Research Update

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AAMAC is very pleased to have recently approved funding for a project at B.C. Children’s Hospital entitled: Single cell profiling of hematopoietic stem cells in pediatric aplastic anemia. We spoke with co-investigator, Derek Chan, a pediatrics resident physician to learn more about this study.

What is this study about?
Healthy lifelong blood production depends on normal functioning stem cells in the bone marrow. Blood stem cells in aplastic anemia (AA), while known to be partially destroyed, are also dysfunctional in their ability to regenerate the blood system. However, the reasons for these underlying deficits remain poorly understood. To advance insights into this area, our study will use the latest sequencing technologies available to profile stem cells in pediatric aplastic anemia at the single cell level to determine if there are distinct biological pathways that explain why AA stem cells fail to restore blood production.

What are the potential benefits to patients?
Our rationale to compare pediatric AA stem cells to normal stem cells at such high resolution rides on the hope that we may be able to eventually identify targetable pathways to correct the underlying issues within AA stem cells. This profiling approach provides a critical blueprint that may ultimately lead to future novel target-oriented therapies that stimulate sustainable blood production in AA as an alternative to stem cell transplants and/or as an adjunct to immunosuppressive therapy.

What got you interested in this topic?
During my final year of medical school, I was moved by how ill pediatric patients diagnosed with AA can get. While a large majority of adult-centric AA research has led to new therapies such as Eltrombopag, a unique thrombopoietin receptor activator, clinical trials in pediatric AA have not shown parallel benefit, hinting at potentially different underlying biology and importantly underscoring a widening knowledge and clinical translation gap for these children. With this project, we hope to be able to shed direct light into pediatric AA and into ways for which blood production may be rescued in this particular patient population.

What is the timeline for the project?
Having access to pediatric AA samples alongside healthy donor samples through the B.C. Children’s Hospital Biobank has provided critical foundations for our study. While the turnaround for single cell profiling itself may be relatively quick, it will take time to make sense of the large datasets that come out of our cell sequencing efforts. We expect to have some preliminary results within 12 months, but in-depth analysis and validation of the results will likely take 2-3 years.

What does the AAMAC funding mean to you?
Funding for research and trainees has become much more limited and competitive over time. While there are many funding opportunities rightfully open for cancer research, there are relatively few that apply to rare disorders and/or non-malignant blood disorders. As such, receiving this funding from AAMAC is very important to researchers like myself who are interested in meaningfully moving the needle in translational research for bone marrow failure. In this study, AAMAC’s support has been matched with a seed grant from B.C. Children’s Hospital Foundation, so we are excited to embark on this work and will look forward to delivering on our findings in the future.

Derek Chan, MD, PhD, is a Pediatrics Resident at B.C. Children’s Hospital and Research Institute, part of the Department of Pediatrics in the Faculty of Medicine at the University of British Columbia.