Projects supported with Cord Blood Products

This list will be updates as research projects are approved.

An automated manufacturing solution for expansion of blood stem cells

An automated manufacturing solution for expansion of blood stem cells

Project Location: Toronto

Estimated Project Start Date: 2018-07-01

Estimated number of cord blood units required to complete this research project: 30

Lay Summary of the Research Project:
Umbilical cord blood has been proven to be a valuable source of blood stem cells for transplantation to treat blood disease and cancer. In order for these cells to be available in sufficient quantities, they can be expanded in culture, outside of the body. By increasing the number of stem cells, each umbilical cord blood unit is made more clinically efficacious. We are developing an automation solution to make this expansion process more robust and reproducible. This will enable these cells to be used in large scale clinical trials and ultimately for commercial scale production of this therapy. 

Hematopoietic stem cells and transfusion medicine

Hematopoietic stem cells and transfusion medicine

Project Location: Ottawa

Estimated Project Start Date: 2018-08-01

Estimated number of cord blood units required to complete this research project: 125

Lay Summary of the Research Project:
Umbilical cord blood units are a source of hematopoietic stem cells used for transplant in patient without a suitable donor. Collection, processing, and storage under freeze are steps involved in cord blood banking. The impact of these processes on hematopoietic stem cell quality and activity need to be investigated to optimize the quality of the cord blood graft distributed by the Canadian Blood Services. Our projects investigate the impact of processing on cord blood quality and potency, investigate the impact that expansion of the graft has on the its quality and finally test new additive solution to improve the recovery of the graft after freeze. Improved quality will translate into improved activity which will reduce issues in patients and hasten their recovery

Regulatory T cells from discarded human thymus for adoptive cell immunotherapy: moving garbage to gold

Regulatory T cells from discarded human thymus for adoptive cell immunotherapy: moving garbage to gold

Project Location: Edmonton

Estimated Project Start Date: 2018-03-01

Estimated number of cord blood units required to complete this research project: 22

Lay Summary of the Research Project:
Patients who received transplant or those with autoimmune diseases often need lifelong treatment with drugs that suppress the immune system. In this regard, special immune cells called ‘regulatory T cells’ (or ‘Tregs’) are of enormous interest because they naturally suppress immune responses. The use of Tregs as a cellular therapy to treat a variety of immune disorders, including diabetes and transplant rejection is currently being explored. In this project, we will develop standard laboratory protocols for isolation, expansion and storage of thymic Tregs. We will test whether these Tregs can suppress immune responses effectively. If so, our work will allow direct clinical testing of thymic Tregs as an “off-the-shelf” therapy. We expect to demonstrate that discarded human thymus is a superior source of therapeutic Tregs that can be moved forward to clinical testing.

Cell-based therapy for experimental acute kidney injury

Cell-based therapy for experimental acute kidney injury

Project Location: Ottawa

Estimated Project Start Date: 2018-02-01

Estimated number of cord blood units required to complete this research project: 36

Lay Summary of the Research Project:
Acute kidney injury (AKI) is a serious condition in which kidney function falls rapidly, often due to loss of blood flow. AKI affects about one out of every twenty people in hospital, and mortality is high. Unfortunately, there are no treatments to improve kidney repair after AKI. Use of stem cells may help kidneys recover from AKI. Recently, we isolated “progenitor” cells from human umbilical cord blood and infused them into mice with AKI caused by temporary blockage of blood flow to the kidneys. The cells reduce kidney damage, and appeared to release factors that stimulated recovery. In fact, the cells shed tiny membrane particles (“exosomes”) that contain a protective substance called “miR-486-5p”. Our proposal represents the critical next step in developing cord blood-derived exosome treatments that could help humans with AKI

Study of immuno-therapeutics anti-tumor efficacy in humanized mice

Study of immuno-therapeutics anti-tumor efficacy in humanized mice

Project Location: Toronto

Estimated Project Start Date: 2018-02-01

Estimated number of cord blood units required to complete this research project: 24

