VC-01™ Combination Product – Improving Diabetes Treatment

ViaCyte has integrated two of its novel technologies, PEC-01™ cells and the Encaptra® drug delivery system, into one therapy called VC-01™ combination product.

We focus on combining state-of-the-art cell process technology with advanced medical device engineering.
Michael Scott, Ph.D., VP, Device R&D and Chief Development Officer, ViaCyte

What is the VC-01 Combination Product?

By acting essentially as a replacement endocrine pancreas, the source of insulin and other regulatory hormones produced in our bodies, ViaCyte’s VC-01 combination product has the potential to be a virtual cure for type 1 diabetes. 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.


How the VC-01 Combination Product Works

Click on video to learn more about diabetes
and ViaCyte’s cell therapy product

The VC-01 combination product is expected to be implanted under the skin of the patient through a simple outpatient surgical procedure. The cells are then expected to further differentiate to produce mature pancreatic cells that will synthesize and secrete insulin and other factors, thereby regulating blood glucose, commonly referred to as blood sugar, levels.

The VC-01 Therapy is Effective in Preclinical Disease Models

The efficacy of the VC-01 combination product has been demonstrated in dozens of preclinical studies and is illustrated in the following graph from a study performed in mice in accordance with Good Laboratory Practices (GLP). In this model, the VC-01 combination product or empty Encaptra drug delivery devices are implanted (the graph shows results from female mice; male mice demonstrate similar results).

Normal blood glucose levels for the mouse are typically 160 to 200 mg/dL, a level that would be considered hyperglycemic in humans. Yet, as expected, animals that received the VC-01 combination product containing human PEC-01 cells have blood glucose closer to human levels because it is being regulated by the grafted human cells (VC-01 group).

Further proof of the efficacy of the VC-01 therapy is seen when animals are treated at 33 weeks (arrow) after implantation with STZ, a chemical that selectively kills native mouse beta cells. As expected, the animals implanted with devices loaded with PEC-01 cells (VC-01 combination product) maintain their blood glucose levels, while animals given empty devices without PEC-01 cells (Empty Device) experience severe hyperglycemia.

The VC-01 Therapy Outlook

The expected attributes of the VC-01 combination product include:

  • Long-term control of blood glucose levels with a single minimally-invasive outpatient implant
  • Insulin independence – blood glucose tightly regulated in the normal range, eliminating the need for insulin injections
  • A significant reduction in serious chronic health conditions caused by swings in blood glucose
  • Significantly reduced risk of hypoglycemia
  • Physiologic production of other pancreatic derived regulatory hormones and co-factors which have the potential to produce other health benefits
  • Ability to terminate treatment by minimally-invasive, out-patient removal of the encapsulated product

The Synergy of Cell Therapy and Medical Device

Like beta cells in the native pancreas, the glucose-sensing insulin-producing cells that develop from PEC-01 cells require a steady supply of oxygen and other nutrients to produce enough insulin to control blood glucose. PEC-01 cells are effectively a tissue transplant, which without protection would be rejected by the patient.

In this mouse study, host blood vessels have begun growing into the VC-01 combination product at 4 weeks.

At 8 weeks, vascularization is developing rapidly.
The Encaptra device is designed to prevent immune rejection by surrounding PEC-01 cells with a permeable, protective membrane.

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.

Preclinical Data are Promising

To date, over a thousand VC-01 pre-clinical study implants have matured into insulin-producing grafts. The VC-01 therapy has consistently been capable of controlling blood glucose in mice at a human set point.

 

After 16 weeks, PEC-01 cells have developed into islet-like structures.

 

The mature cells in the VC-01 combination product produce insulin, glucagon and somatostatin.

What’s Next?

ViaCyte has performed all of the preclinical testing required to assure reliable production of a safe and effective product.  In August 2014 ViaCyte received approval from the U.S. Food and Drug Administration (FDA) to begin evaluation in human clinical trials.  See Clinical Trials.