We can begin the quest for health, healing, and happiness for our children in the womb with in utero cord blood stem cell transplantation
Cord blood stem cells offer a largely unharnessed potential for indications in regenerative medicine. One avenue being pursued is the concept of treating babies in the womb to cure diseases before birth. Often times there are known family histories of hereditary disease in one or both parents. In such cases molecular diagnostics can be used for the detection of genetic abnormalities before birth.
A limited number of in utero (in the womb) cord blood stem cell transplantations to treat genetic diseases diagnosed in the fetus have been reported. However, in general it is still a procedure that begs perfection. In theory, cord blood stem cells are ideal donor cells with their high proliferative capacity and low immunogenicity (low tendency to react with immune cells from others). Equally attractive, the fetal stage of life ought to provide a recipient young enough to embrace donor cells prior to developing an immune system. The expanding fetus is also thought ideal for providing growing bones that can accommodate an influx of donor stem cells.
To understand the limitations of in utero stem cell transplantation observed in the clinic, scientists developed a sheep model for the procedure. Interestingly, the size and physiology of sheep is relevant enough to human physiology allowing comparisons. Scientists developed methodology on sheep which can be used on human patients – such as using ultrasound-guided visualization of the fetus to inject human cord blood stem cells.
A new drug was recently marketed for mobilizing stem cells from the bone marrow into the peripheral blood of adult patients. This drug, plerixafor, has been used in pediatric patients and shown to be well tolerated. Scientists tested the use of this drug in the fetal sheep for mobilizing stem cells out of the marrow to free up niche space for injected donor cells. The thought was that this would lead to therapeutic beneficial levels of donor cell engraftment. Data from their pilot study was promising . This supports the use of plerixafor in developing transplantation regimens for in utero cell therapy for human babies.
HemaCare is excited about developing cord blood stem cells for cell therapy and looks forward to the development of in utero stem cell transplantation for babies with genetic defects. We provide CD34+ cells and CD133+ cells as well as whole cord blood and bone marrow for research purposes.
1: Goodrich AD, Varain NM, Jeanblanc CM, Colon DM, Kim J, Zanjani ED, Hematti P. Influence of a dual-injection regimen, plerixafor and CXCR4 on in utero hematopoietic stem cell transplantation and engraftment with use of the sheep model. Cytotherapy. 2014 Sep;16(9):1280-93. PubMed PMID: 25108653.