The Encapsulated Liver Tissue as a treatment for acute liver failure: proof-of-concept efficacy and safety in large animals using liver organoids composed of primary cells

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Challenge: Acute Liver Failure (ALF) is an extremely severe, progressive syndrome resulting from a sudden insult on the liver that exceeds the organ’s innate regenerative capacity. No effective treatment exists for ALF except for liver transplant, which is limited by organ shortage, heavy morbidity and need of lifelong immunosuppression treatments. Temporary replacement of liver functions while the patient’s own liver physiologically regenerates might eliminate the need for transplant for up to 80% of ALF patients. Therefore, there is an urgent need for effective liver support systems able to replace liver functions for the time needed for the liver to regenerate.

Solution : The team previously designed a method to generate and preserve liver tissue in vitro and developed the Encapsulated Liver Tissue (ELT), an implantable product derived from human induced pluripotent stem cells. The ELT proved to perform mature liver functions in vitro and to treat the ALF in rodent models by significantly improving their survival, treating the hepatic encephalopathy and accelerating the liver regeneration without the need for immunosuppression. To reach clinical trials, a validation of these features in large animals is required. Within this project, the team aims at manufacturing a full-scale ELT, assessing the safety and efficacy profile in large animals, and determining the optimal dose for the clinical trials.

Expected achievements/Impact: Collaborative research will advance the Montreal-based company Morphocell Technologies’ unique and first-in-class treatment for liver disease. The cell therapy product ELT will indeed be well positioned for clinical trials at the end of the project after achieving at-scale manufacturing development and proof-of-concept safety and efficacy. Its unique features and cell delivery approach make the ELT a promising allogeneic cell therapy that, upon successful clinical confirmation, might potentially replace liver transplantation for many patients with ALF. ELT has the potential to save thousands of patients with ALF and, in the future, hundreds of thousands with chronic liver failure worldwide, by drastically improving their lives without the need of immunosuppression.

Principal Investigator:
Massimiliano Paganelli
Morphocell Technologies
Ongoing Project
$979,200 / 1 year
Supported by CQDM through:
– Takeda
And by co-funding partners
– Morphocell Technologies
– Stem Cell Network
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