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Stem-cell replication to treat blood diseases

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Centre de recherche Hôpital uman clinical trials Maisonneuve-Rosemont, who are set to start in will lead the human clinical December to test an trial, this new approach could innovative approach to treatpotentially increase accessiing life-threatening blood bility to treatment by 30% to diseases, such as leukemia. 40%. A team of researchers from The blood of a newborn’s the Institute for Research in umbilical cord is a rich source Immunology and Cancer of hematopoietic stem cells (IRIC) at the Université de for transplantation. Because Montréal have discovered a the donor newborn’s immune new molecule, UM171, that system is still immature, the can be used to increase the stem cells are less likely to number of stem cells found in induce an adverse immune umbilical cord blood. The reaction in the receiver. innovative finding, which has Moreover, it is not necessary been described as a world breakthrough, was published A new technique for replicating stem cells will increase the quantity that the immunological com10-fold for the treatment of some blood-related diseases. patibility between donor and Sept. 19, 2014, in Science. recipient is perfect, unlike a According to the team’s bone marrow transplant. says Marinier, who is IRIC’s director of principal investigator, Dr. Cord blood transplants have been medicinal chemistry. Guy Sauvageau, this new synthetic used worldwide at an increasing rate Tests on mice, then monkeys, molecule could “multiply by 10 the since the late 1990s, but only 5% of showed the potent effect of UM171 in number of units of cord blood available adults can be treated efficiently this promoting the proliferation of blood for transplantation in humans” and way, according to Roy. That could stem cells. The molecule is now being would also substantially reduce complichange. “It could become a life-saving patented for potential commercializacations associated with stem cell transoption, especially for non-Caucasian tion. Marinier credits her experience as a plant. patients who have difficulties finding chemist at a pharmaceutical company When no compatible donor can be compatible donors,” believes Roy. with helping her choose the right comfound, stem cells from umbilical cords In the first and second phase of the pound. She says it’s the first time she’s are transplanted to treat blood diseases human clinical trial, the Centre of Excelseen that her research “could make a difsuch as leukemia, myeloma and lymlence for Cellular Therapy at Hôpital ference in someone’s life and prevent phoma. However, the number of stem Maisonneuve-Rosemont will be distribthem from dying. It is extremely rewardcells obtained from a unit of blood uting the grafts obtained from UM171 to ing for the team and the institute.” from an umbilical cord is usually too 15 patients in Québec City, Montréal Producing the stem cells involves low to treat an adult. In addition, findand Vancouver. The first results should using a bioreactor at a University of ing a source of stem cells for smaller be available in December 2015. Toronto bioengineering lab. The bio­ ethnic groups can be difficult. With the “The number of people waiting for a reactor prevents stem cell differentianew UM171 molecule, it would be postransplant in Canada is impossible to tion and offers a medium in which they sible, according to researchers, to harknow,” says Roy, “but there are about can proliferate without risking contamivest stem cells in culture, and produce one thousand allografts annually. This nation. In short, the bioreactor preenough to treat adults. new molecule could help increase the serves the integrity of the stem cells, “It was a bit of a fluke,” says Anne number of grafts by more than 30%. says Peter Zandstra, a bioengineering Marinier, coauthor of the paper and a And I am being conservative.” The main re­­searcher and director of the lab that chemist who practically hand-picked beneficiaries will be nonwhite patients houses the bioreactor. “We add small the synthetic molecule out of a library who do not have compatible donors. — amounts of media, and we do it in a of 125  000 compounds stored at the Veronique Morin, Montréal, Que. way that is cost-effective and reactive. institute. “When we saw how reactive it There is a real clinical need here.” was on blood stem cells, we were According to Dr. Jean Roy, of the extremely surprised and delighted,” CMAJ 2014. DOI:10.1503/cmaj.109-4925

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CMAJ, November 18, 2014, 186(17)

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Stem-cell replication to treat blood diseases.

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