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New Encapsulated Beta-Cell Replacement Therapy for Type 1 Diabetes

Beta cell encapsulation therapy is a procedure that involves implantation of cells with the ability to secrete insulin in a glucose-responsive manner in a protective barrier into the body….

On July 17th 2014, the Juvenile Diabetes Research Foundation (JDRF) announced that their partner, ViaCyte, Inc. filed an Investigational New Drug (IND) application with the US. Food and Drug Administration (FDA) seeking to conduct a Phase 1 and 2 clinical trial in patients with type 1 diabetes. The purpose of this trial is to evaluate the safety and efficacy of the VC-01 product, a stem cell-derived, encapsulated cell replacement therapy. In addition to the IND, ViaCyte also submitted a Medical Device Master File (MAF) to the FDA regarding the Encaptra® drug delivery system, a device component of the VC-01 product.

Beta cell encapsulation therapy is a procedure that involves implantation of cells with the ability to secrete insulin in a glucose-responsive manner in a protective barrier into the body. The advantage of these encapsulated beta cells is that they can assess the patient’s glucose level in the blood and secrete the correct amount of insulin, while their barrier can protect them from being destroyed by the autoimmune system. More importantly, encapsulation therapy also helps prevent lifetime administration of powerful and toxic immunosuppressive drugs designed to protect the newly introduce islets from the immune system.

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) and an Encaptra drug delivery system (a proprietary immune-protecting and retrievable encapsulation medical device.) The VC-01 combination product is expected to be implanted under the skin of the patient through a simple outpatient surgical procedure. Once inside the body, the cells are expected to differentiate and become mature pancreatic cells with the ability to produce and secret insulin based on the patient’s glucose level.

Based on preclinical studies, the VC-01 therapy has shown to be affective in mice. Normal blood glucose levels for mice are ranging from 160-200mg/dL, which are considered as hyperglycemic in humans. However, when the mice received the VC-01 combination product, their blood glucose levels were closer to human levels. In addition, when these mice received STZ, a chemical that designed to kill native mouse beta cells, the mice still maintained their blood glucose levels.

Study of the synergy of cell therapy and Encaptra medical device also showed positive results. In the mouse study, host blood vessels began to grow into the VC-01 combination product at week 4, supplying a steady amount of oxygen and nutrients to PEC-01 cells. At week 8, vascularization developed quickly. Meanwhile, Encapra device protected the PEC-01 cells from immune rejection with a permeable protective membrane.

Practice Pearls:
  • ViaCyte, Inc. has file an Investigational New Drug (IND) application with the US. Food and Drug Administration (FDA) seeking to conduct a Phase 1 and 2 clinical trial in patients with type 1 Diabetes.
  • VC-01 product, a stem cell-derived, encapsulated cell replacement therapy could be a potential cure for type 1 Diabetes.
  • The advantage of encapsulated beta cells is that they can assess the patient’s glucose level in the blood and secret the correct amount of insulin, while their barrier can protect them from being destroyed by the autoimmune system.

JDRF Partner ViaCyte Announces Key Project Milestone to Advance Innocative Encapsulated Cell Replacement Therapy product for Type 1 Diabetes. JDRF. 3 Aug. 2014. Web. 17 Jul. 2014. VC-01TM Combination Product – Improving Diabetes Treatment. ViaCyte: Regenerating Health. 3 Aug. 2014. Web. n.d.