'Untapped potential for cord blood in disease treatment’
Researchers have been isolating NK cells from cord blood for CAR-NK therapies as well as using it for induced pluripotent stem cell (iPSC) generation, said Donna Regan, director of customer-ready products for the National Marrow Donor Program/Be The Match, including Be The Match Biotherapies (BTMB).
Cord blood has been used as a cell source for hematopoietic stem cell transplant (HSCT) for over 30 years to treat hematologic malignancies and disorders in children and adults.
But there is a plethora of untapped potential uses for cord blood units (CBUs) in relation to disease treatment, according to Regan.
“By working together, cord blood banks (CBBs) and developers can define a mutually sustainable supply chain for the discovery and development of new therapies,” she told BioPharma-Reporter.
In terms of the benefits of using CBUs as CGT source material, she said:
“Practically speaking, CBUs are ‘off the shelf’, ready to be selected.
“Donors have been screened and tested for eligibility in accordance with current US Food and Drug Administration (FDA) requirements, eliminating the time for work up.
“From a biologic perspective, the placental barrier protects CBUs from direct exposure to viral and environmental pathogens, while their age reduces the probability for mutations, and cells are more naïve and plastic than adult stem cells.”
Supply hurdles
But bottlenecks exist in relation to the supply of CBUs for use in cell therapies.
“Donations must be properly consented for commercial use, meaning that donors must be well informed of the variety of ways their gift could be used.
“Developers engaging in the investigation of cord blood derived cell therapies in humans must present protocols that are overseen for ethics and registered with the FDA under an IND, with the intent to license,” continued the specialist.
Developers must be able to articulate their needs, and they also are required to understand what is available in terms of the inventory: volumes, cell concentration, how the CBUs are processed and characterized, the number of reserve samples and their utility, for example, said Regan.
Registries are useful in this regard, she explained. They not only list CBUs from multiple qualified banks, but they are also able to search the inventory for desired attributes, leverage their relationships with suppliers, and capture outcomes on patients.
'Collaboration will aid use of CBUs in advanced therapies'
Enhanced industry relationships and refined logistics processes will also improve cord blood accessibility for use as a CGT starting material, added Regan.
“By collaborating to understand the state of cord blood banking and the vision of developers, the industry can maximize the potential of this abundant and valuable resource.
“CBBs are keen to support the utility of the products in their inventory, not only for traditional transplant, but for the advancement of new therapies.
“They also have decades of experience in GMP manufacturing for clinical use. This expertise can be leveraged to refine practices that meet the needs of developers.”
CBBs, she continued, are capable of modifying processing, testing and storage to meet the needs of developers – but the banks must know what is required.
“That said, not every specification can be met. Customization is inversely proportional to scalability, so adopting the most common themes will facilitate availability.
“Sourcing from a registry that performs robust supplier qualification will do the heavy lifting for developers and eliminate the need to duplicate the process for individual vendors.”
Regulatory benefits
Are there regulatory associated benefits from using CBUs in cell therapies?
“It’s thought that, since the FDA regulates the production of cord blood, sourcing from CBBs will smooth the supplier/material aspect of their BLA. That may be so, however, CBBs comply with regulations for a specific indication which is likely very different from the therapy under development. The final product will still need to be tested for adventitious agents and potency related to the intended use.
“But, again, decades of experience in cord blood banks can refine the manufacturing processes to align with new protocols.”
