Related Experiment Video
Updated: Jun 15, 2026

Ex Vivo Expansion of Hematopoietic Stem Cells from Human Umbilical Cord Blood-derived CD34+ Cells Using Valproic Acid
Published on: April 11, 2019
Overcoming the barriers to umbilical cord blood transplantation
Susan Staba Kelly1, Simrit Parmar, Marcos De Lima
1Department of Pediatrics, University of Texas MD Anderson Cancer Center, Houston, Texas 77030-4009, USA. sskelly@mdanderson.org
Insights
Umbilical cord blood transplantation shows promise, but faces challenges like graft failure. Strategies such as using two cord blood units and ex vivo expansion aim to improve outcomes for patients needing stem cell transplants.
Area of Science:
- Hematology and immunology
- Transplantation science
Background:
- Umbilical cord blood transplantation (UCBT) is increasingly used, particularly in pediatrics.
- Adult UCBT faces challenges including graft failure, delayed engraftment, and poor immune reconstitution, leading to morbidity and mortality.
- Limited cell dose restricts post-transplant therapies like donor lymphocyte infusions.
Purpose of the Study:
- To review current strategies for overcoming limitations in umbilical cord blood transplantation.
- To explore the efficacy of combining two umbilical cord blood units and ex vivo expansion techniques.
- To highlight the potential of UCBT as a stem cell source despite existing barriers.
Main Methods:
- Review of recent studies on umbilical cord blood unit combination therapy.
- Analysis of emerging research on ex vivo expansion of cord blood stem cells.
- Examination of outcomes data related to graft failure, engraftment, and immune reconstitution in UCBT.
Main Results:
- Combining two umbilical cord blood units shows potential but engraftment remains suboptimal.
- Ex vivo expanded umbilical cord blood is suggested to be safe and may improve transplant outcomes.
- UCBT offers advantages like HLA disparity tolerance, rapid procurement, and reduced graft-versus-host disease.
Conclusions:
- Despite challenges, advancements in UCBT, including unit doubling and ex vivo expansion, are enhancing its applicability and outcomes.
- Further research into optimal expansion conditions is crucial for maximizing UCBT benefits.
- Umbilical cord blood remains a promising stem cell source with ongoing efforts to overcome its limitations.
Abstract:
Umbilical cord blood (UCB) transplantation (UCBT) has seen a marked increase in utilization in recent years, especially in the pediatric population; however, graft failure, delayed engraftment and profound delay in immune reconstitution leads to significant morbidity and mortality in adults. The lack of cells available for post-transplant therapies, such as donor lymphocyte infusions, has also been considered a disadvantage. To overcome the cell-dose barrier, the combination of two UCB units is becoming commonplace in adolescent and adult populations, and is currently being studied in pediatrics as well. In some studies, the use of two UCB units appears to have a positive impact on outcomes; however, engraftment is still suboptimal. A possible additional way to improve outcome and extend applicability of UCBT is via ex vivo expansion. Studies to develop optimal expansion conditions are still in the exploratory phase; however, recent studies suggest expanded UCB is safe and can improve outcomes. The ability to transplant across HLA disparities, rapid procurement time and decreased graft-versus-host disease (GvHD) seen with UCBT makes it a promising stem cell source and, while barriers exist, consistent progress is being made to overcome them.
Related Concept Videos
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...
Tissue Transplantation
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...

