Guy Sauvageau and Anne Marinier
Credit: IRIC
Just a few years ago, there was little hope for people suffering from hematological malignancies requiring stem cell transplants, such as acute leukemia and myelodysplastic syndromes. Today, thanks to the work of Université de Montréal professors Guy Sauvageau and Anne Marinier, new treatment options are gradually entering the market worldwide.
Experts in the molecular genetics of stem cells and medicinal chemistry respectively, the two scientists at UdeM's Institute for Research in Immunology and Cancer (IRIC) were behind the discovery of the UM171 molecule in 2014. Ten years later, UM171 cell therapy is poised to change the course of life and prognosis for patients beyond our borders.
To this end, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has just issued an opinion recommending approval of Zemcelpro®, as the first and only treatment authorized for adults with hematological malignancies requiring allogeneic stem cell transplantation, when no other suitable donor cell type is available.
The European Commission's final approval decision is expected by the end of the summer. Similar authorization in Canada and the United States may follow.
As soon as UM171 was developed, its potential to revolutionize blood stem cell transplantation by considerably multiplying the number of umbilical cord blood-derived cells was identified. In successful clinical trials on several blood cancer patients, UM171 significantly reduced graft rejection rates and mortality. This approach also increases the proportion of usable cord blood, facilitating access to compatible transplants. To date, almost 120 people have benefited from this cell therapy in Phase 2 clinical trials. A pivotal Phase 3 study will be launched shortly.
Based on the work of Marinier and Sauvageau, in 2015 a collaboration between research teams at IRIC and IRICoR, a non-profit organization focused on commercializing research, led to the creation of the Montreal-based biotech firm ExCellThera. Holding exclusive rights to the patented UM171 molecule, the company provides molecules and bioengineering solutions to develop the best cell and gene therapies. With official marketing authorization for the European territory, ExCellThera will move from being an R&D company to becoming a commercial-stage biotech.
