Cord blood can go a longer way: Development of hematopoietic stem cell expanding molecules towards the ideal transplant

Anne Marinier, PhD, Université de Montréal

Umbilical cord blood can be a promising source of hematopoietic stem cells (HSCs), used in treating blood diseases. Each unit of cord blood, however, has a low number of HSCs, precluding their widespread use. The development of stem cell expanding molecules is seen as a way to increase the levels of HSCs in units of cord blood, increasing their use in treating and curing malignant and non-malignant blood diseases.

Dr. Anne Marinier of the Université of Montréal has already developed one molecule, UM171, that is being assessed for safety and efficacy as a cord blood expander. Now her lab has identified another molecule, called UM092, that could complement UM171. She and her team are now focusing on identifying the best method of expanding cord blood hematopoietic stem cells for transplantation using these molecules. The team is using its expansion approach to enhance gene transfer, making gene therapy a widely applicable clinical procedure.

The work undertaken in this project will result in the production of UM092, delivering material ready for extended phase II clinical trials. Dr. Marinier has private sector partners who will play key roles in commercializing the novel compound. The outcome will be enhanced access of cord blood to larger numbers of patients, both in Canada and throughout the world, leading to a paradigm shift in the treatment of blood diseases.

Anne Marinier’s project is part of The 2016 Disease Team Research Agreement Program which provided $3.281M for 8 projects. A total of 41 investigators (8 Principal Investigators and 33 Co-Investigators) at 11 institutions and more than 80 trainees are engaged. Multidisciplinary teams supported through this program are focused on novel cellular or stem cell-related therapeutic approaches to treat disease. Commercialization is an important component for this program, as projects must demonstrate a path to market or clinic. Research supported from this competition address treatments for diseases such as arthritis, liver failure, and type 1 diabetes. In addition, support is provided to further ongoing research that is looking at existing drugs for the regeneration of neural tissue after a brain injury.