Canadian researchers have reported positive results from a trial of a molecule that can multiply the number of stem cells in umbilical cord blood units for transplantation.
The molecule, UM171, was tested in a phase 1-2 trial by a team of researchers led by Dr Guy Sauvageau at the University of Montreal, Canada. Out of 27 adult patients with bone marrow cancers recruited, 22 patients received single cord blood unit transplants which had been expanded with UM171.
These patients showed lower mortality rates and risk of graft versus host disease than expected. The results of the trial appeared recently in the journal Lancet Haematology.
Dr Sauvageau says: “Although the rate of graft versus host disease when using umbilical cord blood stem cells is low, these cells are rarely used because the cords are small and do not contain a sufficient quantity of cells to treat an adult.”
However, the UM171 molecule can multiply the number of CD34-positive stem cells in a unit of umbilical cord blood 10- to 80-fold.
“In just seven days, UM171 multiplies the stem cells by an average of 30-fold, while at the same time providing a rejuvenating effect on the cells by blocking the aging process,” Dr Sauvageau added.
Co-senior author Dr Sandra Cohen pointed out that: “The most impressive result is the low mortality rate associated with UM171 transplantation compared to conventional cord transplantation.
“Not a single patient needed immunosuppression treatment after 13 months, whereas with normal transplants, 50% of patients require such treatment at that point. No other biotechnology procedure has produced these kinds of results.”
A phase 3 clinical trial of the technique has been approved and will begin soon, Drs Sauvageau and Cohen say.
Cohen S, Roy J, Lachance S, Delisle JS, Marinier A, Busque L, Roy DC, Barabé F, Ahmad I, Bambace N, Bernard L, Kiss T, Bouchard P, Caudrelier P, Landais S, Larochelle F, Chagraoui J, Lehnertz B, Corneau S, Tomellini E, van Kampen JJA, Cornelissen JJ, Dumont-Lagacé M, Tanguay M, Li Q, Lemieux S, Zandstra PW, Sauvageau G. (2019) “Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, Phase 1-2 safety and feasibility study”, The Lancet Haematology, doi: 10.1016/S2352-3026(19)30202-9