Navigating the Dynamic Landscape of Stem Cell Transplantation

In a cobranded episode between Oncology on the Go, hosted by CancerNetwork^®, and the American Society for Transplantation and Cellular Therapy (ASTCT)’s program ASTCT Talks, Mitchell E. Horwitz, MD, highlighted key developments and ongoing initiatives related to allogeneic and cord blood transplantations among patients with different hematologic malignancies. The conversation touched upon the impact of omidubicel-onlv (Omisirge) on patient outcomes, current research on reducing the risk of graft-vs-host disease (GVHD) among transplantation recipients, and strategies for providing effective prophylaxis during treatment, among other topics.

According to Horwitz, omidubicel has served as an “important graft source” for pediatric patients while improving cord blood transplantation for adults since its FDA approval in April 2023 for patients 12 years or older with hematologic malignancies.¹ He also spoke to the importance of the FDA’s approval of the agent in December 2025 for patients with severe aplastic anemia and no compatible donors following reduced intensity conditioning.² Regarding those with severe aplastic anemia, he noted that omidubicel may considerably improve the feasibility of cord blood as a graft source for transplantation.

Beyond these approvals, Horwitz described ongoing work dedicated to reducing the risk of GVHD following cord blood–derived transplantation, citing a pilot study that he and colleagues are conducting to determine the feasibility of adding a costimulatory blocking monoclonal antibody to help further limit this risk. Additionally, he emphasized surveilling for viruses such as Epstein-Barr virus, human herpesvirus 6, and cytomegalovirus to mitigate the risks of delayed immune recovery following transplantation.

“It’s important to have all these [graft sources], whether it be cord blood, mismatched family members, mismatched unrelated donors, and matched siblings…to be made available [and] studied extensively,” Horwitz concluded. “We need to find what the best niche would be for each of these graft sources and make sure that [they] are utilized at the various institutions. The nuances, such as infection prophylaxis or infection monitoring, [should become] familiar to the transplant centers. By doing that, we can continue this trend of having a graft source for everyone and improving outcomes.”

Horwitz is a professor of medicine, hematologic malignancies, and cellular therapy at Duke University School of Medicine as well as a cellular therapy and stem cell transplant specialist at Duke Cancer Institute.

References

1. FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation. News release. FDA. April 17, 2023. Accessed May 20, 2026. bit.ly/3UEO3kp
2. FDA approves first cellular therapy to treat patients with severe aplastic anemia. News release. FDA. December 8, 2025. Accessed May 20, 2026. https://tinyurl.com/yuu377yt