According to a recent report from BioWorld, there are over 1,000 clinical trials taking place throughout the world in the field of cell and gene therapy. Consequently, there is a huge need for allogeneic donors to supply cells for the manufacturing of those therapies. That demand will only increase as the field matures and as allogeneic cell therapies are commercialized and used to treat greater numbers of patients.
As the industry grows, manufacturers and cell therapy developers alike are facing the challenge of how to scale their capabilities to reach larger numbers of patients. Among other factors, enabling access to a broader pool of donors is one critical next step. That’s according to Mark Flower, Vice President, Business Development and Strategic Partnerships at Be The Match BioTherapies®.
“The number of donors needed today is relatively small, with many therapies in early-stage clinical trials,” Flower said. “But if you fast forward five or 10 years from now, it is possible that companies are going to need exponentially more donors. They will likely be unable to rely on repeat donations long-term.”
Could allogeneic cell therapy “super donors” come to the rescue?
Quality starting material is fundamental to all cell and gene therapies. But not all starting material is equal. These differences can happen for a number of reasons and are difficult to anticipate until a person donates his or her cells.
Some individuals who frequently donate their cells have been informally called “super donors.” Could identifying these individuals and working with them repeatedly help manufacturers scale the production of their allogeneic cell therapies?
Those with decades of experience in matching patients with their life-saving donors see a myriad of limitations to this approach.
“If you identify a donor who generates a really good therapy for your product, you would love to go back to them over and over and over again. But, that’s unlikely to be a long-term option,” said Abby McDonald, PhD, Manager, Solutions Development at Be the Match BioTherapies.
“There are so many life events that can influence whether someone is able to donate. For example, they may experience illnesses, relocations or events or make choices that can influence the quality of their donation,” McDonald explained.
Companies that rely too heavily on a small subset of super donors to develop allogenic products introduce an element of risk into their business strategy.
This situation is further complicated by the fact that many donors, especially those who donate repeatedly, are compensated for donating their cells or their “time.” Regulations regarding compensation are very clear in certain parts of the world and less clear in others.
However, what is clear is that regulations in some major markets prohibit compensation of donors. As a result, utilizing cellular material from compensated allogeneic donors could render cell therapies derived from that donor material unusable for clinical and commercial purposes globally.
Furthermore, if compensation hangs in the balance, donors could be less than forthcoming with their health histories. This leads to potential challenges late in the allogneic cell therapy development process, when undisclosed health events potentially result in contamination of manufactured products resulting in adverse outcomes upon infusion.
Registry size, specifically breadth and depth, matters as allogeneic cell therapy growth scales
Expanding the roster of uncompensated donors as broadly as possible offers a more ethically–sound, long-term solution to the scaling problem.
Be The Match BioTherapies leverages the largest and most diverse donor registry in the world – the Be The Match Registry® – to increase access to high quality, compliant cellular starting material. That registry includes more than 20 million HLA–typed donors who have made the decision to donate their cells without compensation.
In the words of Leah, a Be The Match BioTherapies allogeneic donor, “To think that my cells could be developed into a therapy that would help many patients … it’s pretty amazing. Even if my cells were going to a lab for early-stage development, I would have been ok with it because I would be helping to further science.”
Donors like Leah are a promising starting point for the growth still to come – not just for manufacturers, but across the cell and gene therapy supply chain.
“We want to work with manufacturers now while they are developing their therapies, but we also want to work with them in the future as they grow so that they have the capability to scale when they are ready,” Flower added.