The first transplant using cord blood-derived hematopoietic stem cells was performed in October 1988. Since that year, remarkable achievements have been made in terms of research into umbilical cord blood-derived hematopoietic and progenitor cells. More than 40,000 UCB transplants (UCBTs) have been performed across different geographies of the world, in both children and adults, to treat diseases that include hematologic, metabolic, immunologic, neoplastic, and neurologic disorders.
Moreover, cord blood banking has been strengthened to the point that approximately 800,000 units now stored in public banks and more than 6.75 million units are stored within private banks worldwide. During the past 30 years, research in the umbilical cord blood field has made tremendous progress in the area of HSCT. Today, major efforts are focusing on increasing the rate of engraftment, in order to reduce the risk of infection and cost.
Public Cord Blood Banks
Today, nonprofit public cord blood banks are experiencing financial difficulties, particularly within the U.S. where many public cord blood banks are barely breaking even. Public cord blood banks spend from $1 million to $6 million for processing, testing, storing, and licensing technologies required for their operations. At the same time, revenue from the sales of cord blood units is relatively low and the loss is mostly compensated by government subsidies for registered units, as well as donations and grants.
Annually, a medium-sized public cord blood bank collects approximately 8,500 cord blood units, but ultimately stores only a small fraction of this collection, typically 5% to 35%. In a given year, a public cord blood bank will release only 0.1% to 3% of its stored units. In order to remain viable, some public cord blood banks undertake hybrid banking models. Others improve their financial position by offering processing and testing services to private banks. In some cases, public cord blood banks which are parts of large whole blood centers or hospitals can access cheaper medical transportation and lab work.
Initially, government subsidies and grants encouraged too many public cord blood banks to be formed. However, some of them have now shut down collection sites and others have closed altogether, transferring their inventory to other nearby public cord blood banks. In contrast, private cord blood banks always receive a fee for their cord blood or tissue banking services, and therefore, do not face these problems. However, in the case of public cord blood banks, the societal value outweighs the loss, and consequently, they are supported by the government so that transplant recipients can benefit.
Cord Blood and Tissue Applications
Currently, cord blood is being marketed for two uses: as a transplantation treatment for diseases including leukemia and sickle cell disease and as a potential source of cells for regenerative medicine to repair tissues damaged by everything from heart disease to cerebral palsy.
Hematopoietic stem cells (HSCs) present in the cord blood are used for homologous reconstitution of a patient’s blood and immune system in the same manner as a bone marrow or peripheral blood transplant. Cord blood was recognized as an alternative graft source for hematopoietic stem cell transplant (HSCT) in pediatric and adult patients only in 2001, and until 2012, the number of cord blood units released for transplantation increased. Unfortunately, 2012 was an inflection point and this number has decreased since that time.
Although the number of cord blood transplantations performed per year has been decreasing since 2012, the number of cord blood units released for regenerative medicine clinical trials has been increasing.
Thankfully, there is significant interest in evaluating cord blood as a therapeutic intervention in non-hematopoietic indications. In the mid-2000s, scientists started investigating cord blood in acquired neurological indications. Pilot and clinical trials enrolling pediatric patients with disorders such as cerebral palsy, autism spectrum disorder, and acquired hearing loss have already demonstrated the safety, and in some cases efficacy, of administering minimally manipulated cord blood cryopreserved in an autologous setting.
Another small Phase I study also confirmed the safety and feasibility of infusing allogeneic (donor) unrelated cord blood to treat adult ischemic stroke patients. Based on the observed safety profile and preliminary evidence of efficacy, several additional studies to determine efficacy and to evaluate the safety of this approach in human leukocyte antigen (HLA)-matched related and unrelated donor cord blood are now underway.
Monocytes isolated from cryopreserved cord blood have also been used to develop a cell therapy product called “DUOC-01”, developed by renowned cord blood researcher, Dr. Joanne Kurzberg of Duke University. It is meant for augmenting cord blood transplantation for the treatment of inherited demyelinating conditions of the central nervous system (CNS).
Additionally, MSCs isolated from cryopreserved cord tissue have been finding there way into a wide range of regenerative applications. ClinicalTrials.gov now listed an estimated 12,500 MSC trials using MSCs derived from cord tissue, adipose tissue, dental pulp, and beyond. Importantly, MSCs are also being explored for their potential use in the treatment of COVID-19 induced respiratory and immune complications, such as acute respiratory distress syndrome (ARDS).
The Future of the Cord Blood and Tissue Banking Market
To date, there has been substantial advances made with perinatal tissue applications—including but not limited to
Of course, fresh and cryopreserved cord blood products are also being offered to scientists for research purposes by small and large market competitors alike. Companies offering cord blood products for research purposes include Lonza, STEMCELL Technologies, AllCells, and dozens of others.
Additionally, cord blood and tissue banking market consolidation has been extremely aggressive in recent years, with more than 80% of the global cord blood market now controlled by the world’s ten largest operators. Each geographic market now has one or two large operators who are acquiring their smaller competitors in order to improve cost efficiencies and brand dominance. Meanwhile, investor appetite for cord blood banks has never been stronger, with CooperCompanies’ (NYSE: COO) acquisition of Generate Life Sciences and its 1.1 million stem cell units under management being a notable example.