patients to implanted PSC-derived cells indicates that pancreatic progenitors require growth factors to growth and differentiation. The different ability of T1D patients produce growth factors and support maturation of progenitors causing diversity of findings. During the process of maturation, stem cells require multiple stages of growth and differentiation to produce mature and functioning cells. Each stage of maturation requires specific growth factor and timing for development. Hence, T1D patients require additional supply of specific growth factor to support progenitors development on certain period of time. As all trial patients are undergoing immunosuppressive treatment, the possibility of patients develop cancers for longterm period of time is unavoidable. Of course, those aberrant cells are unable to be removed by autoimmune response. Thus, extensive follow up is essential to prevent the patients from developing cancers. The short period of observation within 1-2 years does not support the safety and efficacy of implanted PSC-derived cells in T1D patients. The longterm follow up of trial patients are essential to protect the health and well being of T1D patients.