Principal Investigator
Dr. Massimiliano Paganelli
CHU Sainte-Justine
Project of $727,507 over 2 years
- Supported by CQDM through:
Ministère de l’Économie, de l’Innovation et de l’Énergie du Québec (MEIE) - And by co-funding partner:
– Morphocell Technologies
– Stem Cell Network
The project in details
Children with urea cycle disorders (UCD) face life-threatening crises caused by their bodies’ inability to eliminate ammonia. These crises can lead to severe brain damage or death if untreated. Current treatments are insufficient, and liver transplantation, the only curative option, becomes often available only once irreversible neurological damage is already done. Gene therapy is being developed and holds promise for a cure but cannot yet address early-life crises. There is a critical need for a fast and effective treatment for these crises that might bridge newborns and young children safely to curative options like gene therapy or liver transplantation. Our project aims to address this gap by repurposing an innovative regenerative medicine product called Encapsulated Liver Tissue (ELT). The ELT, derived from stem cells, mimics liver function and can rapidly reduce ammonia levels without requiring immunosuppression. This product has already shown success in treating liver failure in preclinical studies and is on track for clinical trials in 2027. In this study, we will evaluate the ELT’s effectiveness in managing UCD-related hyperammonemia. First, we will test its ammonia-clearing capacity in the lab, followed by trials in animal models of UCD to assess its safety and ability to prevent brain damage. If successful, this project could bring a transformative treatment to children with UCD, providing them a chance to survive and safely reach curative therapies.
