05 October 2020

A patient suffering from severe aplastic anaemia has been given a life-saving stem cell transplant boosted using the new UM171 molecule, it has been announced.

The case study, published in the European Journal of Haematology, discusses the procedure which was undertaken at the Institute of Hemato-oncology and Cellular Therapy of Maisonneuve-Rosemont Hospital (MRH) and the Institute for Research in Immunology and Cancer, both of which are affiliated with Université de Montréal, Canada.

Severe aplastic anaemia destroys stem cells in bone marrow and halts the production of red blood cells. The usual treatment is allogenic stem cell transplant, with aim of finding a donor whose stem cells are as compatible as possible with those of the recipient.

If no compatible family or unrelated donor can be found, stem cells from a haplo-identical donor may be considered as an alternative source of cells. However, no suitable donors were found for the patient in this case study.

Instead, the 29-year-old man received stem cells from an umbilical cord blood unit which had been expanded using an experimental compound called UM171.

Prof Jean Roy, a haematologist and clinical researcher at the MRH, said: “It was after having exhausted all our treatment options that UM171, which had already proven itself in a clinical trial in blood cancer patients, came into play.”

UM171 increases the number of stem cells in a unit of umbilical cord blood by an average of 35-fold and significantly reduces the risk of graft-versus-host disease, he said.

The stem cell transplant was successful, and 27 months afterwards the man has normal blood counts and no graft-versus-host disease (GvHD). The doctors say the treatment saved the young patient’s life.

Source: Claveau JS, Cohen S, Ahmad I, Delisle JS, Kiss T, Lachance S, Sauvageau G, Busque L, Brito RM, Bambace N, Bernard L, Roy DC, Roy J (2020) “Single UM171‐expanded cord blood transplant can cure severe idiopathic aplastic anemia in absence of suitable donors.” Eur J Haematol, doi: 10.1111/ejh.13504