Lay Summary of the Research Project:
Immunotherapeutic antibodies have been used to treat some cancer patients successfully through boosting the patients’ own immune system. However, scientists are increasingly aware of the complexity of this type of therapy and of the urgent need for developing next-generation antibodies with better efficacy. Therefore, we have generated some next generation immuno-therapeutic antibodies. We will use cord blood as a source to purify hematopoietic stem cells (HSC) and then generate humanized mice. We will use humanized mice as a model system to assess our immunotherapeutic Abs anti-tumor efficacy and advance them to clinical trials

Stem Cells of the musculoskeletal system: An epigenetics study from childhood to adulthood

Stem Cells of the musculoskeletal system: An epigenetics study from childhood to adulthood

Project Location: Ottawa

Estimated Project Start Date: 2018-01-15

Estimated number of cord blood units required to complete this research project: 54

Lay Summary of the Research Project:
Sarcopenia is the age-related loss of muscle mass that occurs when the muscle stem cells fail to meet the regenerative demands leading to muscle atrophy. Past research in mice has demonstrated that there are differences in which genes are active in young individuals compared to older individuals, particularly in muscle regeneration. In this project, we will use adult stem cells (including hematopoietic stem cells from cord blood) isolated from human biopsies to identify and characterize the epigenetic changes that contribute to the progression of age-related muscle wasting. Successful completion of this project is expected to help identify genes that can be targeted in certain diseases and aging processes.  

Characterization of post-thaw recovery of cord blood progenitors cryopreserved with novel recrystallization inhibitors

Characterization of post-thaw recovery of cord blood progenitors cryopreserved with novel recrystallization inhibitors

Project Location:  Ottawa

Estimated Project Start Date: 2015-12-18

Estimated number of cord blood units required to complete this research project: 20

Lay Summary of the Research Project:
Umbilical cord blood is a rich source of stem and progenitor cells with current and future applications in regenerative medicine. The national public cord blood bank (NPCBB) collects and stores cord blood units for future stem cell transplantation. Cryopreservation is the storage of biological products at temperatures below -180 degrees Celsius. The current standard for cord blood banking is to cryopreserve cord blood units with 10% DMSO. However, DMSO is associated with a number of toxic effects. Given that the majority of cellular injury during cryopreservation is a direct result of ice growth, we hypothesize that our novel small molecule ice recrystallization inhibitors will minimize the cellular damage associated with ice growth and will result in improved cord blood unit functionality when thawed.

Application of human NK cell therapy and the use of humanized mice to study prevention and treatment against cancer

Application of human NK cell therapy and the use of humanized mice to study prevention and treatment against cancer

Project Location:  Hamilton

Estimated Project Start Date: 2016-01-05

Estimated number of cord blood units required to complete this research project: 120

Lay Summary of the Research Project:
Until recently, responses of the human immune system to human cancers and human specific infections, such as Hepatitis C virus, were not possible to study in a mouse model. The goal of our research is to use cord blood stem cells isolated from umbilical cord blood to generate "humanized mice" which have a functional human immune system. This gives us the ability to investigate how our immune system works in a living system, against a variety of diseases which are impossible to examine at this level in humans. We aim to study both cancers, such as breast cancer and infections, such as Hepatitis C virus. Through a cell culture method established in our lab, we can produce a large number of immune system cells from a small cord blood sample. These immune cells can then be used to treat humanized mice with either tumors or infection. Ultimately, through cord blood donation, we will be able to generate humanized mice and apply what we learn to create more effective treatments to fight breast cancer and viral infection.

Effects of radiation on cord blood-derived stem cells

Effects of radiation on cord blood-derived stem cells

Project Location: Chalk River

Expected Project Start Date: 2015-09-01

Estimated number of cord blood units required to complete this research project: 30

Lay Summary of the Research Project:
Patients with hematological cancers often undergo stem cell transplantation that requires high dose chemo- or radio-therapy to kill all resident leukemic cells followed by transplantation of blood stem cells from a healthy donor. Unfortunately, 50% of patients undergoing this therapy still succumb to the disease due to leukemia re-emergence (leukemia relapse). One of the hypotheses for leukemia relapse is the existence of leukemic cells that are resistant to chemo-/radio-treatment. However, recent studies have shown that low doses of radiation can induce biological effects that promote cancer cell destruction. We propose to study whether low doses of radiation given to patients prior to chemo-/radio-therapy and stem cell transplantation can promote better leukemia cell destruction. This approach may greatly improve the outcomes of transplantation therapy and prevent future leukemia relapse. Importantly, these studies require the use of human blood stem cells and cord blood is a rich source of these cells.

