Breakthrough UM171 Treatment for Blood Cancers Nears Approval in Europe

Developed at UdeM, the UM171 molecule may soon be approved in Europe to treat blood cancers in patients without a compatible donor, marking a significant step towards commercialization.

Professors Guy Sauvageau and Anne Marinier from Université de Montréal were instrumental in this breakthrough. Sauvageau specializes in molecular genetics of stem cells, while Marinier is an expert in medicinal chemistry. Their discovery of the UM171 molecule in 2014 has paved the way for new treatment options for hematological malignancies worldwide.

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Zemcelpro® for adults with hematological malignancies requiring allogeneic stem cell transplantation when no other suitable donor cell type is available. The final approval from the European Commission is expected soon, with potential authorization in Canada and the United States to follow.

UM171 has shown promise in revolutionizing blood stem cell transplantation by increasing the number of umbilical cord blood-derived cells. Clinical trials have demonstrated a significant reduction in graft rejection rates and mortality among blood cancer patients. Nearly 120 individuals have already benefited from this cell therapy in Phase 2 trials, with a Phase 3 study on the horizon.

In 2015, a collaboration between research teams at IRIC and IRICoR led to the establishment of ExCellThera, a Montreal-based biotech firm. With exclusive rights to the UM171 molecule, ExCellThera is focused on providing bioengineering solutions for cell and gene therapies. The company is set to transition from an R&D entity to a commercial-stage biotech with official marketing authorization in Europe.

According to the source: UdeMNouvelles.