Type-1 Diabetes Mellitus is an autoimmune disorder where the immune system mistakenly attacks insulin producing beta cells in the islets of the pancreas. After a significant portion of these islets have been destroyed, the pancreas is no longer able to produce enough insulin for the body. The standard treatment for this disease is daily glucose monitoring and insulin injection. But despite rigorous insulin therapy, many complications eventually arise including organ failure (kidneys, nerves, heart, etc.), amputations, blindness, stroke, and early death. Alternative therapies, including pancreas and islet cell transplants, are not practical due to the number of donors needed (up to 5 donors for a single treatment) and the requirement for lifelong administration of immunosuppressant drugs which are associated with the development of serious infections, cancers, and kidney disease.
SCT has created a technology that has been shown in preclinical studies to functionally cure type-1 diabetes. Studies are also underway to address type-2 diabetes. The SCT therapy will be administered in a simple out-patient procedure resulting in glucose control identical to that of a normal healthy individual. A single treatment is expected to last a lifetime. A clinical trial in diabetic pet dogs is currently underway, and we are aiming to begin a phase 1/2 human clinical trial in 2018.
How The Technology Works
SCT has developed and patented the technology for a fundamentally new adult stem cell-enabled therapy in which culture-expanded pancreatic islet cells are combined with high numbers of Mesenchymal Stem Cells (MSCs) to form unique three-dimensional geometric cellular clusters we call "Neo-Islets". A single dose of Neo-Islets into the abdomen provides the same lifelong benefits as a fresh pancreas, but with the critical benefit of immune isolation.
Diabetes researchers have struggled for many years to create islet encapsulation devices to protect against the immune-attack problem in Type-1 diabetes. These approaches fail because the body rejects the device, the pores are too large to block the immune attack, or the pores are too small and severe fibrosis forms, blood and oxygen cannot reach the islets, or the engineered islets themselves do not produce the proper hormones. SCT invented Neo-Islets as a way to provide "natural encapsulation' rather than using a device.
The technical details about SCT's Neo-Islet technology have been described at length in a recent publication in the journal Stem Cells Translational Medicine. Our Neo-Islets were featured on the cover of the July 2017 issue. The article can be found here. A PDF version of the publication can be downloaded here. The journal cover can be seen here.
SCT HaS OvercomE ALL Technical Obstacles
There are 5 technical challenges that must be overcome in order to provide a viable, permanent cure for diabetes.
IMMUNE ATTACK Challenge
In type-1 diabetes, the immune system mistakenly seeks and destroys the beta islet cells in the pancreas. A viable cure for diabetes must prevent this immune system attack. The SCT approach makes beta islet cells invisible to the immune system so they can perform their normal functions without being attacked.
Donor Scarcity Challenge
Only beta cells can provide the perfectly balanced mix of insulin and related hormones necessary to maintain natural blood sugar levels in the body. Scientists have struggled to find ways to culture and grow these cells in the lab and have been forced to rely on a scarce supply of pancreas donors. Several organ donors are required in order to supply the necessary quantity of beta cells to treat a single person. This inefficiency is unsustainable if a therapy must be provided to hundreds of millions of people. A viable cure for diabetes must provide a way to produce mass quantities of beta cells from a relatively small number of pancreas donors. By using SCT's therapy, up to a thousand people may be treated from a single pancreas donor.
Oxygen is life. Without a constant flow of ATP/Oxygen from the blood stream beta cells will die. The SCT technology employs adult stem cells that protect and repair beta cells and promote angiogenesis (the creation of new blood vessels) to provide a direct supply of oxygen through the blood stream. This enables a single SCT treatment to be durable for a lifetime with no need of re-treatment, oxygen replenishment, or other periodic therapy.
Insulin Delivery Challenge
In a healthy individual, insulin is released by the pancreas directly into the portal system of the liver. This is the most natural, safe and effective means for insulin delivery. The traditional therapy of injecting insulin into muscle tissue or under the skin is far from ideal. It results in poor glucose control and exposes local tissues and organs to potentially harmful levels of insulin. To further complicate matters, proper insulin delivery is highly dependent on effective glucose sensing which cannot be reliably performed from locations in the skin or muscles. The SCT solution solves the insulin delivery problem by monitoring and releasing insulin from the omentum which goes directly into the portal system of the liver in the same way as a healthy pancreas.
A major drawback of today's dominant therapy for diabetes is the stress, pain, and hassle of multi-day monitoring and injection of insulin. A true cure of the disease will not require periodic monitoring of blood sugar or recurring treatments. The SCT therapy is a one-time, simple out-patient procedure that is expected to last a lifetime.