Human clinical trials are set to start in December to test an innovative approach to treating life-threatening blood diseases, such as leukemia.

A team of researchers from the Institute for Research in Immunology and Cancer (IRIC) at the Université de Montréal have discovered a new molecule, UM171, that can be used to increase the number of stem cells found in umbilical cord blood. The innovative finding, which has been described as a world breakthrough, was published Sept. 19, 2014, in Science.

According to the team’s principal investigator, Dr. Guy Sauvageau, this new synthetic molecule could “multiply by 10 the number of units of cord blood available for transplantation in humans” and would also substantially reduce complications associated with stem cell transplant.

When no compatible donor can be found, stem cells from umbilical cords are transplanted to treat blood diseases such as leukemia, myeloma and lymphoma. However, the number of stem cells obtained from a unit of blood from an umbilical cord is usually too low to treat an adult. In addition, finding a source of stem cells for smaller ethnic groups can be difficult. With the new UM171 molecule, it would be possible, according to researchers, to harvest stem cells in culture, and produce enough to treat adults.

“It was a bit of a fluke,” says Anne Marinier, coauthor of the paper and a chemist who practically hand-picked the synthetic molecule out of a library of 125 000 compounds stored at the institute. “When we saw how reactive it was on blood stem cells, we were extremely surprised and delighted,” says Marinier, who is IRIC’s director of medicinal chemistry.

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