Development and validation of a humanized mouse model for the pre-clinical evaluation of vaccines against liver pathogens

Development and validation of a humanized mouse model for the pre-clinical evaluation of vaccines against liver pathogens

Project Location: Edmonton

Expected Project Start Date: 2015-03-17

Estimated number of cord blood units required to complete this research project: 25

Lay Summary of the Research Project:
Malaria is responsible for nearly 1.24 million deaths annually, mostly young children in sub-Saharan Africa. Despite decades of research, a vaccine to prevent malaria remains elusive.  One of the major challenges in vaccine development is lack of suitable animal models to evaluate new experimental vaccines.  Chimpanzees and other primates are not available for either ethical or financial reason and there is currently no way to test vaccines in mice against the human species of parasites. In order to overcome some of these obstacles, we will develop a mouse transplanted with a human liver and a human immune system established from cord blood. We will use these mice to test new experimental malaria vaccines. The ability to optimize the delivery of the vaccine in mice prior to embarking on expensive human trials will enable the rapid early phase development of promising vaccines against malaria and other pathogens that infect the liver.

Cell production augmentation techniques

Cell production augmentation techniques

Project Location: Kingston

Expected Project Start Date: 2015-03-01

Estimated number of cord blood units required to complete this research project: 20

Lay Summary of the Research Project:
Regenerative medicine involving the transplantation of cells holds great promise for improving the clinical efficacy of conventional treatments. This is possible because the implantation of repair-specific cells (such as stem cells from cord blood) have the ability to restore tissue to normal function. The current procedures to create cell-based products are all manual in nature and hence do not enable high reproducibility and cost-effectiveness. Automated production could potentially be achieved through the robotic duplication of manual actions; however, the general industry challenge is that existing robotic systems for basic cell culture are highly complex, require significant space for operation, and require a high capital equipment investment. We are investigating cell parameters and system requirements to develop an innovative, cost-effective and automated bioreactor-based system to meet the clinical production challenges related to cord blood expansion in order to provide more options for patient transplant.

Strategies for enhancing cell therapy for optimal regeneration in pulmonary arterial hypertension – SECTOR-PAH

Strategies for enhancing cell therapy for optimal regeneration in pulmonary arterial hypertension – SECTOR-PAH

Project Location: Ottawa

Expected Project Start Date: 2014-11-28

Estimated number of cord blood units required to complete this research project: 200

Lay Summary of the Research Project:
Pulmonary arterial hypertension (PAH) is a devastating disease in which high blood pressures in the lung lead to heart failure and eventually death. Emerging regenerative therapies involving endothelial progenitor cells (EPCs) have begun to show some promise in clinical trials. Although EPCs are typically rare in circulating blood, we now know that they are more abundant in cord blood and may also be even more efficient at cardiovascular repair. We intend to use cord blood to efficiently generate EPCs and perform studies so that we may better understand why they are effective in PAH. With a better understanding of their mechanisms, we aim to develop methods to further enhance the activity EPCs to design more effective therapies for cardiovascular diseases such as PAH.

Ex Vivo Differentiation of Human Hematopoietic and Endothelial Progenitor Cells for Genomics and Proteomics Analyses

Ex Vivo Differentiation of Human Hematopoietic and Endothelial Progenitor Cells for Genomics and Proteomics Analyses

Project Location: Ottawa

Expected Project Start Date: 2014-08-05

Estimated number of cord blood units required to complete this research project: 300

Lay Summary of the Research Project:
Cord blood is a rich source of blood stem cells. These cells are currently used therapeutically in patients with beta-thalassemia, leukemia, limb ischemia, myocardial infarction and stroke. However, blood stem cells cannot be isolated in sufficient quantities to treat adults and cannot be expanded in the laboratory. Furthermore, there is room to improve their efficiency in repairing diseased tissue.

