2020 has undoubtedly been a year of unpredictability. With the outbreak of the COVID-19 pandemic, the medical community has shifted its sight on finding innovative treatment options for emerging conditions. There have been multiple clinical trials in progress to test the safety and efficacy of umbilical stem cells in the treatment of respiratory problems induced by the novel coronavirus. Additionally, umbilical stem cells are being utilized in other clinical and therapeutic applications for conditions such as spinal cord injury, autism, multiple sclerosis, brain injury, and osteoarthritis. As medical science unveils the largely untapped potential of cord blood and cord tissue stem cells, we predict to see an increase in the need for expectant parents to store these valuable stem cells.
A Brief Glance into Cord Blood’s Past and Future
To date, over 40,000 cord blood transplants have been performed, and cord blood serves as an FDA-approved treatment for nearly 80 conditions. But, how did we get to this point?
The idea that umbilical stem cells could be cryopreserved and used for later transplantation was demonstrated over three decades ago with the first successful transplantation of a child with Fanconi anemia who received stem cell transplantation via his sister’s cryopreserved cord blood. Other cord blood transplants performed among siblings showed a significant decrease in the occurrence of Graft‐vs‐Host Disease (GvHD). Through the completion of additional clinical trials and successful transplants throughout the following years, cord blood proved to have advantages over bone marrow for human stem cell transplant, in part because this source does not require complete HLA matching. It was also discovered that the dose of cells used in a transplant could be increased by using two cord blood units.
This very notion paved the way for innovative solutions in the cord blood banking industry. Cryo-Cell adopted a five-compartment cord blood storage bag to allow families and treating physicians access to multiple uses of the baby’s cord blood stem cells. Similarly, this design feature opens up the door for future advancements in the ex vivo (outside the body) expansion of stem cells. A few select cellular therapeutic companies, including Gamida Cell, Nohla Therapeutics, Magenta Therapeutics, and ExCellThera, are creating stem cell expansion technologies. These technologies will be used to significantly grow the number of stem cells in hopes of delivering an “off the shelf” cellular product, as well as to expand the number of cells for individual use in personalized treatment.
This advancement comes at a time when the ability to identify a fully matched donor for a recipient in need of an unrelated donor for transplant, in public inventory, is predicted to decline over the next 1-2 generations. Furthermore, as ethnicities become more diversified, these patients are expected to have an even harder time finding an acceptable match through the public cord blood registry. The Parent’s Guide to Cord Blood reported the number of new cord blood donations added to the World Marrow Donation Association public inventory dropped from 69,000 in 2012 to 26,000 in 2018. The article concluded that cord blood banking is continuing to flourish worldwide. Thus, we can conclude, the need for parents of particular ethnicities to store privately becomes even more critical.
Clinical Trials Involving Umbilical Stem Cells
The stem cells found within cord blood and cord tissue have the capability to reconstitute “switched off” cells and turn into other cell types. In turn, their adaptability has been the consideration of many studies. Of cord blood, a source expounds, “The need for cord blood transplantation to treat malignancies, hemoglobinopathies, primary immunodeficiencies, and metabolic diseases within the last decade has prompted the American Academy of Pediatrics (AAP) to revise its 2007 policy statement “Cord Blood Banking for Potential Future Transplantation.”
This past month, Dr. Frances Verter compiled a list of cumulative trials and recruiting trials using cord blood and cord tissue through the end of 2019 and 2020. Since the initiation of her team’s research in 2017, there were a reported 114 cord blood trials and 49 cord tissue trials. As of April 2020, there were a reported 123 recruiting cord blood trials and 155 recruiting cord tissue trials, a number that has more than tripled in size. Indications treated by umbilical cord mesenchymal stem cells include various neurologic, immunologic, hematologic, liver, cardiac, endocrine, musculoskeletal, pulmonary, dermatologic, and ophthalmologic conditions, with a few other outlier conditions noted.
A Further Look at Cord Tissue
Mesenchymal stem cells (MSCs) derived from cord tissue are used for regenerative purposes. Cord tissue stem cells have the function of inhibiting inflammation following tissue damage, secreting growth factors that aid in tissue repair, and differentiating into many cell types, including neural cells, bone cells, fat cells, and cartilage. Since 2016, umbilical cord tissue has become the leading source of MSC for all MSC clinical trials, according to a publication featured in The Journal of Regenerative Medicine. As one source describes it, “Umbilical cord-derived MSCs can claim the title of the new ‘gold standard,’ ousting the renowned bone marrow-derived MSCs.” Furthermore, the article surmises an increased demand for cryopreserved umbilical tissue in the immediate future.
Interestingly, most of the former research used MSCs sourced from fresh cord tissue. However, more recent research is unveiling the need for cryopreserved cord tissue. Another publication elaborates, “While most studies using animal models and even small clinical trials have utilized fresh MSC cultured on-site, cryopreservation of MSCs is essential to the widespread application of MSC-based therapies. Cryopreservation allows for MSCs to be prepared by specialized facilities, in large batches under the application of accepted quality control measures.” The importance of having a specimen for use that has undergone the necessary preparation to ensure integrity and viability remains a key aspect of family banking.
Umbilical Stem Cell Banking, All Signs Point to Yes
The demand for cord blood banking is showing a steady increase, while the need for cord tissue banking is showing a dramatic incline. Dr. Joanne Kurtzberg, our medical director and internationally renowned expert in pediatric blood and marrow transplantation, noted, “The cord blood industry was the first to enter the regulated cell therapy environment, but its experience and progress to date can be leveraged for the development of many promising and exciting therapies to come.”
As these stem cells continue to prove successful in ongoing research and current transplants, new treatment opportunities will likely arise for a growing list of diseases and conditions. Parents who make the important decision to store their baby’s umbilical stem cells do so with the foresight of what can be made possible for their families in the years to come. We will continue to contribute to the mission of advancing regenerative therapies, and will keep you updated each exciting step of the way!