Orgenesis Inc (NASDAQ: ORGS), a developer of advanced cell therapies, announced Tuesday that it has received Institutional Review Board (IRB) approval to collect liver biopsies from patients at the Rambam Medical Center in Haifa, Israel.
This is intended to move forward a study to confirm the suitability of liver cells for cell replacement therapy for patients with insulin-dependent diabetes as a result of pancreatectomy, where a portion of the pancreas is surgically removed to treat serious conditions. Unfortunately, diabetes after pancreatectomy results in lifelong insulin-dependence.
The company said the collected liver cells are intended to be bio-banked for potential future clinical use.
“We believe that this IRB approval is a significant milestone in our product development process as we prepare to commence a key study, which is intended to confirm the suitability of patients’ liver cells to be used as a personalized cell replacement therapy,” said Dr Efrat Assa-Kunik, general manager at Orgenesis Limited.
Study will kick off in May
During the study, liver samples will be collected and then processed and stored in specialized, clinical grade tissue banks for potential clinical use. The study will enroll 20 patients and start next month.
The propagated cells will be maintained in a tissue bank and be utilized in a future clinical study, in which the cells will be transdifferentiated and administered back to the patients as a potential treatment.
This personalized autologous process will be performed under the company’s Autologous Point of Care (POCare) model, said the company, in which the patient liver samples are processed, cryopreserved and potentially re-injected, all in the medical center, under clinical grade/GMP level conditions.
Transdifferentiation platform technology
Orgenesis is a pioneer in transdifferentiation, which involves direct reprogramming of an adult tissue or cell into another type of specialized cell with its distinct function. Transdifferentiation potentially provides the use of a patient's own adult tissues to restore any damaged organs or tissue.
Orgenesis has further developed the science by licensing technology from the Tel Hashomer Medical Research Infrastructure and Services, utilizing liver cells as a source for Autologous Insulin Producing Cells as replacement therapy for islet transplantation. Theses insulin producing cells are derived from liver cells that are taken from the patient liver to be treated and then converted into functional glucose-regulated induced pluripotent stem cells (IPCs) and returned to the patient via injection. The therapy has the potential to provide long-term treatment.
Orgenesis founder and chief scientific officer Professor Sarah Ferber is the inventor of the company’s groundbreaking transdifferentiation platform technology.
“We believe that this key study, our first human trial, will allow us to better understand the autologous immune response following AIP cell implantation in different patient populations (autoimmune or not), ex-vivo and in-vivo, as well as the potency of transdifferentiation as a novel precision medicine platform to predict treatment success,” said Professor Ferber.
Converting a diabetic’s own tissue into insulin-producing cells has the potential to provide a cure for insulin dependence and overcoming donor shortages, costs and risks involved with transplant rejection.
Professor Shehadeh from the Rambam Medical Center is the study’s principal investigator, and says the “breakthrough technology” could provide patients with a potential cure that could “dramatically improve” their quality of life.
“Based on the outcome of this study, our goal is to utilize these cells in a future clinical phase I/IIa study, in which the cryopreserved cells would be administered back to the patient,” said Dr Efrat Assa-Kunik. “If successful, we believe that both of these studies could represent major milestones for the company and, most importantly, could be transformative for patients worldwide.”
Contact Uttara Choudhury at [email protected]tors.com