MONTREAL, March 3, 2021 – The Research Program in Cell Therapy and Genetic Modification of Cells and Tissues for the Treatment of Rare Diseases and Pediatric Cancers, led by Dr. Elie Haddad at CHU Sainte-Justine, has been awarded $6.8 million as part of the 2020 competition of the Canada Foundation for Innovation (CFI) Innovation Fund.

This funding confirms the global leadership of CHU Sainte-Justine's researchers in cell, tissue and gene therapies.

Rare diseases

There are more than 7,000 rare diseases, most of which are genetic diseases or cancers affecting children. Despite the fact that for over 50% of these conditions the causative mechanism is now understood, only around 200 treatments have been approved, making rare diseases a heavy burden for the healthcare and society in general.

Therapies based on the modification and engineering of cells, genes and tissues are currently the main drivers of innovation in the treatment of genetic diseases and in cancer therapies.

"The initial results of these new therapies are promising, but their efficacy, feasibility and safety need to be improved before they can be used in a clinical setting," says Dr. Elie Haddad, clinician-researcher in immunology at CHU Sainte-Justine and professor at Université de Montréal.

Translational research platform:

Innovative program in genetic, cellular and tissue engineering for the treatment of pediatric monogenic diseases and cancers

With its clinical and research expertise, CHU Sainte-Justine is a leader in meeting the challenges of designing and improving innovative therapeutic strategies.

"Our goal is to create a translational research platform to significantly accelerate the development of innovative treatments, from new discoveries to the clinic," says Professor Alexey V. Pshezhetsky, researcher at CHU Sainte-Justine and professor at Université de Montréal, co-PI of the project and a leader of the gene-stem cells therapy program.

To achieve this goal, the team is using cutting-edge technologies in cellular and molecular biology. The deployment of new infrastructures, made possible with CFI's support, will increase performance and could lead to breakthroughs in the development of new treatments.

"The program has the potential to provide a new generation of effective and sustainable therapies for pediatric diseases that are accessible to Canadians," adds Professor Pshezhetsky.

Multidisciplinary research

"Our team will use cutting-edge technologies in the areas of gene modification, stem or primary cell transduction, cell engineering and induced pluripotent stem cells (iPSC)," says Dr. Haddad.

These infrastructures will catalyze the development of new and innovative therapeutic approaches for monogenic diseases and childhood cancers. Building on strategic collaborations with key partner institutions such as the Center of Excellence for Cellular Therapy at Maisonneuve-Rosemont Hospital (CIUSSS de l’Est-de-l’Île de Montréal), this unique program will also provide an attractive environment and an outstanding training opportunity for highly qualified personnel.

Although this program focuses on pediatric genetic diseases and cancers, the technologies developed will also be transposable to regenerative medicine and cancer immunotherapy in adults, significantly expanding the scope and impact of the investment.

CHU Sainte-Justine, leader

In Quebec, CHU Sainte-Justine is responsible for the diagnosis and treatment of nearly 70% of cases of rare diseases or childhood cancers and is the only center with approval for bone marrow transplants.

It is a world leader in contributing to the advancement of knowledge and the development of new therapeutic approaches for prevention, diagnosis and treatment in pediatrics, particularly for rare diseases.

"The achievement of our research objectives will strengthen our institution's position as a leader in gene, cell and tissue therapy in these fields," concludes Dr. Haddad.

About the team

The program is based on cutting-edge research and technological advances of the team members:

Haddad, Elie: Department of Pediatrics, Université de Montréal (UdeM), Head, Research Axis, Immune Disorders and Cancers, Clinician Scientist, Clinical Immunology, CHU Sainte-Justine (CHUSJ)

Pshezhetsky, Alexey V.: Department of Pediatrics, UdeM, Director, Elisa Linton Sanfilippo Research Laboratory, Scientific Supervisor, Biochemical Genetics Laboratory, Researcher, CHUSJ

Andelfinger, Gregor U.: Department of Pediatrics, UdeM, Head, Research Axis, Fetomaternal and Neonatal Pathologies, Clinician Scientist, Pediatric Cardiology, CHUSJ

Beauséjour, Christian: Department of Pharmacology and Physiology, UdeM, Researcher, CHUSJ

Cellot, Sonia: Department of Pediatrics, UdeM, Deputy Head, Research Axis, Immune Disorders and Cancers, Clinician Scientist, Pediatric Hematology, CHUSJ

Decaluwe, Hélène: Department of Pediatrics, UdeM, Deputy Head, Research Axis, Immune Disorders and Cancers, Clinician Scientist, Immunology, CHUSJ

Doyon, Yannick: Department of Molecular Medicine, Université Laval, Researcher, CHU Research Center, Québec-Université Laval

Dumont, Nicolas: Rehabilitation Department, UdeM, Researcher, CHUSJ

Paganelli, Massimiliano: Department of Pediatrics, UdeM, Deputy Head, Research Axis, Metabolic and Cardiovascular Health, Clinician Scientist, Pediatric Gastroenterology, CHUSJ

Touzot, Fabien: Department of Pediatrics, UdeM, Clinician Scientist, Immunology, CHUSJ

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