The recent years have been witness to revolutions in the medical fields in many aspects, and more specifically in the development of applications in regenerative medicine. One of the most promising and fastest growing fields of regenerative medicine is cellular therapy for both personalized and generalized treatments. Indeed, incredibly innovative approaches have been and are being developed to treat degeneration of tissues and organs due to aging or damage using stem and progenitor cells. This led to an increase in cell therapy revenues from $410M in 2008 to $5.4B in 2012, truly marking our entry into the age of the cell and making cell-based therapeutics the next pillar of medicine; virtually every branch of medicine will see direct application of cell therapies.
Stem and progenitor cells are undifferentiated cells, which, under the right conditions, can differentiate to become virtually any type of cell that makes up the tissues and organs in the body. This ability makes stem and progenitor cells ideal for use in proven and novel clinical therapies to replace defective or damaged cells resulting from a variety of disorders and injuries as well as for use in research. Stem and progenitor cells provide the foundation for cell therapies and, amongst the different types of stem and progenitor cells, none has been more researched, funded and commercialized than mesenchymal and hematopoietic stem and progenitor cells (MSPC’s & HSPC’s respectively) typically found in bone marrow in large quantities.
Recent data show however that umbilical cord blood obtained from the umbilical cord and placenta is a rich source of MSPC’s and HSPC’s; the latter can be used for transplantation to treat a range of malignant, genetic, metabolic, and immune disorders. In fact, it was shown that cord blood HSPC’s are more proliferative and have a greater chance of matching family members than stem cells from bone marrow. Additionally, cord blood is not only widely available and easily accessible—collection is relatively non-invasive, safe and painless—but its derived HSPC’s can even be safely infused when they are an incomplete match for the recipient due to their immunologically naïve quality. In spite of this, cord blood is still routinely discarded as hospital waste along with the umbilical cord and placenta following birth when it should be collected, processed, tested and banked for future therapeutic use, especially since, until now, 50 percent of all patients seeking a transplant cannot find a match.
There have been more than 30,000 transplants worldwide using cord blood derived HSPC’s and the demand is still growing fast. Indeed, the National Marrow Donor Program estimates that by the year 2015, there will be 10,000 cord blood transplants worldwide per year as opposed to 3,000 per year in 2010. From 23 active cord bloods as of 2005, the Bone Marrow Donors Worldwide (BMDW) currently list 44 public cord banks from 26 different countries and there are approximately 225 private cord blood banks worldwide. That is an eleven fold increase (1,100 percent) in the companies involved in the industry over only a seven year period, and yet, the market is far from being saturated as the offer still cannot meet the demand—both in number and quality.
There are two main types of cord blood banks: licensed public banks, which ask mothers to donate their umbilical cord blood—only the umbilical cord blood—for future use by anyone who may be a potential match, and private or family banks, which will store cord blood, tissue and/or MSPC’s from either or both the umbilical cord and placenta for future use by the donor or matching family members for an average fee of $1,600 per item plus an annual storage fee of around $100 per item. Historically, public banks have been not-for-profit while private or family banks have been for-profit enterprises. The private cord blood industry has been criticized by a number of professional bodies including the EU ethics Committee and the Royal College of Pediatrics because the medical utility of private cord blood banks is believed to be more potential than actual and, thus, private cord blood banks threaten the supply of cord blood units to the public system which is based on actual needs and not potential ones.
It is important nonetheless to point out that the private banks are founded on the autologous (cells derived or transferred from the same individual’s body) business model, where cells are harvested from the patient to be treated in a facility before being shipped back to the physician for delivery into the same patient, who is thus both the donor and recipient. The allogeneic (cells derived from separate individuals of the same species) business model on the other hand is based on mass production of aliquots of cells from a single healthy donor to be shipped to the physicians for subsequent delivery into the patient—the allogeneic model being the preferred one by the big biopharmaceuticals companies of course. Against common belief, the autologous business model is far from being unsupported or unsound since there are currently over 650 FDA clinical trials involving autologous and allogeneic applications of MSPC’s and HSPC’s with a clear bias to autologous approaches as they don’t provoke an immunological response by the patient. In fact, recent data have shown that, in reality, it is not autologous vs. allogeneic but autologous and allogeneic as there is room for both therapies in treating patients, even within a single diagnosis.
Until recently, only one alternative existed to both the public donation and private storage: directed donation of cord blood donated to public banks to siblings who have an existing condition that may in the future require an allogeneic transplant. This alternative nonetheless was simply not good enough for parents who wanted to take a form of biological insurance to safeguard their children’s future and who understood the potential of autologous therapies and the advantages early-stage stem cells as opposed to adult stem cells—many studies showed greater proliferative capacity and greater plasticity of newborn stem cells as opposed to same types of stem cells harvested from adults. Furthermore, with the growing awareness of parents of stem cell therapeutics, a deeper commitment to research and development of stem cell therapeutics by the banks was expected: regenerative medicine is to be included in the world of tomorrow today. This situation gave rise to new kinds of umbilical cord blood banks—hybrid models that combine aspects of both the public and private systems with a clear commitment to research and development in the field of stem cell therapeutics.
The hybrid models can be classified into two broad categories: the cord blood banks that offer both options, either private storage or public donation, and the cord blood banks that make the privately stored cord blood available to the public system. The first category is the most prominent one in North America and Belgium and keeps gaining popularity with the recent announcement that the major public banks have agreed to expand their services to offer private storage. The second category is the most popular one in Spain, Turkey, the U.K. and Germany, each with its own characteristics. Indeed, In the Turkish model, according to government legislation, 25 percent of all privately stored cord blood is donated to the public system. In the Spanish model, cord blood stored in a private bank is registered on the Official Spanish Register of Bone Marrow Donors and should a patient in need of a transplant find a match in a private bank, parents are obliged to donate said cord blood and the storage fee is reimbursed. In the U.K., the Virgin model comes to mind where 80 percent of the privately stored cord blood is donated to the public system for allogeneic transplantation. And finally, in Germany, should a patient needing a transplant find a match in a private bank, the bank asks for permission to the parents for release, however, as opposed to the Spanish system, the parents are under no obligation to accept.
No matter the outcome of which hybrid model is the best, both public and private cord blood banks are now working together to implement new stem cell therapeutics and allow for a greater number of patients needing a transplant to actually find a matched donor.