PharmaVOICE talked with Dr. Paul Laikind, ViaCyte’s President and CEO, about how the company is developing a unique stem cell-derived therapy for type 1 diabetes. Type 1 diabetes affects an estimated 1.25 million people in the US, many of whom suffer from severe complications including kidney failure, heart disease, and stroke.
Type 1 diabetes occurs when the body attacks and destroys the insulin-producing beta cells in the pancreas. These cells produce insulin in response to high levels of glucose (sugar) in the bloodstream. Improperly regulated blood glucose can cause loss of consciousness, coma, and even death. Replacing insulin through injections helps people with type 1 diabetes manage their blood glucose levels.
ViaCyte is developing PEC-EnCap (also known as VC-01), a first-in-class product candidate designed to provide replacement pancreatic islet cells, including the insulin-producing beta cells. ViaCyte’s product candidate comprises pancreatic precursor cells made from human stem cells and a semi-permeable pouch that protects these cells from the body’s immune system. After the pancreatic precursor cells in the pouch are implanted under the skin, they are designed to mature into functioning pancreatic islet cell types that produce insulin and other blood-glucose regulating hormones. PEC-EnCap is currently being tested in a Phase 1/2 clinical trial, and preliminary results have been promising.
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For more than a decade, ViaCyte and BetaLogics have been independently working toward a stem cell-derived therapy for diabetes. In February of 2016, ViaCyte and Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, agreed to consolidate the assets of the Janssen BetaLogics group into ViaCyte.
As Paul Laikind, Ph.D., ViaCyte President and CEO, explained to FierceBiotech, “By combining the intellectual property and other assets of BetaLogics with ViaCyte, we will further strengthen our advanced program focused on insulin-dependent diabetes and solidify our leadership in the field.”
The acquisition occurred as ViaCyte reported positive preliminary results in the first clinical trial of a stem cell-derived therapy in patients with type 1 diabetes. Diego Miralles, global head of innovation at Johnson & Johnson San Diego, told FierceBiotech that these results showed “it’s clear that ViaCyte has pulled ahead (of BetaLogics).”
ViaCyte reported positive preliminary results from the Company’s Phase 1/2 clinical trial of PEC-EnCap (also known as VC-01) product candidate. The company is the first to advance an encapsulated islet cell replacement therapy for type 1 diabetes into clinical trials.
NBC interviewed ViaCyte’s President and CEO, Dr. Paul Laikind, who explained that “ViaCyte hopes to turn (type 1 diabetes) into a disease that patients don’t have to think about.”
To achieve this goal, ViaCyte has differentiated pancreatic precursor cells from a human stem cell line. ViaCyte has also developed an encapsulation device to protect the cells from the immune system once implanted into a patient.
Dr. Laikind explains, “These cells are human cells, but they’re not the patient’s cells, so the patient’s immune system would want to remove those cells – attack those cells.”
The encapsulation device is also semi-permeable allowing oxygen and nutrients to flow to the implanted cells, and the diffusion of blood-glucose regulating hormones produced by the cells into the patient’s blood.
Once implanted, the cells are designed to differentiate into the various cell types that make up the pancreatic islet, which could allow for production of insulin and other hormones, such as glucagon, needed to control blood sugar levels.
While discussing preliminary clinical trial results, Dr. Laikind told Xconomy that “Three months after implantation, the cells are surviving, they are proliferating…the device is vascularized, and they are differentiating.”
Xconomy also interviewed Derek Rapp, president and CEO of JDRF, who said that these results “move us closer to our goal of a world without type 1 diabetes.”
Randy Mills, president of the California Institute for Regenerative Medicine told California Stem Cell Report that “the latest clinical data from ViaCyte are very encouraging and a clear sign of progress.”
Lisa Hepner, a filmmaker living with type 1 diabetes, and her husband Guy Mossman are producing a feature-length documentary on ViaCyte’s efforts to develop a better treatment for diabetes.
The documentary, called The Human Trial, follows the company and collaborators as ViaCyte is advancing the first stem cell-derived islet cell replacement therapy for type 1 diabetes through clinical trials.
Paul Laikind, Ph.D., ViaCyte’s President and CEO, told TuDiabetes that ViaCyte’s product candidate is designed to “do what the body has lost the ability to do, which is produce insulin naturally from the beta cells…and replace the other cells in the endocrine system that also help to control blood glucose.”
A Sweet Life notes that this “is the first clinical trial of its kind and The Human Trial is the first film to take us backstage into the world of cure research.”
Hepner told A Sweet Life that she wants to show “the Herculean efforts behind most cure research.” She also wants “people with diabetes, particularly type 1 diabetes who are waiting for a cure, not to give up.”