Regenerative medicine company ViaCyte said today that the first patients have been implanted in a trial of its islet cell replacement therapy, PEC-Direct.
San Diego-based ViaCyte is developing the cell therapy as a functional cure for patients with Type I diabetes who are at risk of suffering acute life-threatening complications.
The company’s open-label clinical trial is slated to assess the safety and efficacy of its PEC-Direct product.
The first group of patients with Type I diabetes will receive multiple cell-filled devices, called sentinels, to establish safety and implant viability, according to ViaCyte. After an undisclosed amount of time, the sentinel units will be removed and examined, in the hopes of better understanding engraftment and maturation. The researchers plan to evaluate if the cells develop into insulin-producing beta cells, the company said.
Then, ViaCyte plans to follow a second, 40-patient group and measure the PEC-Direct therapy’s ability to trigger insulin production, as measured by an insulin biomarker.
“Islet transplants have been used to successfully treat patients with unstable, high-risk Type I diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations,” trial investigator Dr. James Shapiro said in prepared remarks. “An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people.”
“Patients with high-risk Type I diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose,” trial investigator Dr. Jeremy Pettus added. “The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need.”
ViaCyte said it hopes to have early reports of efficacy in the first half of next year.
“JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy,” JDRF president & CEO Derek Rapp said. “JDRF is excited to support this clinical development given its potential to help those people with Type I diabetes that need it the most – those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes.”
“There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes,” ViaCyte president & CEO Paul Laikind added. “We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress.”