Stem Cell Research and Development

ARCHIE PATEL – Stem cell research and therapy has become a very hopeful and advanced, albeit controversial, scientific research topic in recent years. Stem cells are unspecialized cells of the human body capable of differentiation and self-renewal, allowing for the production of specialized stem cells. There are several classification of stem cells and multiple stages of stem cell specialization that are not discussed in this article. For more information regarding the various classifications of stem cells, visit: https://stemcellres.biomedcentral.com/articles/10.1186 /s13287-019-1165-5.  This article discusses the world’s first in-utero stem cell transplant trial conducted by University of California San Francisco (UCSF) researchers in depth.

UC San Francisco researchers safely transplanted a woman’s stem cells into her growing fetus four months before birth at the UCSF Benioff Children’s Hospital. The infant was born at UCSF Medical Center at Mission Bay in February after undergoing the procedure performed by Dr. Tippi MacKenzie, the lead pediatric and fetal surgeon who pioneered the procedure with her peers after nearly a decade of research.

Elianna Constantino, the now 11 month-year-old infant who underwent the procedure as a fetus, suffered from alpha thalassemia, a genetic disorder, carried by approximately 5 percent of the world’s population, that reduces the body’s production of healthy blood cells and hemoglobin levels. Doctors diagnosed Constantino’s lethal blood disorder at twenty-one weeks and she was given an in-utero blood transfusion and enrolled in the clinical trial that saved her life. She was the first patient enrolled in the world’s first clinical trial that involved the use of hematopoietic stem cells (blood stem cells), or stem cells that induce the production of other blood cells.  

Fetuses that suffer from alpha thalassemia typically die before birth due to an enlarged, overworked, underperforming and failing heart that also significantly affects their brain development. For Constantino, a fetal ultrasound performed at twenty-one weeks displayed an expanded and overworked heart because she lacked functional red blood cells to effectively transplant oxygen to tissues and her vital organs. Typical management of the condition includes conducting fetal blood transfusions every three to four weeks and continuing these transfusions for life after birth. Several blood transfusions, however, can lead to a buildup of iron, which can be a dangerously fatal condition.  

An in-utero stem cell transplant can bypass such issues as the fetus’s immune system is still underdeveloped and significantly reduce the chance of reduction. The hematopoietic stem cells (HSCs) were extracted from the mother and injected into her abdomen and into the umbilical vein situated within the umbilical cord, where the researchers had hoped the HSCs to colonize and populate in the fetus’s bone marrow. While this procedure was successful in that it allowed Constantino to survive the remaining term, the procedure is not a cure for her condition. Dr. MacKenzie stated that, “it is too early to say how effective the stem cell transplantation will be, but we are encouraged by how well she and her mother have tolerated the treatment. Her healthy birth suggests that fetal therapy is a viable option to offer to families with this diagnosis.” 

Typically, fetuses diagnosed with alpha thalassemia are often terminated due to the poor prognosis and low rate of carrying to term. However, if this fetal stem cell therapy continues to show positive results, as seen in Elianna Constantino, this treatment can be further developed or altered to treat similar fetal hematologic conditions. 

As mentioned earlier, stem cell research has greatly expanded and employed in various subcategories in the biosciences, such as cancer and fetal genetic disorders. However, what will be even more interesting will be the future discoveries and therapies that will be developed using the current knowledge of stem cells.

Photography Source: https://medicalxpress.com/news/2018-06-baby-born-world-utero-stem.html

Sources & References

Baby Born in World’s First In Utero Stem Cell Transplant Trial

UCSF | 25 May 2018

Stem cells: past, present, and future

BMC | 29 February 2019