Liver disease

Liver disease

Liver disease

Liver Disease

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Chronic liver disease affects one in four Canadians, with an estimated 10 million Canadians impacted by metabolic dysfunction-associated steatotic liver disease (MASLD, previously known as non-alcoholic fatty liver disease or NAFLD. There are over 100 different types of liver disease caused by a variety of factors such as viruses, toxins, genetics, alcohol and other unknown causes. MASLD is caused by the accumulation of fat in liver cells, primarily a result of obesity, insulin resistance, high blood sugar or high levels of fats in the blood. As fat accumulates in the liver it becomes inflamed and scarred. If left untreated, this scarring becomes cirrhosis, late-stage liver damage, and can lead to liver cancer, liver failure, and other complications.

Liver cancer, once considered rare, has become one of the fastest-growing cancers in Canada and is the fifth-leading cause of cancer-related deaths among men. For the hundreds of Canadians waiting for a liver transplant, many never receive it. In 2024, there were 655 liver transplants in Canada, with 609 people still waiting for a transplant as of December 31, 2024, and 89 deaths occurring while waiting for a liver transplant.

Stem Cell Network researchers are working to lessen the impact of liver disease on Canadians, developing novel regenerative medicine approaches with the goal of extending patients’ lives. Current research focuses on innovative therapies such as Encapsulated Liver Tissue (ELT)—a stem cell-based product that mimics liver function and could serve as a life-saving bridge for children with urea cycle disorders and other patients in urgent need. From 2016-2028, SCN has invested over $8.7 million in liver disease research.

Dr. Massimiliano Paganelli, Centre hospitalier universitaire Sainte-Justine, Quebec

Cycle 5, Round 1: Fueling Biotechnology Partnership Award (SCN-CQDM Joint-Funding Award) Stem Cell Liver Patches Offer Hope for Children with Rare Metabolic Disease

Children born with urea cycle disorders (UCD) cannot safely remove ammonia from their blood—a toxic substance that, if it builds up, can quickly lead to brain damage or death.

While liver transplants or future gene therapies may offer a long-term cure, these options often come too late, after a child has already suffered permanent harm. That’s why a fast and effective emergency treatment is urgently needed.

Dr. Massimiliano Paganelli and his team are working to address this gap by repurposing an innovative regenerative medicine product called Encapsulated Liver Tissue (ELT)—a stem cell–derived product that mimics liver function and rapidly reduces dangerous ammonia levels. Unlike transplants, ELT doesn’t require immune-suppressing drugs. The therapy has already shown success in treating liver failure in preclinical models and will soon enter clinical trials.

In this project, Dr. Paganelli will test whether ELT can safely lower ammonia in children with UCD, both in the lab and in animal models. If successful, it could serve as a life-saving bridge to more permanent therapies, giving vulnerable infants and young children a fighting chance.

“Our goal is to protect the brains of children with UCD, buying precious time until a cure can be safely delivered.”