Development of novel immunotherapeutic approaches targeting gene fusion-derived neoantigens to cure pediatric acute leukemia


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Challenge: Acute leukemia is the most common blood cancer in children. Aggressive chemotherapy and radiation treatments cure about 80% of affected children. However, they are not perfect cures since a part of children is incurable and dies of leukemia and these treatments kill not only the cancer cells, but also the healthy ones. As a result, children who survive leukemia will develop other major health problems in their adult lives, including infertility, stunted growth, heart failure and secondary cancers. The challenge is to develop new drugs to treat the subgroup of patients who are currently incurable that will target only the cancer cells to reduce the side effects of standard therapy.

Solution: Over the past five years, the research team has learned more about the peculiarities of the genome of childhood acute leukemia, including a subtype of mutation named gene fusions. These are responsible for initiating leukemia in children and are absent in healthy cells. Cancer cells can be detected by small molecular flags on the surface of the cells that indicate whether a cell contains the fusion genes. The researchers propose to discover and develop therapeutic antibodies that can recognize these molecular flags and bind only to cancer cells. These antibodies can then be modified by adding toxic molecules to increase their ability to kill leukemia cells.

Expected Achievements /Impacts: The expertise of leukemia researchers at Sainte-Justine Hospital in collaboration with a Sherbrooke-based biotechnology company specialized in antibody development will ensure the success of this project and position Quebec as a leader in the treatment of pediatric acute leukemia. This project will facilitate the training of specialized workforce and open a new horizon for the accessibility to better targeted and less toxic health care to avoid the deleterious effects of current treatments on the long-term health of children.

Principal Investigator:
Étienne Caron
CHU Ste-Justine
Co-Investigators :
– Sonia Cellot, Isabelle Sirois, Mélanie Bilodeau / CHU Ste-Justine
– Arnaud Droit / CHUQ-UL
– Simon Gaudreau / Immune Biosolutions
Project of
$2,000,000 $ over 3 years
Supported by CQDM through :
– Ministère de l’Économie, de l’Innovation et de l’Énergie du Québec (MEIE)
And by co-funding partners:
– Neomabs Biotechnologies
– Canadian Cancer Society
– Oncopole-pôle cancer du FRQS
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