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Promise of Newborn Stem Cells to Revolutionize Clinical Practice

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Shweta Sharma, PhD, Post-Doc Associate at Sanford Burnham Prebys Medical Discovery Institute, La Jolla, California and Scientific Writing Consultant at APEX Think Corporation

A decade ago, I embarked on my PhD training in translational medicine using stem cells as therapeutic agents. Recent and rapid advances in the field encouraged me to participate in the 13th Annual International Cord Blood Symposium (ICBS) roundtable discussion earlier this year, organized by Cord Blood Registry®, the world’s largest private newborn stem cell bank. There, I had the fortuitous opportunity to interact with some of the top global leaders in the cord blood and regenerative medicine space. These experiences not only enlightened me as to the existing body of knowledge regarding the therapeutic potential of newborn stem cells, but also got me thinking about why we as scientists should consider an Umbilical Cord Blood (UCB) bank as a potential untapped source of samples for our research and clinical trials.

Following the first successful stem cell transplant in 1988, which was performed on a child suffering from Fanconi’s anemia [1], UCB transplantation gained popularity amongst clinicians and scientists. Since then, UCB banking has continued growing as an industry, to fulfill medical needs and to keep up with consumer demand. These dedicated facilities play a pivotal role in safely securing high quality UCB units, many of which are already being collected in accordance with federal regulatory standards. Their main aim is to provide a better product when required for clinical utility. Just over a decade ago there were only 23 active cord blood banks in existence; now there are 485 cord blood banks worldwide. [2] So far, according to a recent publication in Cytotherapy this year, more than 35,000 UCB transplantations have been performed in the treatment of certain malignant and non-malignant diseases worldwide [3].

To date, the vast majority of transplantations that have been performed involved unrelated allogeneic donor cord blood. However, autologous stem cell transplants--where the transplanted cells are from the patient’s own cord blood--show great potential for regenerative medicine. Autologous stem cell transplantation eliminates the need for donor matching and therefore poses no risk of patient rejection. 

The cord blood industry is an expanding field and is gaining in popularity. Related biomedical fields, such as gene editing, were initially over-hyped as the breakthrough technology of the future, yet slow to be adopted by the somewhat skeptical scientific and medical community. After an initial bumpy start, these emerging fields are truly starting to deliver and be adopted by the mainstream.  The field of gene editing, and its use of CRISPR technology, is a great example. Back when CRISPR was first discovered, there was a great deal of concern and reservation about adopting this groundbreaking methodology. However, in recent years CRISPR technology has become widely accepted by biologists as a search-and-replace tool to alter DNA, correcting down to a single base in the code. It is now considered a promising new approach for gene editing without affecting germ cells in people with devastating illnesses [4,5]. Similarly, based on the tremendous therapeutic potential and initial pre-clinical success of cord blood stem cells, scientists are optimistic that UCB stem cells will be able to deliver new treatment modalities for many medical conditions in the next few years.

Currently, several FDA-regulated clinical trials using stem cells derived from UCB are underway in an attempt to find a treatment for such diseases as autism, pediatric stroke and cerebral palsy, etc. 

Promising preliminary results have paved the way for these trials to move into Phase II studies. For example researchers at The University of Texas Health Science Center at Houston, in collaboration with Cord Blood Registry, are commencing phase 2 of an innovative FDA-regulated clinical trial to investigate stem cell therapy in children diagnosed with cerebral palsy. The study aims to compare the safety and efficacy of an intravenous infusion of either banked cord blood or freshly harvested bone marrow [6].

Dr. Charles S. Cox, Jr., the principal investigator of the study, and one of the panelists at the recent ICBS Roundtable Discussion notes that in regard to this trial "there is preclinical data indicating that the ongoing neuroinflammatory response is a driver of further injury in CP, so the hope is to reduce this neuroinflammation." "Our goal is to break the cycle of inflammation and injury" adds Cox. [7]

Stem cell research is continuously evolving and umbilical cord blood banking will likely become more effective as cell-based technologies advance in the near future. However there are some technical challenges that the industry faces, and scientists are working hard to resolve them. One of the critical factors that can significantly influence and delay UCB engraftment is the limited number of stem cells present in a single UCB collection. Utilization of cord blood units that fail to meet cell dose guidelines established for transplant medicine can lead to graft failure and delayed engraftment kinetics in recipients. Therefore, it is an absolute priority and a real challenge to develop an efficient protocol for UCB cell expansion. In the last two decades, multiple strategies have been evolved such as optimization of cytokine cocktails, co-culture systems, small molecules, and expression of specific self-renewal related genes to improve the outcome of hematopoietic stem cell and HSC-progenitor cell expansion protocols. The ultimate focus is to increase the cell dose of a UCB graft.

Another technology, the ability to generate induced pluripotent stem cells (iPSCs), has changed the direction of stem cell research around the world and may represent a potential cell source for future personalized medicine. Cord blood banks provide an ideal source of cells for such an endeavor because they contain large numbers of well-documented cell populations. In addition, UCB cells are derived from a juvenescent source and are less likely to contain potentially deleterious mutations than cells derived from adult tissues. 

Early lab studies have demonstrated that UCB stem cells could be considered a promising source from which to generate iPSCs. Such UCB-derived stem cells could then be used to treat a wide variety of diseases including metabolic disease, genetic disease, congenital malformation and also, in wider application, many of the various injuries suffered with the onset of degenerative disease [8]. 

Therapeutic potential and initial clinical successes of UCB stem cell-based therapies has encouraged clinicians to launch a number of clinical trials in the past decade. Today, cord blood has been used in the treatment of more than 80 diseases and in the future, that number may grow. Laboratory/preclinical research results have shown that cord blood stem cells may also serve as a viable medical tool to help repair injured tissues or organs. It is clearly an emerging field that stands at the juncture of a variety of rapidly developing scientific disciplines and I am very excited and intrigued to be a part of this and see how this field unfolds. 

Disclosure: I was financially compensated by Cord Blood Registry® for my time and travel expenses to attend the 13th Annual International Cord Blood Symposium (ICBS) roundtable discussion 


[1] Gluckman E1, Broxmeyer HA, Auerbach AD, Friedman HS, Douglas GW, Devergie A, Esperou H, Thierry D, Socie G, Lehn P, et al. Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. N Engl J Med. 1989;321(17):1174-8. 

[2] Bioinformant: Biotechnology. Capitalizing on Opportunities in Cord Blood Industry Growth. Online publication Nov. 2013.

[3] Aldenhoven M1, Kurtzberg J2. Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions. Cytotherapy. 2015 Jun;17(6):765-74.

[4] Baltimore et al., A prudent path forward for genomic engineering and germline gene modification. Science 2015; 348 (6230): 36-38.

[5] Miler et al., Germline gene therapy: We're ready. Science 2015; 348 (6241): 1325. 

[6] https://clinicaltrials.gov/show/NCT01988584 

[7] http://www.cordblood.com/stem-cell-research/cord-blood-research/cerebral-palsy

[8] Zhou H et al., Rapid and Efficient Generation of Transgene-Free iPSC from a Small Volume of Cryopreserved Blood. Stem Cell Rev. 2015 Aug;11(4):652-65.