Umbilical cord blood is often discarded as medical waste after a baby’s birth. However, this abundant resource is rich in cellular source material, which makes it an attractive option for cell and gene therapy developers.
The evolution of cord blood to treat disease
For nearly 30 years, physicians have used cord blood as a cell source for hematopoietic stem cell transplant (HSCT) to treat hematologic malignancies and disorders in children and adults. Researchers continue to seek new uses for cord blood. For example, cord blood has been used in clinical studies to determine the safety and efficacy of treating conditions such as inherited metabolic disorders, neurological disorders and stroke.
Cell and gene therapy researchers have also demonstrated value for the use of cord blood units (CBUs) as a source material. For example, researchers have isolated NK cells from cord blood for CAR-NK therapies as well as using cord blood for induced pluripotent stem cell generation.
The benefits of cord blood as a source material for cell and gene therapies
Cord blood stem cells are at a more naïve state
The naivety of cord blood cells can be advantageous to some cell and gene therapies. Cord blood is collected from a newborn infant who has not had the environmental and viral exposures of an adult donor. Because the cells are young, there is less of a chance of undesired mutations than in older cells.
For cell and gene therapy sponsors who are creating cord blood-derived induced pluripotent stem cells or NK cells, for example, this more malleable cell state is often desirable.
CBUs are highly characterized and readily available off-the-shelf
CBUs are collected at the time of a healthy baby’s birth with no risk of harm to the mother or baby. There are no donor availability or eligibility issues that cell and gene therapy sponsors may experience with adult donors.
Cryopreserved CBUs stored at cord blood banks are available off-the-shelf and are in abundant supply. In fact, there are currently more than 266,000 CBUs on the Be The Match Registry® and nearly 806,000 CBUs on U.S. and global registries.
Using the Be The Match Registry as an example, the CBUs have been highly characterized with specifications such as total nucleated cell count, CD34+ count, HLA type, volume and viability. The units meet donor eligibility and safety requirements for use in humans. These types of CBUs are available for clinical and commercial use.
In addition, research-use-only CBUs are available for non-clinical use. These CBUs are also characterized, but do not meet eligibility and safety requirements for use in humans. However, they can be useful for sponsors validating, qualifying and investigating internal procedures, processes and assays.
That ease of availability is a major advantage of using CBUs as a cellular source material for non-clinical, clinical or commercial use.
Cord blood banks understand the regulatory requirements of an FDA-approved product
Cord blood banks have regulatory experience that some may not expect. The blood-forming stem cells derived from cord blood are FDA-approved for use in patients who need unrelated HSCT for a disorder that affects the hematopoietic system.
These cord blood banks have experience manufacturing a GMP-grade product that meets the FDA licensure requirements. While the indication is different, the source material is manufactured in a way that the FDA oversaw, regulated and approved.
Considerations for contracting with multiple cord blood banks
The CBUs with the characteristics you need may be spread across multiple cord blood banks. Contracting with each cord blood bank individually is one option for accessing these CBUs. However, this can take significant time and resources. Working with Be The Match BioTherapies® can streamline this process and also has added benefits.
Cord blood banks must meet rigorous requirements
A robust supplier qualification process is a critical first step to ensure a cord blood bank can meet your needs. This is true whether you choose to contract with centers on your own or work with an organization like Be The Match BioTherapies as a single point of access to multiple cord blood banks.
Companies that work with Be The Match BioTherapies can take advantage of a streamlined contracting and qualification process. That is because Be The Match BioTherapies is part of the National Marrow Donor Program® (NMDP)/Be The Match®. The NMDP/Be The Match has a network of 16 member cord blood banks that manufacture and store the more than 266,000 CBUs listed on the Be The Match Registry.
All member cord blood banks have gone through the supplier qualification process, a process that takes approximately one year. Banks must meet rigorous participation requirements that are reviewed annually. For example, each bank must:
- Hold a BLA or be actively pursuing a BLA
- Be accredited by AABB or NetCord-FACT
- Have at least 500 HPC(CB) units that meet NMDP/Be The Match current inventory requirements for new CBUs
- Be actively collecting HPC(CB)
Holding or actively pursuing a BLA is critical. This demonstrates that the cord blood bank complies with FDA-regulations and can demonstrate that compliance to the FDA. That could help streamline your application with the FDA.
Be The Match BioTherapies centralizes this process for cell and gene therapy sponsors and cord blood banks, just as the NMDP/Be The Match does for transplant. Tapping into established processes—and the knowledge of cord blood bank processes and procedures—adds efficiencies for cell and gene therapy sponsors.
Cell and gene therapy sponsors have access to a searchable database and bioinformatics insights
Once you have identified the product attributes you need for your therapy, you must find cord blood units that match those attributes. Rather than your company working with each individual bank to identify those CBUs, Be The Match BioTherapies can search the Be The Match Registry for the CBUs that match your product attributes.
Our experienced Bioinformatics team offers additional support to help you analyze the inventory and select the best cord blood units for your therapy.
Having access to a large database of characterized CBUs can also help companies scale efficiently as their needs grow.
Moving CBUs from bank to company is time sensitive and takes experience
While it may seem easy to transport cord blood units from point A to point B, experience and oversight are crucial. The containers must be managed properly to ensure there are not temperature fluctuations that could compromise the CBU before its arrival. In addition, the dry shippers only maintain their temperature for a specific timeframe, so the products cannot get lost in transit.
It is critical to use an experienced team for supply chain management and logistics. This is an area where the experience of Be The Match BioTherapies and the NMDP/Be The Match cannot be equaled Our teams coordinate the time-sensitive, safe transport of more than 550 CBUs annually.
While cord blood has been used to provide life-saving treatments to patients for almost 30 years, its full potential has not yet been tapped. Using CBUs as source material for new cell and gene therapies is a fantastic use of this resource and can provide more treatment options to patients.