Our laboratory is using innovative technologies to decipher the role of specific proteins that control the function of blood stem cells. This will allow us to identify specific drugs to ameliorate the ability of these cells to treat disease. We anticipate that our study will lead to the development of new strategies to 1) expand blood stem cells in the laboratory such that enough cells can be produced to treat adults; and 2) improve the function of blood stem cells such that they are more efficient in repairing damaged blood vessels.

Evaluation of culture media for cord blood-derived progenitor cells

Evaluation of culture media for cord blood-derived progenitor cells

Project Location: Vancouver

Expected Project Start Date: 2014-08-05

Estimated number of cord blood units required to complete this research project: 20

Lay Summary of the Research Project:
A set of blood-derived progenitor cells could be useful for the treatment of diseases as diverse as vascular disease, stroke, cancer, and diabetes. However, these circulating cells are extremely rare, and must be cultured (grown in medium) to expand their numbers before use. We are evaluating several cell culture media for the isolation and expansion cord blood-derived progenitor cells, which could be useful in the future in human cell therapy.

Elucidating the role of PCL2 in normal hematopoiesis and leukemogenesis

Elucidating the role of PCL2 in normal hematopoiesis and leukemogenesis

Project Location: Ottawa

Expected Project Start Date: 2014-08-20

Estimated number of cord blood units required to complete this research project: 75

Lay Summary of the Research Project:
Acute Myeloid Leukemia (AML) is one of the deadliest types of cancers. 30-40% of AML patients younger than 60 years of age are long-term survivors, while only 10-15% of patients over 60 years of age are long-term survivors. Less than 10% of patients with relapsed AML survive long-term. The standard treatment has not changed in over 30 years, which is why survival rates have not improved. We recently identified a protein called PCL2, which is abnormally expressed in most AML patients. We believe that PCL2 could be a target for new chemotherapeutic drugs to treat AML patients that harbour PCL2 mutations. To pursue this strategy, we need to understand the role of PCL2 in normal and leukemic blood development. Therefore we will manipulate the expression of PCL2 in umbilical cord blood cells to help us understand the role of PCL2 with the ultimate goal of developing better therapies for AML.

Developing bioprocesses to expand cord blood hematopoietic stem cells for clinical use

Developing bioprocesses to expand cord blood hematopoietic stem cells for clinical use

Project Location: Toronto

Expected Project Start Date: 2014-09-10

Estimated number of cord blood units required to complete this research project: 540

Lay Summary of the Research Project:
Hematopoietic stem cell (HSC) transplantation is the only stem cell therapy routinely performed in patients with leukemia and lymphoma. However 40% of patients will not find a suitable bone marrow donor. Using umbilical cord blood (UCB) as an alternative source of HSCs has several advantages, including non-invasiveness, ready availability, and a higher tolerance for HLA mismatches. The limited number of stem and progenitor cells in a single UCB unit has impaired the success rate of UCB transplants. Our lab has developed an automated closed-system bioprocess that robustly expands UCB-derived HSCs and progenitors. Our next steps involve the use of a novel small molecule and integrating the detection system into the bioreactor to automate media injection, both of which will maximize the expansion of stem cells and enable the real-time control of the UCB cell culture in a sample-specific manner while minimizing the media use and the associated cost.

Characterization of the hematopoietic reconstitution enhancing activity of osteoblasts derived from human mesenchymal stromal cells

Characterization of the hematopoietic reconstitution enhancing activity of osteoblasts derived from human mesenchymal stromal cells

Project Location: Ottawa

Expected Project Start Date: 2014-09-22

Estimated number of cord blood units required to complete this research project: 75

Lay Summary of the Research Project:
Umbilical cord blood transplantation provides the opportunity for patients without a suitable donor to receive a life-saving stem cell transplantation. This procedure is however associated with a slower recovery of blood cells. Cord blood stem cells can be expanded in culture, and these have been shown to be able to produce blood cells faster. In this proposal, we will investigate how bone marrow cells regulate the growth of cord blood stem cells. We will also try to identify molecules that are responsible for these activities. This research will foster new knowledge on the role of bone cells as important regulators of blood cell production, and may lead to new solutions to improve cord blood transplantation. Indeed, new approaches are urgently needed to address the needs of the increasing number of Canadian patients who will soon rely on UCB transplantation for their continuing health.