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Friday, November 13 2015

Fighting liver diseases in children

A 2015 Gilead Sciences Award goes to Massimiliano Paganelli

MONTREAL, November 13, 2015 – Children born with liver metabolism diseases face severe difficulties and even death, as treatment is rarely possible. Dr. Massimiliano Paganelli, a professor at the University of Montreal and a clinician and researcher at the affiliated mother-child hospital CHU Sainte-Justine, got a boost today in his effort to develop cell therapy for these conditions, as he received the 2015 Gilead Sciences International Research Scholars Program in Liver Disease Award – The Americas. This is the first time this prestigious award, which is endowed with a major two-year financing, is conferred to a non-American institution.

Inborn errors of liver metabolism are caused by a single gene mutation entailing multiple severe and often lethal consequences, both in and outside the liver. “The quality of life of people with tyrosinemia, the specific disease on which my team and I will be working, is often very poor,” Dr. Paganelli explained. “My study aims at assessing the feasibility of treating them with autologous cell therapy, which basically consists in transplanting healthy liver cells.” Such a therapy could cure the patients while freeing them from the need for liver transplantation – often associated with a number of long-term complications – lifelong treatments and compelling diets. More than 80% of patients with a disease like tyrosinemia, if left untreated, die before 2 years of age.

Specifically, Paganelli’s project aims at generating stem cells from the patients’ blood. Using genome editing, the causative mutation is to be removed from the stem-cells. Edited stem cells will then be used to generate healthy liver cells, which will in turn be transplanted back to the patient in order to cure their liver disease. “Because the patients receive their own cells, their body won’t reject them. This eliminates the need for lifelong immunosuppressive treatment,” Dr. Paganelli explained. At first, Paganelli’s team is going to test such an approach on murine models. Once the safety and effectiveness of the procedure are established, they will move to a clinical trial. Such an approach could be used for several other liver diseases. Proven successful, it has the potential to completely change the life of children and adults with inborn errors of liver metabolism.

Gilead Sciences’ mission is to advance the care of patients suffering from life-threatening diseases. Through its research scholars program, the company wants to support innovative scientific research that will advance knowledge and understanding in the field of liver disease.

About the CHU Sainte-Justine Research Center

CHU Sainte-Justine Research Center is a leading mother-child research institution affiliated with Université de Montréal. It brings together more than 200 research investigators, including over 90 clinician-scientists, as well as 360 graduate and postgraduate students focused on finding innovative prevention means, faster and less invasive treatments, as well as personalized approaches to medicine. The Center is part of CHU Sainte-Justine, which is the largest mother-child center in Canada and second most important pediatric center in North America. More on research.chusj.org

CHU Sainte-Justine

Communications, CHU Sainte-Justine Research Center

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About the researcher

Dr. Massimiliano Paganelli is a pediatric gastroenterologist-hepatologist at CHU Sainte-Justine, a researcher at the CHU Sainte-Justine Research Centre, and an Assistant Professor at the University of Montreal’s Department of Pediatrics. The University of Montreal is officially known as Université de Montréal.

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Updated on 11/13/2015
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