News
UM171 breaks new ground
Published on June 26, 2025
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 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 IRIC scientists and UdeM professors were behind the discovery of the UM171 molecule, which saw the light of day in 2014.
10 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. If positive, it will apply to all 27 member states of the European Union (EU), plus Iceland, Norway and Liechtenstein. The use of Zemcelpro® in other patient populations and for other types of hematological disease is also being studied.
The expected European authorization will pave the way for similar authorization in Canada and the United States. Efforts in this direction are already underway.
“These advances are spectacular. To think that it only took a decade to reach the many milestones, from the discovery of UM171 in the laboratory in Montreal to its clinical application in patients around the world, is almost a fantasy. I’m extremely moved by what can be achieved when there’s a shared vision and a clear goal,” says Anne Marinier, Director of the Drug Discovery Unit and Principal Investigator at IRIC.
“It’s proof that bold science, when tenaciously supported by scientists, clinicians, technologists, partners and public investment, can really translate into targeted therapies for patients. I have in mind, among others, the collaborative work of the IRIC and IRICoR teams, ExcellThera, C3i, Maisonneuve-Rosemont Hospital, financial partners such as the Stem Cell Network, and philanthropists, to name but a few. I can’t wait to hear about the next steps and the global successes that await the UM171 molecule,” adds Guy Sauvageau, Principal Investigator at IRIC and hematologist at Maisonneuve-Rosemont Hospital.
“We are proud to see that a molecule discovered by IRIC scientists is poised to revolutionize patient care. Behind this major breakthrough lies the hard work of our teams, who often work behind the scenes, but who are determined to pursue our mission. This kind of news is an incredible source of motivation for all researchers who want to make a difference for our society”, explains Marc Therrien, IRIC’s Chief Executive Officer.
UM171 cell therapy: from discovery to clinical application
As soon as the molecule 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, the use of 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.
ExCellThera: a key player behind UM171’s success
In 2015, it was the joint work of IRIC and IRICoR teams, based on the work of Anne Marinier and Guy Sauvageau, that led to the creation of ExCellThera. A Montreal-based biotech company, ExCellThera provides molecules and bioengineering solutions to develop the best cell and gene therapies. ExcellThera holds exclusive rights to the patented UM171 molecule. With the official marketing authorization for the European territory, ExCellThera will officially move from a leading R&D company to a commercial-stage biotech.
